Neuromuscular Disease Trials

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.

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. 

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 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. 

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 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 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.

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. 

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.

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.

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.