Neuromuscular Clinical Trials

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UPCOMING PROJECTS

  • HEALEY ALS Platform Trial
    Condition studied: amyotrophic lateral sclerosis (ALS)
  • Safety and Efficacy Study of Ravulizumab in Adults with ALS
  • Conditions studied: amyotrophic lateral sclerosis (ALS)
  • Safety and Tolerability of AT-1501 in Adults with ALS
    Conditions studied: amyotrophic lateral sclerosis (ALS)
  • An Extension Study of Treatment with BIIB067 in SOD1-ALS Subjects
    Conditions studied: amyotrophic lateral sclerosis (ALS)
  • Open-label Extension of HYQVIA/HyQvia and GAMMAGARD LIQUID/KIOVIG in CIDP
    Conditions studied: chronic inflammatory demyelinating polyradiculoneuropathy (CIDP)
  • Registry for Patients with Myasthenia Gravis who are on Soliris
  • Conditions studied: Generalized myasthenia gravis (MG)

COMPLETED PROJECTS

  • A Retrospective Study Regarding the Use of Radicava
    Condition Studied: amyotrophic lateral sclerosis (ALS)
  • A Pilot Trial of Leukapheresis in ALS and Controls
    Condition studied: amyotrophic lateral sclerosis (ALS)
  • Efficacy, Safety and Tolerability Trial of Compound ARGX-113 (ADAPT)  
  • Conditions studied: myasthenia gravis

 

For more information about neuromuscular studies and/or other research opportunities, please contact the research team below:

 

 

 


 

T-regulatory Cells in ALS (Tregs in ALS)

This study is a randomized, placebo-controlled, phase 2a trial to study the biological activity, safety, and tolerability of regulatory T Lymphocytes (Tregs) taken and expanded outside of the body and returned back to the same person whose Tregs were removed, given back by IV infusion (intravenously) and in combination with low-dose IL-2 in people with Amyotrophic Lateral Sclerosis (ALS).

  • Study director (local PI): Jason R. Thonhoff, MD, PhD
  • Sponsor: Houston Methodist Hospital System
  • Recruiting?: No
  • Official study title: Phase 2a Study of the Expansion and Infusion of Autologous T-Regulatory Cells in Amyotrophic Lateral Sclerosis
  • ClinicalTrials.gov identifier: NCT04055623
  • Conditions studied: Amyotrophic Lateral Sclerosis (ALS)
  • Intervention Drugs: The first 6-months is the double-blind part of the study where participants are randomized to Treg cell infusions and low dose Interleukin-2 (IL-2) injections OR placebo (inactive) infusions and placebo IL-2 injections. This is followed by a second 6-months of Treg infusions and IL-2 injections for Cohort 1 (6 participants) and Cohort  2 has only the second 6-months of Treg infusions and IL2 injections.
  • Phase: Phase 2a
  • Duration of participation: Up to 8 week screening period and 52 week treatment period
  • IRB #: Pro00022167
  • IRB approval date: 08/07/2019
Eligibility - Inclusion criteria: Patients must meet all of the following criteria to be eligible for study participation:
  • ALS meeting El Escorial criteria for possible, probable, lab- supported probable, or definite ALS.
  • At least 18 years old.
  • Provided informed consent and authorized use of protected health information (PHI) in accordance with national and local patient privacy regulations.
  • Capable of complying with all study procedures, including the study drug delivery procedure, in the Investigator’s opinion.
  • On a stable regimen of riluzole for at least 30 days at the time of screening. If not on riluzole at the time of study entry, willing to refrain from initiation of the agent for the duration of the trial.
  • Patients on edaravone willing to refrain from taking edaravone on the same day as they will receive the Tregs infusion for the duration of the trial. If not on edaravone at the time of study entry, willing to refrain from initiation of the agent for the duration of the trial.
  • Medical record documentation of a decline in ALSFRS-R total score of at least two points in the 90 days prior to screening or at least four points over the 180 days prior to screening.
  • Forced vital capacity (FVC) ≥65% of predicted capacity for age, height, and gender at screening.
  • Patient able and willing to undergo leukapheresis.
Exclusion criteria: Patients meeting any of the following criteria are not eligible for study participation:
  • Presence of any of the following clinical conditions that would interfere with the safe conduct of the study, as determined by the Investigator:
    • Unstable neurological, cardiovascular, cerebrovascular, pulmonary, renal, hepatic, endocrine, or hematologic disease; active malignancy or infectious disease; or other medical illness.
    • Unstable psychiatric illness defined as psychosis (hallucinations or delusions), unstable major depression, or substance abuse within 180 days prior to screening.
    • Persistent asthma, prior history of acute systemic reactions involving IgE-dependent mechanisms, history of angioedema, or history of anaphylactic reactions to any medication.
  • Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3× the upper limit of normal (ULN) , or complete blood count (CBC) with white blood cells (WBC) <4 or >13, hematocrit (HCT) <30 or >54, or platelets <90 or >500 at screening.
  • Serum creatinine >1.8 mg/dL or creatinine clearance <40 mL/min at screening.
  • History of, or positive test result for human immunodeficiency virus (HIV), hepatitis C virus, or hepatitis B virus (i.e. positive for both hepatitis B surface antigen and hepatitis B core antibody) at  screening.
  • Tracheostomy.
  • If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or unwilling to use effective contraception for the duration of the trial and for 90 days after treatment.
  • If male of reproductive capacity, unwilling to use effective contraception for the duration of the trial and for 90 days after treatment.
  • Enrollment in any other interventional study.
  • Treatment with another investigational drug, biological agent, or device within 30 days or 5 half-lives of screening, whichever is longer. Patient participation in an observational/non-interventional clinical study is allowed and to be discussed prior to study enrollment with the Medical Monitor.
  • Prior gene or cell therapy treatments for ALS.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
What is involved?
  • Testing: Neurological and physical examinations, Leukapheresis, FVC and MIP Measurements, ECGs, blood, and urine specimen collection, vital signs
  • Frequency of visits:
      • Up to 8 week screening period
      • 14 visits for randomized controlled portion of the trial
      • 14 visits for open-label treatment period
  • Materials needed prior to evaluation: Prior ALS diagnosis meeting El Escorial criteria for possible, probable, lab-supported probable, or definite ALS
  • Costs: No costs will be charged for any of the study procedures. Parking will be validated for all study visits

 

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Treatment with BIIB067 in SOD1-ALS Subjects

The primary objective of Parts A and B of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of BIIB067 (Tofersen) in adults with ALS. The primary objective of Part C of this study is to evaluate the clinical efficacy of BIIB067 administered to adult participants with ALS and confirmed superoxide dismutase 1 (SOD1) mutation. The secondary objective of Parts A and B of this study is to evaluate the effects of BIIB067 on levels of SOD1 protein in the cerebrospinal fluid (CSF). The secondary objectives of Part C are to evaluate the safety, tolerability, and pharmacodynamic (PD) effects of BIIB067 administered to adult participants with ALS and confirmed SOD1 mutation.

