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Spinal Muscular Atrophy Clinical Trials

A listing of Spinal Muscular Atrophy medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

RESULTS

Found (16) clinical trials

Single and Multiple Dose Study of BIIB067 in Adults With Amyotrophic Lateral Sclerosis (ALS)

The primary objective of the study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of BIIB067 in adults with ALS. The secondary objective is to evaluate the effects of BIIB067 on levels of superoxide dismutase 1 (SOD1) protein in the cerebrospinal fluid (CSF).

Phase

A Biomarker Study to Evaluate MN-166 (Ibudilast) in Subjects With Amyotrophic Literal Sclerosis (ALS)

This is a multi-center, open-label study of MN-166 (ibudilast) in subjects with ALS. To be eligible subjects must meet the El Escorial criteria of possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS. Safety, tolerability, blood, neuro-imaging biomarkers, and clinical outcomes will be collected on all subjects. ...

Phase

A Study to Investigate the Safety Tolerability Pharmacokinetics Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

Multi-center, randomized, double-blind, placebo-controlled, Phase II study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RO7034067 in adult and pediatric participants with Type 2 and Type 3 SMA. The study consists of two parts, an exploratory dose finding part (Part 1) of RO7034067 for 12 weeks and a ...

Phase

Investigate Safety Tolerability PK PD and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy

Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of RO7034067 in infants with Type 1 spinal muscular atrophy (SMA). The study consists of two parts, an exploratory dose finding part (Part 1) and a confirmatory part (Part 2) which will investigate RO7034067 ...

Phase

Study of Inherited Neurological Disorders

The Neurogenetics Branch (NGB) within the National Institute of Neurological Disorders and Stroke (NINDS) is conducting a study to evaluate and provide genetic diagnosis to participants with various diagnosed and undiagnosed neurological conditions. OBJECTIVES The primary objective of this protocol is to provide a resource of patients for enrollment into ...

Phase N/A

Accurate Test of Limb Isometric Strength (ATLIS) in ALS

Cedars-Sinai Medical Center will enroll and collect data on 100 subjects. Subjects will ideally be tested every 1 to 4 months according to each patient's site visit schedule. Subjects will be tested at each visit using ATLIS, the ALSFRS-R questionnaire and EIM.

Phase N/A

Genetic Characterization of Movement Disorders and Dementias

Objective The objective of this study is to ascertain individuals with a clinical diagnosis of a movement disorder or dementia, their affected and unaffected family members, and unrelated, healthy individuals (to provide control samples); to characterize their phenotypes; and to identify and further characterize genetic contributions to etiology by collecting ...

Phase N/A

A Study of RO7034067 in Adult and Pediatric Participants With Spinal Muscular Atrophy

This is a multi-center, exploratory, non-comparative and open-label study to investigate the safety, tolerability, pharmacokinetic (PK) and PK/pharmacodynamic (PD) relationship of RO7034067 in adults and children with Type 2 and Type 3 Spinal Muscular Atrophy (SMA) who have been previously enrolled in a study with a survival of motor neuron ...

Phase

Answer ALS: Individualized Initiative for ALS Discovery

Patients will have 5 study visits; screening, 3, 6, 9 and 12 months. There will be a one year post-participation follow-up period, during which they will receive an email or phone call interview once every 3 months. During the first year, samples will be collected, breathing, muscle strength, spasticity, general ...

Phase N/A

Delineating Swallowing Impairment and Decline in ALS

This research study is being performed to determine screening tools or tests that are able to identify and track swallowing problems associated with Amyotrophic Lateral Sclerosis (ALS) over time. Also, this study will provide insight into the natural progression of swallowing impairment in persons with ALS over time. Participants enrolled ...

Phase N/A