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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.
Inclusion 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
Ajroud-Driss, SendaAjroud-Driss, Senda
  • Map it 675 N. St. Clair St. Suite 20-100
    Chicago, IL
STU00048864
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cIRB: Topiramate as a disease altering therapy for Cryptogenic Sensory Peripheral Neuropathy (CSPN)

This is a 96-week placebo-controlled trial of topiramate at a target dose of 100 mg daily (50 mg twice daily) as a potentially disease altering therapy for Cryptogenic Sensory Peripheral Neuropathy
INCLUSION: 1. Age 18-75; 2. Diagnosis of confirmed cryptogenic symptomatic distal symmetric peripheral polyneuropathy; 3. Prediabetes based on American Diabetes Association; 4. No history of prior therapy with topiramate; 5. Waist circumference >102 cm for men, >88 cm for women
Menichella, Daniela MMenichella, Daniela M
  • Map it Lavin Pavillion 259 E. Erie St., Suite 19-100
    Chicago, IL
NCT02878798 STU00206049
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Radicava® (Edaravone) Biomarker Study in Participants with Amyotrophic Lateral Sclerosis

This is a non-interventional (no study drug), observational study of patients with ALS who elect to begin treatment with Edaravone (radicava).This study is designed to investigate a selected panel of biomarkers in patients with ALS, treated with Edaravone. Biomarkers of oxidative stress, inflammation and neurodegeneration will be explored. …
This is a non-interventional (no study drug), observational study of patients with ALS who elect to begin treatment with Edaravone (radicava).This study is designed to investigate a selected panel of biomarkers in patients with ALS, treated with Edaravone. Biomarkers of oxidative stress, inflammation and neurodegeneration will be explored. Epigenetic and protein biomarkers will also be investigated.Edaravone will NOT be provided by the study sponsor. 
1. Sporadic or familial ALS diagnosed as possible, probable, probable-laboratory supported or definite as defined by the World Federation of Neurology revised El Escorial criteria2. Decision made to prescribe Edaravone prior to screening3. Participant will likely be able to obtain commercial Edaravone and likely to complete 6 cycles of treatment, per site investigator estimation4. Participant either naïve to Edaravone or who did not receive any Edaravone dose within 28 days prior to screening5. Participant with a contraindication to Edaravone may not participate6. Participant is participating in an interventional clinical trial may not participate
Ajroud-Driss, SendaAjroud-Driss, Senda
  • Map it Lavin Pavillion 259 E. Erie St., Suite 19-100
    Chicago, IL
NCT04259255 STU00210057
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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. 
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
Ajroud-Driss, SendaAjroud-Driss, Senda
  • Map it Lavin Pavillion 259 E. Erie St., Suite 19-100
    Chicago, IL
STU00209860
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Oxidative Markers and Efficacy in ALS/MND Phenotypes Treated with Edaravone

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.
Inclusion:Either possible, probable, or definite ALS,predominantly lower motor neuron disease Predominantly upper motor neuron disease, orbulbarWith or without cognitive involvementWilling to participateOn no experimental treatmentAges 18 - 85No prior exposure to RadicavaOn a stable dose of riluzole for 30 days or offriluzoleMale or femaleFemales of childbearing age must usecontraceptionExclusion:Unstable medical illnessAbnormal liver function (>2x ULN)Unlikely to survive for at least 26 weeks
Ajroud-Driss, SendaAjroud-Driss, Senda
  • Map it Lavin Pavillion 259 E. Erie St., Suite 19-100
    Chicago, IL
NCT04097158 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 - TrehaloseNew 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.
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 months6. No history stem cell treatment7. No history of cancer within the last 5 years
Ajroud-Driss, SendaAjroud-Driss, Senda
  • Map it Lavin Pavillion 259 E. Erie St., Suite 19-100
    Chicago, IL
NCT04297683 STU00212680
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ALS Platform
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Use of Glenohumeral Injections for Management of Shoulder Pain in Individuals with ALS