 

  • Study director (local PI): Ericka P. Greene
  • Sponsor: Biogen
  • Recruiting?: Yes
  • Official study title: A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB067 Administered to Adult Subjects With Amyotrophic Lateral Sclerosis and Confirmed Superoxide Dismutase 1 Mutation
  • ClinicalTrials.gov identifier: NCT02623699
  • Conditions studied: Amyotrophic Lateral Sclerosis (ALS)
  • Intervention Drugs: Subjects will be randomized to receive BIIB067 (antisense oligonucleotide inhibitor of SOD1 mRNA) or placebo in a 2:1 (active:placebo) ratio. Study drug (BIIB067 or placebo) will be administered 8 times over approximately 24 weeks.
  • Study Type: Interventional
  • Phase: Phase 1/2/3
  • Duration of participation: Up to 28 day screening period and 24 week treatment period
  • IRB #: Pro00021877
  • IRB approval date: 11/20/2019
Eligibility - Inclusion criteria: 
  • Weakness attributable to ALS and confirmed SOD1 mutation at Screening Visit.
  • SVC ≥ 65% of predicted value as adjusted for sex, age, and height.
  • If taking riluzole, participant must be on a stable dose for ≥30 days prior to Day 1 and expected to remain at that dose until the final study visit.
  • If taking edaravone, participant must have initiated edaravone ≥60 days (2 treatment cycles) prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study. Edaravone may not be administered on dosing days of this study.
  • Medically able to undergo the study procedures and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.
 Exclusion criteria:
  • History of or positive test result for human immunodeficiency virus.
  • Current hepatitis C infection (defined as positive hepatitis C virus [HCV] antibody and detectable HCV ribonucleic acid [RNA]). Participants with positive HCV antibody and undetectable HCV RNA are eligible to participate in the study (United States Centers for Disease Control and Prevention).
  • Current hepatitis B infection (defined as positive for HBsAg and/or anti-HBc). Participants with immunity to hepatitis B from previous natural infection (defined as negative HBsAg, positive anti-HBc, and positive anti-HBs) or vaccination (defined as negative HBsAg, negative anti-HBc, and positive anti-HBs) are eligible to participate in the study.
  • Treatment with another investigational drug (including investigational drugs for ALS through compassionate use programs), biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering RNA, stem cell therapy, or gene therapy is allowed.
  • Current enrollment in any other interventional study.
  • Current or recent (within 1 month) use, or anticipated need, in the opinion of the Investigator, of copper (II) (diacetyl-bis(N4-methylthiosemicarbazone)) or pyrimethamine.
  • Current or anticipated need, in the opinion of the Investigator, of a DPS during the study period.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
What is involved?
  • Procedures: Neurological and physical examinations, FVC and SVC, HHD,  ECGs, blood, and urine specimen collection, vital signs
  • Frequency of visits:
      • Up to 28 day screening period
      • 9 visits for randomized controlled portion of the trial
      • 1 visit for safety follow-up
  • Costs: No costs will be charged for any of the study procedures. Parking will be validated for all study visits

 

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Open-label Oral Edaravone for Treatment of ALS

The objective of this study is to evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral Sclerosis (ALS) over 24 and 48 weeks.

  •  Study director (local PI): Jason R. Thonhoff, MD, PhD
  • Sponsor: Mitsubishi Tanabe Pharma Development America, Inc.
  • Recruiting?: Yes
  • Official study title: Safety Study of Oral Edaravone Administered in Subjects With ALS
  • ClinicalTrials.gov identifier: NCT04165824
  • Conditions studied: Amyotrophic Lateral Sclerosis (ALS)
  • Intervention Drugs: Subjects will receive open label oral edaravone administered 14 times over approximately 48 weeks.
  • Study Type: Interventional
  • Phase: Phase 3
  • Duration of participation: Up to 21 day screening period, 48 week treatment period and 2 week safety follow-up period.
  • IRB #: Pro00023214
  • IRB approval date: 12/31/2019
Eligibility - Inclusion criteria:
  • Subjects will be male or female, ≥ 18 to 75 years of age at the time the ICF is signed
  • Subjects will be diagnosed with Definite ALS, Probable ALS, Probable laboratory-supported ALS, or Possible ALS according to the El Escorial revised criteria for the diagnosis of ALS.
  • Subjects will be living and functioning independently (eg, able to eat, excrete, ambulate independently without assistance of others). The use of supportive tools and adaptive utensil is allowed
  • Subjects will have a baseline forced vital capacity percentage (%FVC) ≥ 70%.
  • Subjects whose first symptom of ALS occurred within 3 years of the time of providing written informed consent.

 Exclusion criteria: 

  • Subjects who have the presence or history of any clinically significant disease (except ALS) that could interfere with the objectives of the study (the assessment of safety and efficacy) or the safety of the subject, as judged by the Investigator.
  • Subjects of childbearing potential unwilling to use acceptable method of contraception from the screening visit until 3 months after the last dose of study medication. Subjects who are sexually active who do not agree to use contraception during the study period.
  • Subjects who are female and pregnant (a positive pregnancy test) or lactating at the screening visit (Visit 1).
  • Subjects who have a significant risk of suicidality. Subjects with any suicidal behavior or suicidal ideation of type 4 (active suicidal ideation with some intent to act, without a specific plan) or type 5 (active suicidal ideation with specific plan and intent) based on the Columbia-Suicide Severity Rating Scale (C-SSRS) within the 3 months before the screening visit.
  • Subjects who have ALT or AST elevations greater than 2 times the ULN at screening.
  • Subjects with a Glomerular Filtration Rate (GFR) <30 mL/Min Per 1.73 m2.
  • Subjects with history of hypersensitivity to edaravone, any of the additives or inactive ingredients of edaravone, or sulfites.
  • Subjects with hereditary fructose intolerance.
  • Subjects who participated in another study and were administered an investigational product within 1 month or 5 half-lives of the investigational agent, whichever is longer before providing informed consent for the present study.
  • Subjects who are unable to take their medications orally.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
What is involved?
  • Procedures: Neurological and physical examinations, FVC,  ECGs, blood, and urine specimen collection, vital signs
  • Frequency of visits:
      • Up to 21 day screening period
      • 5 in-person visits and 8 telephone visits for open-label treatment period
      • 1 visit for safety follow-up
  • Costs: No costs will be charged for any of the study procedures. Parking will be validated for all study visits. Subjects will be given $50 per visit to cover the cost for time and travel.

 

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Radicava/Edaravone Findings in Biomarkers From ALS (REFINE-ALS)

REFINE-ALS is a prospective, observational, longitudinal, multicenter study designed to identify biomarkers to serve as quantifiable biological non-clinical measures of Edaravone effects in ALS. Epigenetic and protein biomarkers will also be investigated.

