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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 (44) clinical trials

Clinical Trial of Ezogabine (Retigabine) in ALS Subjects

One of the major disease features of ALS is the progressive death of motor neurons. Human, rodent and stem cell-based model studies support the hypothesis that neuronal hyperexcitability may contribute to neurodegeneration in both sporadic and familial ALS. The investigators are doing this research study to find out whether retigabine ...

Phase

Safety and Biomarker Study of EPI-589 in Subjects With ALS

Open label with 30-day run in phase to establish baseline parameters and a 90-day withdrawal phase to determine duration of treatment response.

Phase

A Trial of Tocilizumab in ALS Subjects

This is a multicenter, randomized, double-blind, placebo-controlled 16-week study evaluating the safety and tolerability of tocilizumab in subjects with sporadic ALS. The primary objective of the study is to determine the safety and tolerability of intravenous administration of 8 mg/kg of tocilizumab every 4 weeks vs. matched intravenous placebo administered ...

Phase

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 Study of CK-2127107 in Patients With Spinal Muscular Atrophy

This is the first study being conducted in these patients and is designed to assess the effect of 8 weeks of dosing of CK-2127107 on measures of muscle function in both ambulatory and non-ambulatory patients with SMA. The plasma concentration of CK-2127107 will be measured at selected time points during ...

Phase

A Study of GDC-0134 to Determine Initial Safety Tolerability and Pharmacokinetic Parameters in Participants With Amyotrophic Lateral Sclerosis

This first-in-human, double-blind, placebo-controlled Phase I study will be conducted in participants with amyotrophic lateral sclerosis (ALS) to explore safety, tolerability, and pharmacokinetic (PK) properties of GDC-0134. It will include two components: a Single-Ascending-Dose (SAD) stage and a Multiple-Ascending-Dose (MAD) stage.

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

Oral Nutritional Supplementation in Amyotrophic Lateral Sclerosis (ALS) Patients

Amyotrophic lateral sclerosis (ALS) is a rare neurodegenerative disease with a median age at time of diagnosis of 65 years. In France, the incidence ranges between 1.5 and 2.5/100 000 person-year of follow-up. The disease is related to progressive degeneration of motor neurons in the two voluntary motor pathways. It ...

Phase N/A

Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene

Spinal Muscular Atrophy (SMA) is a neuromuscular disorder characterized by loss of motor neurons in the anterior horn of the spinal cord and leading to muscle atrophy. SMA has an autosomal recessive inheritance and affects 1 in 6000 infants with a carrier frequency of 1 in 40. In most cases, ...

Phase N/A

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

CoRDS collects contact, sociodemographic and health information about participants. This information is entered into CoRDS and linked to a unique coded identifier. Below are some examples of information requested on the Questionnaire that will be entered into CoRDS: - Contact information: Name, Mailing Address, Phone Number, Email Address - Sociodemographic ...

Phase N/A