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Neuromuscular Disease Trials

As part of an academic medical center, the Division of Neuromuscular Disease at Northwestern University Feinberg School of Medicine aims to improve human health through scientific research. Clinical trials test or study drugs, surgical procedures, medical devices or interventions with human subjects. They look to determine their safety and effectiveness in relation to treating specific diseases. Clinical trials are part of clinical research and are at the heart of all medical advances.

The following searchable list includes all the neuromuscular disease clinical trials currently looking for participants.

Trials

Peripheral Neuropathy Research Registry (PNRR)

National Peripheral Neuropathy Research Registry (PNRR), a collection of different types ofinformation, such as patient medical, family, and social histories and blood samples. Theinformation is carefully maintained so that it can be studied repeatedly in the future. The registryaims to help researchers’ access large amounts of information about people with …
National Peripheral Neuropathy Research Registry (PNRR), a collection of different types ofinformation, such as patient medical, family, and social histories and blood samples. Theinformation is carefully maintained so that it can be studied repeatedly in the future. The registryaims to help researchers’ access large amounts of information about people with PN. By using thisregistry, researchers will facilitate both basic and clinical research studies that will bring improvedunderstandings of the etiology (origination) and pathogenesis (development) of PN. They willspecifically ask why some patients with peripheral neuropathy develop neuropathic pain and othersdo not, and what the characteristics of patients with painful peripheral neuropathy are in terms oftheir symptoms, examination findings, and blood tests. Ultimately this research may result inimproved diagnosis, more effective treatments, and possibly prevention.
Eligibility CriteriaInclusion criteria: 1. Diabetic Peripheral Neuropathy 2. Chemo-therapy Induced Peripheral Neuropathy 3. HIV-induced Peripheral Neuropathy 4. Idiopathic Peripheral Neuropathy; Exclusion criteria: Any other type of Peripheral Neuropathy
Principal InvestigatorAjroud-Driss, SendaAjroud-Driss, Senda
Location(s)
  • Map it 675 N. St. Clair St. Suite 20 100
    Chicago, IL
IRB number STU00048864
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Natural history study of ALS and other motor neuron disorders

This is one of the largest non-interventional observational study of patients with ALS and other motor neuron disorders. It is both prospective and retrospective. It does not require blood sampling.

Eligibility Criteria

1. A clinical diagnosis of El Escorial of suspected, possible, probable, or definite ALS.

2. Other motor neuron disorders, including but not limited to spinobulbar muscular atrophy (SBMA, Kennedy’s disease), Spinal Muscular Atrophy (SMA), Primary Lateral Sclerosis (PLS), Progressive Muscular Atrophy (PMA), and Progressive Bulbar Palsy (PBP).

3. Excluded are any disease that does not meet criteria for any motor neuron disorder

Principal InvestigatorAjroud-Driss, SendaAjroud-Driss, Senda
Location(s)
  • Map it 259 E. Erie St. Lavin Pavillion, Suite 19 100
    Chicago, IL
IRB number STU00209860
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Oxidative Markers and Efficacy in ALS/MND Phenotypes Treated with Edaravone (Loma Linda)

We are only recruiting patients who have not started their edaravone treatment.Location of study: Les Turner ALS Center at Northwestern Medicine, 259 E. Erie St., Lavin 19, Chicago, IL 60611.…

We are only recruiting patients who have not started their edaravone treatment.

Location of study: Les Turner ALS Center at Northwestern Medicine, 259 E. Erie St., Lavin 19, Chicago, IL 60611.

Eligibility Criteria

Inclusion:

Either possible, probable, or definite ALS,predominantly lower motor neuron disease Predominantly upper motor neuron disease, orbulbar

With or without cognitive involvement

Willing to participate

On no experimental treatment

Ages 18 - 85

No prior exposure to Radicava

On a stable dose of riluzole for 30 days or offriluzole

Male or female

Females of childbearing age must usecontraception

Exclusion:

Unstable medical illness

Abnormal liver function (>2x ULN)

Unlikely to survive for at least 26 weeks

Principal InvestigatorAjroud-Driss, SendaAjroud-Driss, Senda
Location(s)
  • Map it 259 E. Erie St. Lavin Pavillion, Suite 19 100
    Chicago, IL
ClinicalTrials.gov IdentifierNCT04097158IRB number STU00211350
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Platform Trial for the Treatment of Amyotrophic Lateral Sclerosis (ALS): A perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.