 

  •  Study director (local PI): Sheetal Shroff, MD
  • Sponsor: Mitsubishi Tanabe Pharma Development America, Inc.
  • Recruiting?: No
  • Official study title: Radicava® (Edaravone) Findings in Biomarkers From ALS (REFINE-ALS)
  • ClinicalTrials.gov identifier: NCT04259255
  • Conditions studied: Amyotrophic Lateral Sclerosis (ALS)
  • Intervention Drugs: During an estimated 12-month period, eligible participants who are prescribed Edaravone within the approved indication will be invited to participate in the study.
  • Study Type: Interventional
  • Phase: Phase 4
  • Duration of participation: Participants will be followed from enrollment up to 24 weeks after treatment initiation (6 treatment cycles - 28 days per cycle, corresponding to a treatment period of approximately 24 weeks) or premature study discontinuation.
  • IRB #: Pro00023890
  • IRB approval date: 04/29/2020
Eligibility - Inclusion criteria:
  • Male and female aged 18 years or older at enrollment
  • Sporadic or familial ALS diagnosed as possible, probable, probable-laboratory supported or definite as defined by the World Federation of Neurology revised El Escorial criteria
  • Decision made to prescribe Edaravone prior to screening
  • Participant will likely be able to obtain commercial Edaravone and likely to complete 6 cycles of treatment, per site investigator estimation
  • Participant either naïve to Edaravone or who did not receive any Edaravone does within 1 month prior to screening
  • Signed informed consent by the subject, or a witness if a subject cannot read or write or is physically unable to talk or write, obtained before any study-related activities are undertaken
 Exclusion criteria:
  • Participant with a contraindication to Edaravone
  • Participant is participating in an interventional clinical trial
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
What is involved?
  • Procedures: Vital capacity, King’s Clinical Staging,  Strength testing, blood, and urine specimen collection, vital signs
  • Frequency of visits:
      • Up to 12 week screening period
      • 7 visits for 24-week treatment period
  • Costs: Study funds will pay for study-related procedures that are done only for research. Subjects will be responsible for payment of any deductibles and co-payments required by their insurer for this routine care or other billed care.

 

  • Costs: No costs will be charged for any of the study procedures. Parking will be validated for all study visits

 

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HYQVIA/HyQvia and GAMMAGARD LIQUID/KIOVIG in CIDP

This is a Phase III, prospective, multicenter study with two study parts (epochs) to evaluate the efficacy, safety, and tolerability of HYQVIA/HyQvia (IGI, 10% with recombinant human hyaluronidase (rHuPH20) administered subcutaneously) for maintenance therapy to prevent relapse (Epoch 1) and GAMMAGARD LIQUID/KIOVIG (immunoglobulin G intravenous (IGIV) 10% administered intravenously) for the treatment of CIDP (Epoch 2). This study will enroll adult subjects with a confirmed diagnosis of CIDP and who have remained on a stable dosing regimen (monthly equivalent dose of 0.4 to 2.4 g/kg body weight (BW) with a dosing interval of 2 to 6 weeks) of IGIV therapy for at least 3 months prior to screening. In this double-blind, placebo-controlled phase of the study (Epoch 1), eligible subjects will be randomized in a 1:1 ratio to receive either HYQVIA/HyQvia or 0.25% albumin placebo solution with rHuPH20 every two, three, or four weeks in a double-blind fashion for a period of 6 months or until relapse. Subjects who relapse during Epoch 1 will enter Epoch 2 to receive IGIV treatment for a period of 6 months.

  • Study director (local PI): Ericka P. Greene, MD
  • Sponsor: Baxalta now part of Shire
  • Recruiting?: Yes
  • Official study title: A Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Immune Globulin Infusion 10% (Human) With Recombinant Human Hyaluronidase (HYQVIA/HyQvia) and Immune Globulin Infusion (Human), 10% (GAMMAGARD LIQUID/KIOVIG) for the Treatment of Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
  • ClinicalTrials.gov identifier: NCT025497170
  • Conditions studied: Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
  • Intervention Drugs: Participants will receive SC HYQVIA/HyQvia at a dose of 80 Unit per gram (U/g) immunoglobulin (IgG) which will be same as the participants pre-randomization monthly equivalent IgG dose (or at matching infusion volume for participants in the placebo group) when administered at a dosing frequency of every 2, 3, or 4 weeks for 6 months or until relapse. OR
    Participants will receive sequential 0.25% albumin placebo with rHuPH20 at a dose of 80U/g IgG subcutaneously for 6 months or until relapse. Dosing regimen for placebo treatment will be the same as the participant's pre-randomization monthly equivalent IgG infusion volume when administered every 2, 3, or 4 weeks.
    OR
    Participants will receive an induction dose of 2 Gram per kilogram (g/kg) Intravenous immunoglobulin G (IGIV), followed by maintenance infusions at the same monthly dose as the participant's pre-randomization IgG dose, every 3 weeks for 6 months or until relapse.

  • Study Type: Interventional
  • Phase: Phase 3
  • Duration of participation: The maximum overall duration of this study is estimated to be approximately 68 months from study initiation (ie, first subject enrolled) to study completion (ie, last subject last visit), including:
    • An enrollment period of approximately 61 months
    • A screening/baseline period of up to 8 weeks
    • Epoch 1 (subcutaneous [SC] treatment period) of 6 months
    • Epoch 2 (intravenous [IV] treatment period) of 6 months - for subjects who relapse during Epoch 1.
  • IRB #: Pro00022113
  • IRB approval date: 12/31/2019

 

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Expanded Access Protocol for Firdapse

The primary objective of the study to provide patients with CMS access to amifampridine phosphate therapy until the product becomes commercially available or development is discontinued.
The secondary objective of the study is to assess the long-term safety of amifampridine phosphate in patients with CMS

 

  •  Study director (local PI): Sheetal Shroff, MD
  • Sponsor: Catalyst pharmaceuticals, Inc.
  • Recruiting?: No
  • Official study title: An Open-Label, Expanded Access Protocol for Amifampridine Phosphate Treatment in Patients With Congenital Myasthenic Syndrome (CMS)
  • ClinicalTrials.gov identifier: NCT02189720
  • Conditions studied: Congenital Myasthenic Syndrome (CMS)
  • Intervention Drugs: Subjects will receive amifampridine phosphate based on Investigator assessment of optimal neuromuscular benefit. Doses range from 30 mg to 80 mg, divided into doses taken 3 to 4 times per day, with a maximum single dose of 20 mg.
  • Study Type: Interventional
  • Phase: Phase 4
  • Duration of participation: Until amifampridine phosphate becomes commercially available for these indications or until further development for the indication is discontinued.
  • IRB #: Pro00013058
  • IRB approval date: 02/22/2016

Eligibility - Inclusion criteria: 

  • A diagnosis of sporadic or familial ALS, defined by the El Escorial criteria (possible, laboratory-supported probable, probable, or definite ALS).
  • ALS onset ≤ 36 months from Screening.
  • Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment.
  • Upright slow vital capacity ≥ 65% predicted at Screening.
  • If on riluzole, participant must be on a stable dose for 30 days; if on edaravone, participant must be on a stable dose for 60 days (2 cycles).
  • Body weight ≥ 40 kilograms at Screening.
  • Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

 Exclusion criteria: 

  • History of Neisseria meningitidis infection.
  • Human immunodeficiency virus (HIV) infection (evidenced by HIV 1 or HIV 2 antibody titer).
  • Dependence on invasive or non-invasive mechanical ventilation.
  • Previously or currently treated with a complement inhibitor.
  • Exposure to an investigational drug or device within 30 days of Screening or 5 half-lives of the study drug, whichever is greater.

What is involved?