The purpose of this study is to develop a practice for measuring the efficacy of ultrasound-guided shoulder joint injections in patients with ALS who have exhibited shoulder pain and/or adhesive capsulitis. Patients will receive corticosteroid injections as part of their normal or standard care for shoulder pain in …
The purpose of this study is to develop a practice for measuring the efficacy of ultrasound-guided shoulder joint injections in patients with ALS who have exhibited shoulder pain and/or adhesive capsulitis. Patients will receive corticosteroid injections as part of their normal or standard care for shoulder pain in ALS (not provided by the study). They will then be asked to complete questionnaires at Baseline and over the phone over approximately 3 months. 
1. Diagnosed with ALS2. Being referred for evaluation for glenohumeral shoulder injections for management of shoulder pain3. Able to utilize written, typed or verbal communication, independently or with the assistance of a caregiver4. Able to understand study procedures, answer questionnaires, and provide informed consent
Franz, Colin KFranz, Colin K
  • Map it Lavin Pavillion 259 E. Erie St., Suite 19-100
    Chicago, IL
STU00213465
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A prospective, multicenter, randomised, double-blind, placebo-controlled, parallel groups, phase 3 study to compare the efficacy and safety of masitinib in combination with Riluzole versus placebo in combination with Riluzole in the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS)

This is a phase 3, placebo-controlled, study comparing the safety and efficacy of two doses of MASITINIB (4.5 mg and 6.0 mg) and riluzole vs. placebo and riluzole over 48 weeks. …
This is a phase 3, placebo-controlled, study comparing the safety and efficacy of two doses of MASITINIB (4.5 mg and 6.0 mg) and riluzole vs. placebo and riluzole over 48 weeks. 
Basic Inclusion/ Exclusion criteria:Patient treated with stable dose of riluzole for 3 monthsPatient able to obtain riluzole through insurance and remain on riluzole throughout the studyDisease duration (weakness) from diagnosis less than 24 monthsMust have a sub-score of at least 2 on every sub-section of the ALSFRS-RFVC over 60%
Ajroud-Driss, SendaAjroud-Driss, Senda
  • Map it Lavin Pavillion 259 E. Erie St., Suite 19-100
    Chicago, IL
NCT02588677 STU00213943
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A Phase 3b, Multicenter, Randomized, Double-Blind Study to Evaluate Efficacy and Safety of Oral Edaravone Administered for a Period of 48 Weeks in Subjects with Amyotrophic Lateral Sclerosis (ALS)

This is a phase 3b, randomized, study evaluating the safety and efficacy of ORAL EDARAVONE/ RADICAVA administered over 48 weeks. Eligible participants are randomized to two groups with different dosing schemes.…
This is a phase 3b, randomized, study evaluating the safety and efficacy of ORAL EDARAVONE/ RADICAVA administered over 48 weeks. Eligible participants are randomized to two groups with different dosing schemes.
Basic Inclusion/ Exclusion criteria:No prior use of radicava/edaravoneALS weakness onset within the last 2 yearsFVC over 70%Must score at least a 2 on each sub-section of the ALSFRS-REl Escorial scored Probable or Definite ALS
Ajroud-Driss, SendaAjroud-Driss, Senda
  • Map it Lavin Pavillion 259 E. Erie St., Suite 19-100
    Chicago, IL
NCT04165824 STU00213716
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A Phase 2a, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Assess the Safety of Engensis in Participants with Amyotrophic Lateral Sclerosis