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 …

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). Each regimen will have a separate ClinicalTrials.gov posting, which will include specific information about the regimen. All regimen-specific outcome measures will be detailed in each regimen posting.

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 A - Zilucoplan Regimen B - Verdiperstat Regimen C - CNM-Au8 Regimen D - Pridopidine Regimen E - 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.

Eligibility Criteria

The basic eligibility criteria include:

1. Onset of ALS WEAKNESS within the last 3 years.

2. FVC (breathing test) > 50%

3. If on riluzole, must be on a stable dose for 30 days. Must not start riluzole during the study.

4. If on radicava, must be on a stable dose for 30 days. Must not start riluzole during the study.

5. Must be able to swallow for the next 6 months

6. No history stem cell treatment

7. No history of cancer within the last 5 years

Principal InvestigatorAjroud-Driss, SendaAjroud-Driss, Senda
Location(s)
  • Map it 259 E. Erie St. Lavin Pavillion, Suite 19 100
    Chicago, IL
ClinicalTrials.gov IdentifierNCT04297683IRB number STU00212680
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Keywords ALS Platform
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PLS Natural History Study (PNHS)

This is a non-interventional (no study drug), natural history study of patient with primary lateral sclerosis (PLS). The purpose of this study is to to develop a natural history dataset and biorepository of early PLS and well-established PLS cases for future clinical trials. The study will also evaluate …

This is a non-interventional (no study drug), natural history study of patient with primary lateral sclerosis (PLS).

The purpose of this study is to to develop a natural history dataset and biorepository of early PLS and well-established PLS cases for future clinical trials. The study will also evaluate differences in disease progression in early PLS and well-established PLS cases.

Patients will be enrolled over 24 months and complete assessments in person and over the phone.

Eligibility Criteria

Some of the BASIC, but not full, list of eligibility criteria are below:

1. PLS diagnosis is based on the new PLS diagnostic criteria

2. Symptom onset was no more than 15 years prior to baseline

3. Ability to independently walk with or without an assistive device (e.g., walker) at the baseline evaluation

4. Some bulbar symptoms (dysarthria, dysphagia, drooling or pseudobulbar affect)

5. UMN symptoms and signs in a region other than the legs

Principal InvestigatorAjroud-Driss, SendaAjroud-Driss, Senda
Location(s)
  • Map it 259 E. Erie St. Lavin Pavillion, Suite 19 100
    Chicago, IL
IRB number STU00214272
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A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

Tofersen (also called BIIB067) is currently being evaluated for the treatment of adults with familial ALS associated with a mutation in the SOD1 gene. The optimal timing for initiation (i.e., prior to or after the emergence of clinically manifested disease) of tofersen is unknown. This study will evaluate the …

Tofersen (also called BIIB067) is currently being evaluated for the treatment of adults with familial ALS associated with a mutation in the SOD1 gene. The optimal timing for initiation (i.e., prior to or after the emergence of clinically manifested disease) of tofersen is unknown. This study will evaluate the impact of initiating tofersen based on biomarker evidence of disease activity, prior to the emergence of clinical symptoms or signs that definitively indicate ALS.

Eligibility CriteriaThere are four parts to this study, each with different eligibility criteria. Please contact the study coordinator and he/she will determine whether you are eligible. In order to enroll in the study (Part A), here are a few basic (but not complete) eligibility criteria:

1. 18 years or older

2. Must have one of the following SOD1 mutations confirmed by a central reader. Please contact the study coordinator for a complete list.

3. If a SOD1 mutation is not listed, your mutation will be adjudicated by a Mutation Adjudication Committee.

4. Plasma neurofilament (NfL) level less than 44 pg/mL during Screening

5. Clinically pre-symptomatic for ALS (i.e., must not have clinically manifested ALS) at Part A Screening Visit

6. History or positive test result at Screening for HIV, Hep-B, or Hep-C

Principal InvestigatorAjroud-Driss, SendaAjroud-Driss, Senda
Location(s)
  • Map it 259 E. Erie St. Lavin Pavillion, Suite 19 100
    Chicago, IL
ClinicalTrials.gov IdentifierNCT04856982IRB number STU00214617
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ArgenX 2011 Open Label Extension

This study will look at the long-term safety and effect (how well it works) of efgartigimod in people with active idiopathic inflammatory myopathy, also known as myositis. The study will include adult participants with different forms of myositis: dermatomyositis, immune-mediated necrotizing myopathy, and polymyositis (including antisynthetase syndrome). Efgartigimod …