  • Procedures: Physical examination, ECG, blood, and urine specimen collection, vital signs, questionnaires
  • Frequency of visits: Patients will report to the clinic at least once per year, based on the Neurologist’s discretion.
  • Costs: Patients or their insurer will be billed for the costs of any standard medical care they receive during their participation in the study, and they will be responsible for any associated co-payments and deductibles. The study sponsor will supply and pay for the cost of supplying the study drug.

 

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Safety and Efficacy Study of Ravulizumab in Adults with Generalized Myasthenia Gravis

The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab for the treatment of participants with generalized myasthenia gravis (gMG).

  •  Study director (local PI): Ericka P. Greene, MD
  • Sponsor: Alexion Pharmaceuticals
  • Recruiting?: Yes
  • Official study title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis
  • ClinicalTrials.gov identifier: NCT03920293
  • Conditions studied: Myasthenia Gravis (MG)
  • Intervention Drugs: Patients randomized to the ravulizumab group will receive a blinded loading dose of ravulizumab on Day 1, followed by blinded maintenance doses of ravulizumab on Day 15 (Week 2) and q8w thereafter, for a total of 18 weeks of treatment. Patients randomized to placebo will receive a blinded dose of placebo on Day 1, followed by blinded doses of placebo on Day 15 (Week 2) and q8w thereafter, for a total of 18 weeks. Both ravulizumab and placebo will be administered by intravenous infusion.

After the 26-Week Randomized-Controlled Period and assessments on Day 183 (Week 26), patients in the placebo group will receive a blinded loading dose of ravulizumab and patients in the ravulizumab group will receive a blinded ravulizumab dose of 900 mg; the 900-mg dose was chosen to ensure maintenance of complete C5 inhibition until the next scheduled maintenance dose at Week 28 (Day 197). Starting at Week 28, all patients will begin open-label ravulizumab maintenance doses q8w.

  • Study Type: Interventional
  • Phase: Phase 3
  • Duration of participation: Up to 2 week screening period, 26 week treatment period and 132week open-label extension period.
  • IRB #: Pro00021711
  • IRB approval date: 08/01/2019

Eligibility - Inclusion criteria: 

  • Diagnosed with Myasthenia Gravis at least 6 months (180 days) prior to the date of the Screening Visit as confirmed by specific criteria.
  • Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at screening.
  • MG-ADL profile must be ≥ 6 at screening and randomization (Day 1).
  • Vaccinated against meningococcal infections within 3 years prior to, or at the time of, initiating study drug to reduce the risk of meningococcal infection (N meningitidis).

 Exclusion criteria: 

  • Any active or untreated thymoma. History of thymic carcinoma or thymic malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥ 5 years before screening.
  • History of thymectomy within the 12 months prior to screening.
  • History of N meningitidis infection.
  • Use of the following within the time period specified below:
  • IV immunoglobulin within 4 weeks of randomization
  • Use of plasma exchange within 4 weeks of randomization
  • Use of rituximab within 6 months of screening
  • Participants who have received previous treatment with complement inhibitors (for example, eculizumab).

 NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

What is involved?

  • Procedures: Neurological and physical examinations, FVC,  ECGs, blood, and urine specimen collection, vital signs and pulse oximetry, and questionnaires
  • Frequency of visits:
      • Up to 2 week screening period
      • 12 visits for randomization-controlled treatment period
      • 18 visits for open-label extension
  • Costs: No costs will be charged for any of the study procedures. Parking will be validated for all study visits. Subjects will be given $50 per visit to cover the cost for time and travel.

 

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Safety and Efficacy Study of Rozanolixizumab in Adult Patients with Generalized Myasthenia Gravis (MycarinG)

The purpose of the MycarinGstudy is to demonstrate the clinical efficacy and to assess safety and tolerability of rozanolixizumab in patients with generalized myasthenia gravis (MG).

 

  • Study director (local PI): Sheetal Shroff, MD
  • Sponsor: UCB Biopharma S.P.R.L
  • Recruiting?: Yes
  • Official study title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
  • ClinicalTrials.gov identifier: NCT03971422
  • Conditions studied: Myasthenia Gravis (MG)
  • Intervention Drugs: Subjects will receive either 7mg/kg rozanolixizumab, 10 mg/kg rozanolixizumab or a placebo dose during weekly infusion at Visits 2, 4, 5, 6, 8, and 9.
  • Study Type: Interventional
  • Phase: Phase 3
  • Duration of participation: Up to 4 week screening period, 6 week treatment period and 8 week observational period.
  • IRB #: Pro00022515
  • IRB approval date: 09/27/2019

Eligibility - Inclusion criteria: 

  • Study participant must be ≥18 years of age, at the time of signing the informed consent
  • Study participant has documented diagnosis of generalized myasthenia gravis (gMG) at Visit 1, based on study participant's history and supported by previous evaluations
  • Study participant has a confirmed positive record of autoantibodies against acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) prior to Visit 1
  • Study participant has Myasthenia Gravis Foundation of America (MGFA) Class II to IVa at Visit 1
  • Study participant with a myasthenia gravis-activities of daily living (MG-ADL) score of at least 3 AND a quantitative myasthenia gravis (QMG) score of at least 11 at Visit 1 and at Baseline

 Exclusion criteria: 

  • Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of investigational medicinal product (IMP)
  • Study participant has experienced hypersensitivity reaction after exposure to other anti-neonatal Fc receptor (FcRn) drugs
  • Study participant with severe (defined as Grade 3 on the MG-ADL scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis a Visit 1

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

What is involved?

  • Procedures: Neurological and physical examinations, spirometry, ECG, blood and urine specimen collection, vital signs, and questionnaires
  • Frequency of visits: participation in the trial will last up to approximately 18 weeks, from the beginning of the Screening Period to the end of the Observation Period. During this trial, there will be approximately 14 visits to the trial site.
  • Costs: No costs will be charged for any of the study procedures. Parking will be validated for all study visits. Subjects will be given $43 per visit to cover the cost for time and travel.

 

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Extension Study of Rozanolixizumab in Adult Patients with Generalized Myasthenia Gravis (MycarinG Extension)

The purpose of the MycarinGstudy is to demonstrate the clinical efficacy and to assess safety and tolerability of rozanolixizumab in patients with generalized myasthenia gravis (MG).