This is a phase 2, placebo-controlled, study assessing the safety of ENGENSIS. Participants will be randomized to study drug (66%) or placebo (34%). This study drug or placebo is administered via intramuscular injections. 128 injections are administered in different parts of the body on one treatment day. There are …
This is a phase 2, placebo-controlled, study assessing the safety of ENGENSIS. Participants will be randomized to study drug (66%) or placebo (34%). This study drug or placebo is administered via intramuscular injections. 128 injections are administered in different parts of the body on one treatment day. There are 6 treatment days when the study drug or placebo is administered. The study lasts approximately 6 months.
Basic Inclusion/ Exclusion criteria:Onset of ALS weakness within the last 4 yearsSVC over 50%Stable dose of riluzole for 1 month, or not start during studyStable dose of radicava for 1 month, or not start during studyLimb onset of ALS requiredNo personal history of cancer within the last 3 years
Ajroud-Driss, SendaAjroud-Driss, Senda
  • Map it Lavin Pavillion 259 E. Erie St., Suite 19-100
    Chicago, IL
NCT04632225 STU00214139
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ALS Engensis
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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. 
Some of the BASIC, but not full, list of eligibility criteria are below:1.  PLS diagnosis is based on the new PLS diagnostic criteria2.  Symptom onset was no more than 15 years prior to baseline3.  Ability to independently walk with or without an assistive device (e.g., walker) at the baseline evaluation4. Some bulbar symptoms (dysarthria, dysphagia, drooling or pseudobulbar affect)5. UMN symptoms and signs in a region other than the legs
Ajroud-Driss, SendaAjroud-Driss, Senda
  • Map it Lavin Pavillion 259 E. Erie St., Suite 19-100
    Chicago, IL
STU00214272
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An Intermediate-Size Expanded Access Protocol for Amyotrophic Lateral Sclerosis with Verdiperstat (BHV-3241)

The primary objective of this intermediate-size Expanded Access Protocol (EAP) is to provide access to investigational product verdiperstat for participants with ALS who do not qualify for the HEALEY ALS Platform Trial.…
The primary objective of this intermediate-size Expanded Access Protocol (EAP) is to provide access to investigational product verdiperstat for participants with ALS who do not qualify for the HEALEY ALS Platform Trial.
The BASIC, but not full, list of eligibility criteria are below:1. Sporadic or familial ALS diagnosed as suspected, possible, probable, lab-supported probable, or definite ALS defined by El Escorial criteria2. Age 18 years or older3. Participant must be unable to participate in the HEALEY ALS Platform Trial4. Participants have established care with a physician at the specialized ALS center involved in the protocol and will maintain this clinical care throughout the duration of the EAP5. Participants must have the ability to swallow whole pills without crushing or chewing6. Participants who are enrolled in another EAP or a Clinical Trial
Ajroud-Driss, SendaAjroud-Driss, Senda
  • Map it Lavin Pavillion 259 E. Erie St., Suite 19-100
    Chicago, IL
NCT04081714 STU00215197
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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.
There 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 older2. 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 Screening5. Clinically pre-symptomatic for ALS (i.e., must not have clinically manifested ALS) at Part A Screening Visit6. History or positive test result at Screening for HIV, Hep-B, or Hep-C
Ajroud-Driss, SendaAjroud-Driss, Senda
  • Map it Lavin Pavillion 259 E. Erie St., Suite 19-100
    Chicago, IL
NCT04856982 STU00214617
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Evaluation of digital technologies for home-based assessment of ALS patients

This is a non-interventional study (no study drug). We are measuring home-based digital assessments. The purpose of this study is to evaluate the feasibility, patient-acceptability, adherence and validity of home-based collection of digital measures that are relevant to the assessment of disease in ALS patients. The …
This is a non-interventional study (no study drug). We are measuring home-based digital assessments. The purpose of this study is to evaluate the feasibility, patient-acceptability, adherence and validity of home-based collection of digital measures that are relevant to the assessment of disease in ALS patients. The goal of this study is to determine which home-based digital measurements are reliable predictors of change in ALS status, when compared to the conventional scale, the ALSFRSR.
1. Non-ventilator dependent at the screening visit.2. Able to ambulate short distances (at least 3 m) with or without walking aid.3. Able to grasp a pen and able to write4. For the dedicated C9orf72 group only (10-15 participants) presence of a C9orf72 repeat expansion.5. Having a study partner who agrees to participate in the study and who is intellectually, visually, and verbally capable, and fluent in, and able to read, the language in which study assessments are administered.
Ajroud-Driss, SendaAjroud-Driss, Senda
  • Map it Lavin Pavillion 259 E. Erie St., Suite 19-100
    Chicago, IL
NCT00000419 STU00215205
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ALS BASICHR0053
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