This study will look at the long-term safety and effect (how well it works) of efgartigimod in people with active idiopathic inflammatory myopathy, also known as myositis. The study will include adult participants with different forms of myositis: dermatomyositis, immune-mediated necrotizing myopathy, and polymyositis (including antisynthetase syndrome). Efgartigimod is being tested in this study, it is the same study drug tested in ARGX-113-2007. It is the same study drug tested in the ARGX-113-2007 study. It is a piece of a human antibody modified to bind better to a specific protein called FcRn. Antibodies are proteins that fight and prevent infections. FcRn helps maintain antibody levels. Antibody levels are reduced after efgartigimod binds to FcRn. This means that the levels of antibodies attacking your own body are also reduced. IRB #: STU00220056-MOD0004 Approved by NU IRB for use on or after 3/31/2025 through 2/16/2026. Permission to Take Part in a Human Research Study ARGX-113-2011 Participant Informed Consent Document US version 4.0 12Feb2025 NU site version date: 26Feb2025 Page 2 of 13 HRP-592 / v.11-10-2023 Efgartigimod is similar to antibodies naturally present in the human body. It is called a “biological” drug because it is a piece of protein produced in cells of mammals. Efgartigimod may help improve myositis. Efgartigimod has been approved by the Food and Drug Administration (FDA) as a treatment for adult patients with myasthenia gravis, a rare disease causing muscle weakness. It is considered experimental because health authorities have not approved it for myositis. Everyone will get efgartigimod during the treatment period

Principal InvestigatorSeth, ArjunSeth, Arjun
ClinicalTrials.gov IdentifierNCT05979441IRB number STU00220056
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Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod PH20 SC Administered by Prefilled Syringe in Adult Participants With Ocular Myasthenia Gravis

The aim of this study is to measure the efficacy (how well it works) and safety ofefgartigimod compared with placebo (inactive substance) in participants with ocularmyasthenia gravis (oMG). The study will also measure the long-term efficacy, safety, andtolerability of efgartigimod.…

The aim of this study is to measure the efficacy (how well it works) and safety of

efgartigimod compared with placebo (inactive substance) in participants with ocular

myasthenia gravis (oMG). The study will also measure the long-term efficacy, safety, and

tolerability of efgartigimod.

Principal InvestigatorSeth, ArjunSeth, Arjun
ClinicalTrials.gov IdentifierNCT06558279IRB number STU00222478
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PREVENT ALL ALS

The primary purpose of this study (also referred to as “PREVENT”) is to collect a wide range of biofluid samples (cerebral spinal fluid and blood), clinical information, and other health and well-being information from individuals who are genetically at risk for ALS or a related disorder, Frontotemporal Dementia (FTD). …

The primary purpose of this study (also referred to as “PREVENT”) is to collect a wide range of biofluid samples (cerebral spinal fluid and blood), clinical information, and other health and well-being information from individuals who are genetically at risk for ALS or a related disorder, Frontotemporal Dementia (FTD). This will help us better understand ALS and how the disease progresses by looking for measurable differences that will help us identify how and when the body changes in response to ALS causative gene variants.

Samples of blood and fluid surrounding the spinal cord and brain, known as cerebral spinal fluid (CSF), collected during this study will be stored in a secure, central location, called a biorepository. Other data that are collected will be stored in a secure central data repository. The data and samples will be maintained for many years and will be made available to researchers for use in future studies. We hope the information we collect will guide the direction of future research and lead to more informative, targeted drug development for halting, repairing, and/or preventing ALS.

Principal InvestigatorAjroud-Driss, SendaAjroud-Driss, Senda
IRB number STU00223130
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A Phase 2a Biomarker-driven trial of oral digoxin in Individuals with Amyotrophic Lateral Sclerosis (ALS)- The Acacia Trial, an ALS MyMatch Trial

We are doing the research to learn about the safety and tolerability of digoxin in individuals with ALS and also to better understand if digoxin has an effect on slowing neurodegeneration in ALS. Digoxin is approved by the U.S. Food and Drug Administration (FDA) to treat heart failure and …

We are doing the research to learn about the safety and tolerability of digoxin in individuals with ALS and also to better understand if digoxin has an effect on slowing neurodegeneration in ALS. Digoxin is approved by the U.S. Food and Drug Administration (FDA) to treat heart failure and atrial fibrillation in adults. Digoxin is not approved by the FDA to treat ALS.

Principal InvestigatorAjroud-Driss, SendaAjroud-Driss, Senda
ClinicalTrials.gov IdentifierNCT07047209IRB number STU00224368
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