  •  Study director (local PI): Sheetal Shroff, MD
  • Sponsor: UCB Biopharma S.P.R.L
  • Recruiting?: Yes
  • Official study title: A Randomized, Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
  • ClinicalTrials.gov identifier: NCT04124965
  • Conditions studied: Myasthenia Gravis (MG)
  • Intervention Drugs: Subjects will receive 52 doses of rozanolixizumab during weekly infusions.
  • Study Type: Interventional
  • Phase: Phase 3
  • Duration of participation: The treatment period is 52 weeks and the observation period is 8 weeks.
  • IRB #: Pro00023823
  • IRB approval date: 03/23/2020

Eligibility - Inclusion criteria: 

  • Participant must be ≥18 years of age at the time of signing the informed consent
  • Participant was eligible for MG0003 [NCT03971422] at the time of enrollment into MG0003 and the participant either completed the Observation Period of MG0003 or required rescue therapy during the Observation Period of MG0003
  • Body weight >35 kg at Visit 1
  • Study participants may be male or female
  • A male study participant must agree to use contraception
  • Female study participants of childbearing potential must agree to use a highly effective method of birth control
  • A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:
    • Not a woman of childbearing potential (WOCBP) OR
    • A WOCBP who agrees to follow the contraceptive guidance

 Exclusion criteria: 

  • Evidence of active or latent tuberculosis (TB) as documented by medical history and examination, if applicable, chest X-rays (posterior anterior and lateral), and TB testing by a positive (not indeterminate) QuantiFERON®-TB Gold test
  • Participant has received a live vaccination within 8 weeks prior to the Baseline visit; or intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of study medication
  • Study participant has experienced hypersensitivity reaction after exposure to other anti-neonatal Fc receptor (FcRn) drugs
  • Study participant with severe (defined as Grade 3 on the myasthenia gravis-activates of daily living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis
  • Participant has absolute neutrophil count <1500 cells/mm3
  • Participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at randomization, and could jeopardize or compromise the study participant's ability to participate in this study
  • Participant has 12-lead electrocardiogram (ECG) with findings considered to be clinically significant upon medical review. The clinical significance of the findings needs to be assessed by the Investigator to determine eligibility, and any queries regarding continuation of the study participant must be addressed with the Medical Monitor
  • Study participant has renal impairment, defined as serum creatinine level of ≥1.4 mg/dL for females and ≥1.5mg/dL for males
  • Study participant has >2x upper limit of normal (ULN) of any of the following at Visit 1: alanine aminotransferase (ALT), aspartate aminotransferase (AST), alkaline phosphatase (ALP), or bilirubin >1.5xULN (isolated bilirubin >1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin <35%). If study participant has elevations only in total bilirubin that are >ULN and <1.5xULN, fractionate bilirubin to identify possible undiagnosed Gilbert's syndrome (ie, direct bilirubin <35%). For randomized study participants with a baseline result >ULN for ALT, AST, ALP, or total bilirubin but <1.5xULN, a baseline diagnosis and/or the cause of any clinically meaningful elevation must be understood and recorded in the electronic Case Report form (eCRF). Tests that result in ALT, AST, or ALP up to 25% above the exclusion limit (>2xULN) may be repeated once for confirmation
  • Study participant has positive human immunodeficiency virus antibody test
  • Study participant met any mandatory withdrawal or mandatory study drug discontinuation criteria MG0003 [NCT03971422] or discontinued study medication in MG0003, with the exception of discontinuation due to a need for rescue treatment
  • Study participant is not considered capable of adhering to the protocol visit schedule, or medication intake according to the judgment of the Investigator
  • Study participant has a lifetime history of suicide attempt (including an active attempt, interrupted attempt, or aborted attempt), or had suicidal ideation since the last visit in MG0003 as indicated by a positive response (Yes) to either Question 4 or Question 5 of the Columbia-Suicide Severity Rating Scale (C-SSRS) at Screening

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

What is involved?

  • Procedures: Neurological and physical examinations, spirometry, ECG, blood and urine specimen collection, vital signs, and questionnaires
  • Frequency of visits: subjects will have come to the site for a minimum of 52 visits during the Treatment Period and 1 visit during the Observation Period.
  • Costs: No costs will be charged for any of the study procedures. Parking will be validated for all study visits. Subjects will be given $43 per visit to cover the cost for time and travel.

 

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Amifampridine Phosphate Study in Patients with MuSK Antibody Positive Myasthenia Gravis, and a Sample of AChR Antibody Positive Myasthenia Gravis

Efficacy and safety of amifampridine phosphate in improving the activities of daily living for patients with antibody positive MuSK myasthenia gravis.

 

  •  Study director (local PI): Ericka P. Greene, MD
  • Sponsor: Catalyst pharmaceuticals, Inc.
  • Recruiting?: Yes
  • Official study title: A Randomized, Placebo-control, Parallel Group Study to Evaluate the Effect of Amifampridine Phosphate in Patients With MuSK Antibody Positive Myasthenia Gravis, and a Sample of AChR Antibody Positive Myasthenia Gravis Patients
  • ClinicalTrials.gov identifier: NCT03304054
  • Conditions studied: Antibody positive MuSK Myasthenia Gravis (MG)
  • Intervention Drugs: During the study, subjects will receive increasing doses of amifampridine every 3 to 4 days until their condition is stable. They will need to come to the clinic 4 times during the study. The study will last approximately 45-52 days.
  • Study Type: Interventional
  • Phase: 3
  • Duration of participation: The study will last approximately 45-52 days.
  • IRB #: Pro00018850
  • IRB approval date: 06/04/2018

Eligibility - Inclusion criteria: 

  • Willing and able to provide written informed consent after the nature of the study has been explained and before the start of any research-related procedures.
  • Male or female ≥18 years of age.
  • Positive serologic test for anti-MuSK antibodies or anti-AChR antibodies as confirmed at Screening or by previous antibody test, with report available.
  • Confirmatory EMG or EMG report.
  • Myasthenia Gravis Foundation of America (MGFA) Class II to IV at Screening.
  • MG-ADL score of ≥6 at Screening, with more than 50% of this score attributed to non-ocular items.
  • Patients receiving steroids or pyridostigmine should not have any modification of drug regimen during the month before Screening.
  • Female patients of childbearing potential must have a negative pregnancy test (serum human chorionic gonadotropin [HCG] at screening); and must practice an effective, reliable contraceptive regimen during the study and for up to 30 days following discontinuation of treatment.
  • Ability to participate in the study based on overall health of the patient and disease prognosis, as applicable, in the opinion of the Investigator; and able to comply with all requirements of the protocol, including completion of study questionnaires.

 Exclusion criteria: 

  • Epilepsy and currently on medication.
  • Concomitant use of medicinal products with a known potential to cause QTc prolongation.
  • Patients with long QT syndromes.
  • History of thymectomy within 12 months before Screening.
  • An electrocardiogram (ECG) within 6 months before starting treatment that shows clinically significant abnormalities, in the opinion of the Investigator.
  • Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.
  • Patients receiving immunomodulatory treatment (e.g. plasma exchange [PE], therapeutic plasma exchange [TPE], intravenous immunoglobulin G [IVIG]) should not have any treatment in the previous 4 weeks prior to Randomization or at any time during the study.
  • Use of rituximab or other similar biologic medications for immunomodulation within 6 months prior to Screening.
  • Treatment with an investigational drug (other than amifampridine) or device within 30 days before Screening or while participating in this study.
  • Any medical condition that, in the opinion of the Investigator, might interfere with the patient's participation in the study, poses an added risk for the patient, or confound the assessment of the patient.
  • History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s).

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

What is involved?

  • Procedures: Physical examinations, spirometry, ECG, blood and urine specimen collection, vital signs, electromyogram and questionnaires
  • Frequency of visits: There will be 4 clinic visits over approximately 45-52 days.
  • Costs: The study drug will be given at no charge and subjects will not have to pay for any study tests or procedures. Subjects will receive financial support for transportation, parking and some other expenses for their participation in the study.

