Neuromuscular Clinical Trials

JOIN A CLINICAL TRIAL

• HEALEY ALS Platform Trial
  Condition studies: amyotrophic lateral sclerosis (ALS)
  Study director: Stanley H. Appel, MD 

JOIN A RESEARCH STUDY

• Leukapheresis in ALS
  Condition studies: amyotrophic lateral sclerosis (ALS)
  Study director: Jason R. Thonhoff, MD, PhD
• Biological Markers Study for ALS
  Condition studies: amyotrophic lateral sclerosis (ALS)
  Study director: Stanley H. Appel, MD
• Compilations of Clinical Findings in ALS
  Condition studied: amyotrophic lateral sclerosis (ALS)
  Study director: Stanley H. Appel, MD
• Neuromuscular Observational Research Data Hub (MOVR)
  Conditions studied: amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and Becker muscular dystrophy (BM)
  Study director: Ericka P. Greene, MD
• The Dystrophy Myotonic Clinical Research Network (DMCRN) to Establish Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
  Conditions studied: Myotonic dystrophy type 1
  Study director: Ericka P. Greene, MD
• Myotonic Dystrophy Database
  Conditions studied: Myotonic dystrophy
  Study director: Ericka P. Greene, MD
• Registry for Patients with Myasthenia Gravis who are on Soliris
  Conditions studied: Generalized myasthenia gravis (MG)
  Study director: Ericka P. Greene, MD
• COVID-19+ Patients for Neurological Manifestations
  Conditions studied: COVID-19
  Study director: Sheetal Shroff, MD

Completed Studies

 

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

• Rachel Applegate, CCRC
  rgdavis@houstonmethodist.org
  713.441.9120
• Aramide Balogun
  abalogun@houstonmethodist.org
  713.441.6937

 

 

 

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HEALEY ALS Platform Trial

The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial. In this trial, multiple investigational products for ALS will be tested simultaneously or sequentially. Each investigational product will be tested in a regimen. Each regimen consists of a placebo-controlled trial, meaning that the active investigational product and matching placebo will be tested in each regimen. The additional details that govern the testing of each investigational product will be summarized in separate regimen-specific appendices (RSAs). Participants will have an equal chance to be randomized to all regimens that are active at the time of screening. Once randomized to a regimen, participants will be randomized in a 3:1 ratio to either study drug or placebo. The following regimens are active in the trial: Regimen E - SLS-005 Trehalose. New regimens will be continuously added as new investigational products become available. The HEALEY ALS Platform Trial will enroll additional participants as each new regimen is available.

• Study director (local PI): Stanley H. Appel, MD
• Sponsor: Merit E. Cudkowicz, MD
• Recruiting?: Yes
• Official study title: HEALEY ALS Platform Trial
• ClinicalTrials.gov identifier: NCT04297683
• Conditions studied: Amyotrophic Lateral Sclerosis (ALS)
• Intervention Drugs:Regimen B – Verdiperstat: Participants are randomized to receive either active verdiperstat or matching placebo at 600mg twice daily. Regimen C – CNM-Au8: Participants are randomized to receive either active CNM-Au8 or matching placebo at either 30mg or 60mg daily. Regimen D – Pridopidine: Participants are randomized to receive either active Pridopidine or matching placebo at 45mg twice daily. Regimen E – SLS-005 Trehalose: Participants are randomized to receive either active SLS-005 Trehalose or matching placebo at 0.75 g/kg weekly.
• Phase: Phase 2/3
• Duration of participation: Treatment duration of placebo-controlled regimens is a maximum of 24-weeks for each regimen. An optional open label extension (OLE) may be offered.
• IRB #: Pro00025436
• IRB approval date: 06/25/2020

Eligibility - Inclusion criteria: Patients must meet all of the following criteria to be eligible for study participation:

• Sporadic or familial ALS diagnosed as clinically possible, probable, lab-supported probable, or definite ALS defined by revised El Escorial criteria.
• Age 18 years or older.
• Capable of providing informed consent and complying with study procedures, in the SI's opinion.
• Time since onset of weakness due to ALS ≤ 36 months at the time of the Master Protocol Screening Visit.
• Vital Capacity ≥ 50% of predicted capacity for age, height, and sex at the time of the Master Protocol Screening Visit measured by Slow Vital Capacity (SVC), or, if required due to pandemic-related restrictions, Forced Vital Capacity (FVC).
• Participants must either not take riluzole or be on a stable dose of riluzole for ≥ 30 days prior to the Master Protocol Screening Visit. Riluzole-naïve participants are permitted in the study.
• Participants must either not take edaravone or have completed at least one cycle of edaravone prior to the Master Protocol Screening Visit. Edaravone-naïve participants are permitted in the study.
• Participants must have the ability to swallow pills and liquids at the time of the Master Protocol Screening Visit and, in the SI's opinion, have the ability to swallow for the duration of the study.
• Geographically accessible to the site.

Exclusion criteria: Patients meeting any of the following criteria are not eligible for study participation:

• Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant, according to SI's judgment (e.g., cardiovascular instability, systemic infection, untreated thyroid dysfunction, or clinically significant laboratory abnormality or EKG changes).
• Lab abnormalities include, but are not limited to: Hemoglobin < 10 g/dL, White Blood Cells < 3.0 x 103/mm3, Neutrophils, Absolute ≤ 1000/mm3, Eosinophilia (absolute eosinophil count of ≥ 500 eosinophils per microliter), low platelet counts (< 150 x 109 per liter), alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 3 times the upper limit of normal (ULN), eGFR < 30 mL/min/1.73m2, thyroid-stimulating hormone (TSH) levels >10 mIU/L or <0.01 mIU/L.
• Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, in the SI's opinion.
• Active cancer or history of cancer, except for the following: basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years.
• Use of investigational treatments for ALS (off-label use or active participation in a clinical trial) within 5 half-lives (if known) or 30 days (whichever is longer) prior to the Master Protocol Screening Visit.
• Exposure at any time to any gene therapies under investigation for the treatment of ALS (off-label use or investigational).
• If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or of child-bearing potential and unwilling to use effective contraception for the duration of the trial and for 3 months, or longer as specified in each RSA, after discontinuing study treatment.
• If male of reproductive capacity, unwilling to use effective contraception for the duration of the trial and for 3 months, or longer as specified in each RSA, after discontinuing study treatment.
• Anything that would place the participant at increased risk or preclude the participant's full compliance with or completion of the study, in the SI's opinion.
• If a participant is being re-screened, the disqualifying condition has not been resolved, or the mandatory wash-out duration has not occurred.
• For those participating in the optional CSF collection, contraindication to undergoing a lumbar puncture (LP) in the SI's opinion. Participants undergoing the LP must not be currently taking anticoagulation medications such as warfarin that would be a contraindication to LP; aspirin and non-steroidal anti-inflammatories are allowed.

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

What is involved?

• Testing: Neurological and physical examinations, muscle strength testing, questionnaires, FVC Measurements, ECGs, blood, and urine specimen collection, vital signs
• Frequency of visits:
• Up to 42 day screening period
• 10 visits for randomized controlled portion of the trial (clinc-, phone-, or telemedicine-based)
• At least 11 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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Leukapheresis in ALS

A Study to Perform Apheresis (Leukapheresis) on Patients with Amyotrophic Lateral Sclerosis to Collect Regulatory T-Lymphocytes (Tregs) 

• Study director (local PI): Jason R. Thonhoff, MD, PhD
• Recruiting?: Yes
• Official study title: A Study to Perform Apheresis (Leukapheresis) on Patients with Amyotrophic Lateral Sclerosis to Collect Regulatory T-Lymphocytes (Tregs)
• ClinicalTrials.gov identifier: N/A
• Conditions studied: Amyotrophic Lateral Sclerosis (ALS)
• Study Type: Observational
• Phase: N/A
• Duration of participation: Subjects will be part of the study for approximately one month or less depending on when we can schedule the leukapheresis. Study participation will involve the screening visit and the leukapheresis procedure.
• IRB #: Pro00033074
• IRB approval date: 11/18/2021

Eligibility
Inclusion criteria: 

• Participants with a sporadic or familial ALS diagnosis must meet the El Escorial criteria for possible, probable, lab-supported probable, or definite ALS.
• At least 18 years old.
• Capable of providing 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 in the Investigator’s opinion.
• Geographically accessible to the site.
• Participant must agree not to take live attenuated vaccines (including seasonal flu vaccine) 30 days before leukapheresis.
• Participant is able and willing to undergo leukapheresis.

