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 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 Location(s)
IRB number STU00048864 Keywords Peripheral Neuropathy; Registry; Copy Study URL to Clipboard Copy |
Natural history study of ALS and other motor neuron disordersThis 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 Location(s)
IRB number STU00209860 Copy Study URL to Clipboard Copy |
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 Location(s)
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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 Location(s)
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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 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 Location(s)
IRB number STU00214272 Copy Study URL to Clipboard Copy |
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 MutationTofersen (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 Location(s)
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