 

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Open Label Extension Study of Amifampridine Phosphate in Patients with MuSK Antibody Positive and AChR Antibody Positive Myasthenia Gravis

Evaluate the long-term safety of amifampridine phosphate in the symptomatic relief of antibody positive MuSK-MG.

  • Study director (local PI): Ericka P. Greene, MD
  • Sponsor: Catalyst pharmaceuticals, Inc.
  • Recruiting?: Yes
  • Official study title: Long Term Safety Study of Amifampridine Phosphate in Patients With MuSK Antibody Positive and AChR (Acetylcholine Receptor) Antibody Positive Myasthenia Gravis
  • ClinicalTrials.gov identifier: NCT03579966
  • Conditions studied: Antibody positive MuSK Myasthenia Gravis (MG)
  • Intervention Drugs: During the study, subjects will receive tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day
  • Study Type: Interventional
  • Phase: N/A
  • Duration of participation: Amifampridine will be continued as long as patients benefit from the treatment until the drug is approved by Regulatory Agencies or until the development of the drug is discontinued for this indication.
  • IRB #: Pro00018871
  • IRB approval date: 07/17/2018

Eligibility
Inclusion criteria: 

  • Participated in the MSK-002 study

Exclusion criteria: 

  • Epilepsy and currently on medication
  • Clinically significant abnormalities in ECG, in the opinion of the Investigator

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

What is involved?

  • Procedures: Physical examinations, spirometry, ECG, blood and urine specimen collection, vital signs, and questionnaires
  • Frequency of visits: Patients will be seen in the clinic at the end of Months 3, 6, 9, 12, 15, 21, 27, 33 and 39.
Costs: The study drug will be given at no charge and subjects will not have to pay for any study tests or procedures. Subjects will receive financial support for transportation, parking and some other expenses for their participation in the study.


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Join a Research Study

 

 

 

Biological Markers Study for ALS

Evaluate the long-term safety of amifampridine phosphate in the symptomatic relief of antibody positive MuSK-MG.

 

  • Study director (local PI): Stanley H. Appel, MD
  • Recruiting?: Yes
  • Official study title: Biological Markers for Nervous System Immune and Free Radical-Mediated Processes in Amyotrophic Lateral Sclerosis (ALS)
  • ClinicalTrials.gov identifier: N/A
  • Conditions studied: Amyotrophic Lateral Sclerosis (ALS)
  • Study Type: Observational
  • Phase: N/A
  • Duration of participation: Subjects will be in the study as long as they are a patient of the Houston Methodist Neurological Institute or until they choose to withdraw consent. Samples are kept for up to 15 years and used randomly to conduct a variety of tests.
  • IRB #: Pro00018871
  • IRB approval date: 07/17/2018

Eligibility
Inclusion criteria: 

  • Individuals with ALS, at least 18 years of age, capable of giving informed consent (or legal representative has provided that consent) for one or more of the following:
    • Collection and storage of 24-80 cc of blood at 3-6 month intervals, for sole use in laboratory experiments (sub-study quantities of blood may differ, specified in consent form.)
    • Collection and storage of 60-100 cc of urine at 3-6 month intervals, for sole use in laboratory experiments
    • One time release of 2-5 cc of CSF, originally collected for non-research purposes, for storage and eventual use in laboratory experiments
    • One time donation of tissue from a skin biopsy
    • One time donation of tissue (CNS and other tissues) at autopsy, for storage and eventual use in laboratory experiments
    • In addition, patients or legal representative must be able to give consent allowing researchers to utilize information regarding their disease, and certain personal identifiers (e.g., age, gender, degree of disability from disease, length of disease) that allows those researchers to interpret their experimental results in appropriate clinical context
  • Individuals without ALS, but who have another neurologic disease (e.g., Alzheimer's disease, Parkinson's disease, etc.). These "disease control" individuals must be at least 18 years old, and capable of giving informed consent (or legal representative has provided that consent) for one or more of the following:
    • One time collection and storage of 24-80 cc of blood, for eventual use in laboratory experiments (sub-study quantities of blood may differ, specified in consent form.)
    • Collection and storage of 60-100 cc of urine at 3-6 month intervals, for sole use in laboratory experiments
    • One time release of 2-5 cc of CSF, originally collected for non-research purposes, for storage and eventual use in laboratory experiments.
    • One time donation of tissue from a skin biopsy
    • One-time donation of tissue (CNS and other tissues) at autopsy for storage and eventual use in laboratory experiments.
    • In addition, patients must be able to give consent allowing researchers to utilize information regarding their disease, and certain personal identifiers (e.g., age, gender, disease type, length of disease) that allows those researchers to interpret their experimental results in appropriate clinical context
  • Healthy, normal volunteers (usually spouses of patients with ALS but not excluding employees who request to be part of this research), at least 18 years of age, and capable of giving informed consent for the following:
  • One-time collection and storage of 24-80 cc of blood, for eventual use in laboratory experiments (sub-study quantities of blood may differ, specified in consent form.)
  • Collection and storage of 60-100 cc of urine at 3-6 month intervals, for sole use in laboratory experiments
  • Permission to reveal certain personal identifiers (e.g., age, gender) that allows researchers to interpret their experimental results in appropriate clinical context.
  • One time donation of tissue from a skin biopsy

Exclusion criteria: 

  • Subjects under age 18
  • Subjects or legal representative unable or unwilling to provide informed consent for collection of specimens (blood, post-mortem tissue)
  • Next-of-kin or legal representative unable or unwilling to provide informed consent for collection of specimens (blood, post-mortem tissue)
  • Subjects unable or unwilling to provide informed consent for use of other biological specimens (CSF)

What is involved?

  • Procedures: Blood, urine, CSF, and tissue donation
  • Frequency of visits: Subjects will be in the study as long as they are a patient of the Houston Methodist Neurological Institute or until they choose to withdraw consent.
  • Costs: The study will cover the cost of collecting, receiving and processing the samples. Subjects will not be paid for taking part in the study.

 

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Compilations of Clinical Findings in ALS

The purpose of this study is to maintain a database of clinical information about people with Amyotrophic Lateral Sclerosis (ALS)

  • Study director (local PI): Stanley H. Appel, MD
  • Recruiting?: Yes
  • Official study title: Compilations of Clinical Findings in Amyotrophic Lateral Sclerosis (ALS)
  • ClinicalTrials.gov identifier: N/A
  • Conditions studied: Amyotrophic Lateral Sclerosis (ALS)
  • Study Type: Observational
  • Phase: N/A
  • Duration of participation: Subject’s data will be maintained in this computer program for as long as research is needed to find treatments and/or a cure for ALS.
  • IRB #: Pro00001058
  • IRB approval date: 10/08/2010

Eligibility - Inclusion criteria: 

  • Individuals with ALS, at least 18 years of age, capable of giving authorization (or legal representative has provided that authorization) for one or more of the following:
    Collection and storage of clinical findings at 3-6 month intervals allowing researchers to utilize information regarding their disease, and certain personal identifiers (e.g., age, gender, degree of disability from disease, length of disease) that allows those researchers to interpret their results in appropriate clinical context

Exclusion criteria: 

  • Subjects under age 18
  • Subjects or legal representative unable or unwilling to provide authorization for collection of data
  • Next-of-kin or legal representative unable or unwilling to provide authorization for collection of data

What is involved?