Exclusion criteria: 

• 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 disease, cognitive impairment, or dementia that would impair ability of the subject to provide informed consent, according to the PI’s judgement, or a history of active substance abuse within the prior year.
• Pregnant or breastfeeding or planning to become pregnant
• Known immune deficiency or history of lymphoma or leukemia.
• History of lymphopenia.
• History of acquired or inherited immune deficiency syndrome, including leukopenia.
• History of severe untreated chronic obstructive sleep apnea.
• 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) 13, hematocrit (HCT) 54, or platelets 500 at screening.
• Serum creatinine >1.8 mg/dL or creatinine clearance

What is involved?

• Procedures: Blood, urine, physical and neurological exam, leukapheresis, and vitals
• Frequency of visits: Subjects will be part of the study for approximately one month or less depending on when we can schedule the leukapheresis. Study participation will involve the screening visit and the leukapheresis procedure.
• 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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Biological Markers Study for ALS

This is a biospecimen-banking protocol including blood, urine, CSF collected during diagnostic evaluations, tissue from biopsies, and autopsies. 

• 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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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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Registry for Patients with Myasthenia Gravis who are on Soliris

A need exists for the collection of real-world data on effectiveness, safety, and patterns of drug utilization for C5 Inhibition Therapies in patients with myasthenia gravis to complement the body of scientific evidence and better inform current and future clinical practice.

• Study director (local PI): Ericka P. Greene, MD
• Sponsor: Alexion Pharmaceuticals, Inc.
• Recruiting?: Yes
• Official study title: Long-Term, Observational, Registry of Patients With Generalized Myasthenia Gravis Who Have Received Treatment With Complement C5 Inhibition Therapies
• Conditions studied: Myasthenia Gravis (MG)
• Study Type: Observational
• Phase: N/A
• Duration of participation: The duration of data collection for the Registry will be up to 5 years from when the first patient is enrolled.
• IRB #: Pro00025919
• IRB approval date: 2/3/2021

Eligibility - Inclusion criteria: Patients are eligible to be included in the Registry only if all of the following criteria apply:

• Patients ≥ 18 years of age at the time of enrollment.
• Patients with gMG who have ever received treatment with ALXN-C5IT according to the label at the time of enrollment in the Registry.
• Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
• Patients must have all of the following historical data available (within 6 months prior to the initiation of ALXN-C5IT) to be enrolled in the Registry
• MGFA class (worst class to date)
• MG-ADL Score

Exclusion criteria: Patients currently enrolled in an Alexion-sponsored interventional clinical study for treatment of gMG cannot be enrolled in the Alexion gMG Registry while enrolled/participating in the clinical study for gMG therapy.
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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COVID-19+ Patients for Neurological Manifestations

Observational Study of COVID-19 Positive Patients for Neurological Manifestations and Their Outcomes

• Study director (local PI): Sheetal Shroff, MD
• Recruiting?: Yes
• Official study title: Observational Study of COVID-19 Positive Patients for Neurological Manifestations and Their Outcomes
• Conditions studied: COVID-19 positive patients with neurological manifestations
• Study Type: Observational
• Phase: N/A
• Duration of participation: This is an observational study consisting of: retrospective chart review from October 1st, 2019 to August 15, 2020 and prospective chart review from July 6th 2020 through estimated date June 30th 2021.
• IRB #: Pro00026462
• IRB approval date: 5/7/2021

Eligibility - Inclusion criteria: 

• COVID-19 PCR positive and clinically presumed positive patients.
• Age inclusion between18-99 years-old, when treated beginning in October 2019.
• Hospital admission between October 2019 for neurological symptoms, work up and care; includes the results of the blood work (inflammatory markers, cell count, chemistry), imaging CT/MRI/Doppler, EEG/VEP/BSAEP/SSEP, EMG/NCS, spinal fluid analysis, nerve and/or muscle biopsy and neuromuscular ultrasound.
• All treatment and/or work up for patient’s wellbeing will be determined by the treating/ admitting physician in the hospital.

Exclusion criteria:

• COVID-19 PCR negative patients

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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