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.
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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 i… 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
STU00048864 |
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
NCT02878798 STU00206049 |
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 biom…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 criteria 2. Decision made to prescribe Edaravone prior to screening 3. Participant will likely be able to obtain commercial Edaravone and likely to complete 6 cycles of treatment, per site investigator estimation 4. Participant either naïve to Edaravone or who did not receive any Edaravone dose within 28 days prior to screening 5. Participant with a contraindication to Edaravone may not participate 6. Participant is participating in an interventional clinical trial may not participate
NCT04259255 STU00210057 |
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
STU00209860 |
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, 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
NCT04097158 STU00211350 |
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 simultane… 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. 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
NCT04297683 STU00212680 |
A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients with Polyneuropathy of Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with a V122I or T60A Mutation This is a Phase 4 observational study to evaluate the effectiveness of patisiran in patients with… This is a Phase 4 observational study to evaluate the effectiveness of patisiran in patients with polyneuropathy of Hereditary Transthyretin-Mediated (ATTRv) Amyloidosis with a V122I or T60A Mutation. We are looking for patients who are either a.) naïve to patisiran, b.) on commercial patisiran therapy for less than 12 months at enrollment, and c.) exposed to patisiran within the last 12 months regardless of current treatment options. Patisiran will NOT be provided by the study sponsor. 1. Diagnosed with ATTRv amyloidosis with polyneuropathy, with a documented V122I or T60A mutation. 2. PND score of I-IIIB at baseline 3. Naïve to patisiran or on commercial patisiran therapy for less than 12 months or exposed to commercial therapy during the last 12 months regardless of current treatment. 4. Cannot have participated in a clinical trial for any investigational agent in the 6 months prior to baseline.
STU00211987 |
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. Patient… 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 ALS 2. Being referred for evaluation for glenohumeral shoulder injections for management of shoulder pain 3. Able to utilize written, typed or verbal communication, independently or with the assistance of a caregiver 4. Able to understand study procedures, answer questionnaires, and provide informed consent
STU00213465 |
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. Basic Inclusion/ Exclusion criteria:
NCT02588677 STU00213943 |
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 adm… 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:
NCT04165824 STU00213716 |
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… 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:
NCT04632225 STU00214139 |
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… 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 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
STU00214272 |
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… 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 older 3. Participant must be unable to participate in the HEALEY ALS Platform Trial 4. 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 EAP 5. Participants must have the ability to swallow whole pills without crushing or chewing 6. Participants who are enrolled in another EAP or a Clinical Trial
NCT04081714 STU00215197 |
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 … 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 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
NCT04856982 STU00214617 |
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-b… 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 write 4. 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.
NCT00000419 STU00215205 |