  • Procedures: Procedures will include: review of medical records and entry into the secure data base.
  • Frequency of visits: All of the procedures performed will be part of the subjects’ routine care. The research part of the study is the maintenance of this information in a dedicated computer program. Subjects’ data will be maintained in a computer program for as long as research is needed to find treatments and/or a cure for ALS.
  • Costs: The study will cover the cost of all study activities.


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Genomic Translation for ALS Clinical Care

The purpose of this study is to uncover factors causing ALS variability in order to better diagnose and treat ALS/MND.

 

  • Study director (local PI): Stanley H. Appel, MD
  • Recruiting?: Yes
  • Official study title: Genomic Translation for ALS Clinical Care
  • ClinicalTrials.gov identifier: N/A
  • Conditions studied: Amyotrophic Lateral Sclerosis (ALS) and Motor Neuron Disease (MND)
  • Study Type: Observational
  • Phase: N/A
  • Duration of participation: The study will last 3 years. Subjects’ samples and data will be kept and used indefinitely.
  • IRB #: Pro00015886
  • IRB approval date: 02/10/2017

Eligibility - Inclusion criteria: 

  • Men or women of any race or ethnicity aged 18 or older
  • Diagnosis of familial or sporadic ALS (definite, probable, or possible according to El Escorial Criteria, Appendix 1), or those with primary lateral sclerosis or progressive bulbar/muscular atrophy forms of motor neuron disease. All-comers with ALS/MND should be enrolled without regard to familial vs sporadic or gene mutation status (i.e. participants with known gene mutations should still be enrolled), or phenotype.
  • Capable of providing informed consent and following study procedures (in the case that a subject lacks the ability to provide informed consent, informed consent will be sought from the subject’s surrogate representative).
  • Willing to return to clinic site (or another participating center) for follow-up care.

Exclusion criteria:

  • Invasive ventilation (i.e. tracheostomy) in place.
  • Non-invasive ventilation dependent (defined as >22 hours per day)
  • Pregnancy.
  • Known HIV, chronic Hepatitis B, or Hepatitis C (because cells will be frozen down for future cell line generation).

What is involved?

  • Procedures: Procedures will include: Blood collection, spirometry, neurological exam, and questionnaires
  • Frequency of visits: Study visits are every 3-4 months for up to 3 years.
  • Costs: The study will cover the cost of all study activities.


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Neuromuscular Observational Research Data Hub (MOVR)

The Muscular Dystrophy Association (MDA) wants to collect information about individuals with neuromuscular disease to better understand the disease progression and ultimately improve the medical care, quality of life, and survival of those with neuromuscular disease.

 

  • Study director (local PI): Ericka P. Greene, MD
  • Sponsor: Muscular Dystrophy Association (MDA)
  • Recruiting?: Yes
  • Official study title: Muscular Dystrophy Association Neuromuscular Observational Research (MOVR) Data Hub Protocol
  • ClinicalTrials.gov identifier: N/A
  • Conditions studied: Amyotrophic Lateral Sclerosis (ALS), Spinal Muscular Atrophy (SMA), Duchenne Muscular Dystrophy (DMD), and Becker Muscular Dystrophy (BM)
  • Study Type: Observational
  • Phase: N/A
  • Duration of participation: Each person who participates in the MOVR Data Hub will have his/her information collected for as long as the person is being seen at an MDA Care Center and information is still being collected, unless and until the person requests that the information no longer be provided to the MOVR Data Hub. 
  • IRB #: Pro00021554
  • IRB approval date: 05/03/2019

Eligibility - Inclusion criteria: 

  • Men or women of any age
  • Diagnosis ALS, DMD, BMD, and/or SMA

Exclusion criteria:

  • There are not exclusion criteria for patients with neuromuscular disease

What is involved?

  • Procedures: There are no additional or different medical treatments, medical procedures, or medical tests separate from standard-of-care.
  • Frequency of visits: There are no additional clinic visits apart of standard-of-care
  • Costs: The sponsor will cover the cost of all study activities.


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The Dystrophy Myotonic Clinical Research Network (DMCRN) to Establish Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

The purpose of the study is to determine the best ways to assess how people are affected by DM1.
  • Study director (local PI): Ericka P. Greene, MD
  • Sponsor: Myotonic Dystrophy Foundation
  • Recruiting?: Yes
  • Official study title: Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
  • Conditions studied: Myotonic Dystrophy Type 1
  • Study Type: Observational
  • Phase: N/A
  • Duration of participation: The expected time subjects will be in the study is 13.5 hours.
  • IRB #: Pro00018199
  • IRB approval date: 11/15/2014

Eligibility - Inclusion criteria: 

  • Age 18 to 70 (inclusive)
  • Competent to provide informed consent
  • Clinical diagnosis of DM1 based on research criteria1 or positive genetic test

Comment: The clinical research criteria require myotonia, muscle weakness in a characteristic distribution, and history of similar findings in a first degree relative. Genetic testing confirmed the diagnosis of DM1 in > 99% of individuals who satisfied these criteria.
Inclusion criteria for muscle biopsy sub-study

  • Of the 20 patients undergoing biceps muscle biopsy, at least half will have at least moderate weakness of elbow flexion, defined as MRC score ≤4+

Exclusion criteria:

  • Symptomatic renal or liver disease, uncontrolled diabetes or thyroid disorder, or active malignancy other than skin cancer.
  • Current alcohol or substance abuse
  • Concurrent enrollment in clinical trial for DM1, or participation in trial within 6 months of entry.
  • Concurrent pregnancy, or planned pregnancy during the course of the study.
  • Concurrent medical condition that would, in the opinion of the investigator or clinical evaluator, compromise performance on study measures.

Note: non-ambulatory participants are not excluded, but are limited to <15% of enrollment.
Exclusion criteria for muscle biopsy sub-study

  • Known CTG repeat expansion size less than 100 repeats, unless there are clear cut signs of limb weakness and muscle wasting.
  • Use of anticoagulant (e.g., warfarin, dabigatran)
  • Platelet count <50,000 (if known)
  • History of a bleeding disorder
  • Advanced wasting of tibialis anterior (TA) muscle that precludes needle muscle biopsy
  • Previous muscle biopsy of either TA

What is involved?

  • Procedures: Blood collection, ECG, functional measures and questionnaires
  • Frequency of visits: There are 4 study visits over the course of 2 years (initial visit, 3 months, 12 months and 24 months).
Costs: The sponsor will cover the cost of all study activities (including blood draw and muscle biopsy).

 

 

 

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Myotonic Dystrophy Database

The purpose of this protocol is to make available clinical findings, including blood pressure, pulmonary function tests, cardiac function tests, clinical evaluations, cognitive evaluations, performance tests, medications, routine blood work evaluations, weight, and progression of disease to allow Houston Methodist Neurological Institute researchers access to a large pool of patient findings. Since Myotonic Dystrophy is a rare disease, such experiments must rely upon previously banked clinical findings rather than immediately available subjects. Information will be collected from patients with DM who have signed an Informed Consent for Research Review.

  • Study director (local PI): Ericka P. Greene, MD
  • Recruiting?: Yes
  • Official study title: Myotonic Dystrophy Compilation of Clinical Findings
  • Conditions studied: Myotonic Dystrophy (DM)
  • Study Type: Observational
  • Phase: N/A
  • Duration of participation: Study duration is indefinite as this will encompass banking of clinical data.
  • IRB #: Pro00018337
  • IRB approval date: 11/29/2017

Eligibility - Inclusion criteria: 

  • Individuals with Myotonic Dystrophy, at least 18 years of age, capable of giving informed consent (or legally authorized representative when applicable)

Exclusion criteria:

  • Subjects under age 18
  • Subjects or legally authorized representative unable or unwilling to provide informed consent for collection of data

What is involved?

  • Procedures: There are no additional or different medical treatments, medical procedures, or medical tests separate from standard-of-care.
  • Frequency of visits: There are no additional clinic visits apart of standard-of-care
  • Costs: The study will cover the cost of all study activities.

 

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Observational Study of SPINRAZA in Adult Patients with Spinal Muscular Atrophy

The purpose of the study is to measure the safety and effectiveness of Spinraza in adults with SMA.

 

  • Study director (local PI): Jason R. Thonhoff, MD, PhD
  • Sponsor: Biogen MA, Inc.
  • Recruiting?: Yes
  • Official study title: A prospective, multi-center, observational study of the safety, tolerability and effectiveness of SPINRAZA in adult patients with Spinal Muscular Atrophy
  • Conditions studied: Spinal Muscular Atrophy (SMA)
  • ClinicalTrials.gov identifier: NCT03709784
  • Study Type: Observational
  • Phase: Phase 4
  • Duration of participation: The duration of the study is 48 months including an estimated 12-month period for screening and enrollment of participants, a 30-month assessment period and an additional 6 months for study closure activities.
  • IRB #: Pro00021067
  • IRB approval date: 04/08/2019

Eligibility - Inclusion criteria: 

  • Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.
  • Males and females with SMA type II or type III, aged 18 to 70 years at the time of enrollment.
  • Genetic documentation of 5Q SMA homozygous gene deletion, mutation, or compound heterozygote.
  • Are treatment naïve for SPINRAZA (nusinersen).
  • Have been prescribed SPINRAZA (nusinersen) by the treating physician as part of their clinical care for SMA following the FDA approved prescribing information guidelines as follows: dose level (12 mg), dosing schedule (3 loading doses administered at 14-day intervals, and the fourth loading dose administered 30 days after the third dose and subsequent maintenance doses administered every 4 months) and safety lab monitoring (CBC, PT, INR, PTT, UA) done prior to each dose administration.
  • Believed to be able to complete all study procedures, measurements and visits.
  • Estimated life expectancy at least 30 months from first dosing, in the opinion of the Investigator.
  • Revised upper limb module (RULM) score ≥ 4 (more than marginal upper extremity function/strength.
  • Must meet either Group 1 or Group 2 criteria, outlined below.

Inclusion criteria for Group 1 subjects:

  • May be ambulatory or non-ambulatory (defined as being wheelchair reliant at least 75% of time and unable to walk at least 10 meters without assistance).
  • RULM score of 4-34, inclusive.

Inclusion criteria for Group 2 subjects:

  • Ability to walk at least 10 meters without assistance (i.e., four point walking aid).
  • Be free of major orthopedic deformities that limit ambulation.
  • An ambulatory subject can qualify for both Group 1 and Group 2 if the RULM score is ≤ 34.

Exclusion criteria:

  • Revised upper limb score ≤ 3.
  • Respiratory insufficiency, defined by the medical necessity for invasive or noninvasive ventilation for >16 hours during a 24-hour period, at screening.
  • Hospitalization for major medical event including: surgery (i.e., scoliosis surgery, other surgery), cardiac event, pulmonary event, or other major medical problem within 2 months of screening or planned major surgical procedure likely to impact the clinical assessments during the duration of the study. Outpatient surgical procedure (i.e., placement of feeding tube) is not considered an exclusionary major medical event.
  • Presence of a symptomatic severe active infection or illness during the screening period that is likely to impact the performance on the clinical assessments.
  • Prior exposure to SPINRAZA (nusinersen).
  • Prior disorder, injury (e.g., upper or lower limb fracture) or surgical procedure which impacts the subject’s ability to perform any of the outcome measure testing required in the protocol and from which the subject has not fully recovered or achieved a stable baseline.
  • Treatment with an investigational drug, biological agent, or device within 1-month of Screening or 5 half-lives of study agent, whichever is longer. Patients on ≥3 months of clinically prescribed medications for SMA at non-escalating dosing of valproic acid, albuterol/salbuterol, carnitine, and/or creatine are eligible for the trial. Patients using intrathecal drug delivery devices, including investigational devices with an active IDE designation in the United States, may be eligible but require Study PI approval prior to enrollment.
  • Any history of exposure to gene therapy, antisense oligonucleotide therapy, or cell transplantation that was intended for the treatment of SMA.
  • Ongoing medical condition that according to the Clinical Center Investigator would interfere with the conduct and assessments of the study. Examples are medical disability (e.g., wasting or cachexia, severe anemia, etc.) that would interfere with the assessment of safety or would compromise the ability of the subject to undergo study procedures.

What is involved?

  • Procedures: Physical and neurological exam, pulmonary function test, CSF and blood collection, vital signs, assessment of upper limb motor function and questionnaires
  • Frequency of visits:
    • Up to 31 day screening period
    • 11 in-person visits for treatment period
  • Costs: The sponsor will cover the cost of the research parts of the study. The cost of the Spinraza infusion and all of the safety labs will be the patient’s/patient’s insurance company’s responsibility.

 

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Comparison of Quality of Life and Patient Satisfaction Between Two Headache Populations

The purpose of this study is to evaluate patient-reported outcomes (PROs) and quality of life (QoL) in two general neurology clinical populations – private pay patients at Houston Methodist Neurological institute (HMNI) and charity care patients at San Jose Clinic (SJC) in Houston, TX.

  • Study director (local PI): Ericka P. Greene, MD
  • Recruiting?: No
  • Official study title: A Comparison of the Quality of Life and Patient Satisfaction Between Two Headache Populations
  • Conditions studied: Headaches
  • Study Type: Observational
  • Duration of participation: Participation in the study is expected to last six months.
  • IRB #: Pro00018629
  • IRB approval date: 03/04/2019

Eligibility - Inclusion criteria: 

  • Individuals at least 18 years of age, but no older than 80
  • New patients referred to San Jose Clinic and Houston Methodist Neurological institute for evaluation and treatment of headache
  • Individuals willing and able to provide informed consent

Exclusion criteria:

  • Individuals under the age of 18 and over the age of 80
  • Established patients at San Jose Clinic and the Houston Methodist Neurological Institute
  • Diagnosis or treatment for the following: Cancer and Pregnancy
  • Individuals unwilling or unable to provide informed consent

What is involved?

  • Procedures: Administration of surveys
  • Frequency of visits: 2 in-person visits
  • Costs: The study will cover the cost of all study activities.

 

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