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Neuromuscular

For information on Neuromuscular clinical trials, please contact Xin Di Dong.

Current Recruiting Studies

Pompe Disease Registry

Genzyme has established large, multinational databases to which physicians contribute clinical data on patients with several lysomal storage disorders (LSDs) such as Pompe disease. These are called registries. Registries help pool knowledge and improve understanding of these rare diseases. The Registry program was started worldwide in 1991 for patients with Gaucher disease, to help health care providers better understand that disease. Since that time, additional registries have been added for Pompe disease, Fabry disease and MPS I. Since these rare diseases have no known cure, it is expected that each Registry will continue for a number of years. People with these rare diseases can participate in the Registry regardless of whether or not they are receiving any treatment. The Registry is sponsored and run by Genzyme, a Sanofi company.

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A multicenter, prospective, randomized, placebo-controlled, double-blind, parallel group clinical trial to assess the efficacy and safety of Immune Globulin Intravenous (Human) Flebogamma® 5% DIF in patients with Post-Polio Syndrome

From clinicaltrials.gov:

This is a multicenter, prospective, randomized, placebo-controlled, double-blind, parallel group clinical trial with adaptive dose selection in subjects with post polio syndrome (PPS).

The main purpose of this study is to select a dose of Flebogamma 5% DIF and confirm the efficacy of the selected Flebogamma 5% DIF dose by assessing physical performance, as measured by 2 Minutes Walk Distance (2MWD) test.

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A Phase 2a, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of RVT-1401 in Myasthenia Gravis Patients

From clinicaltrials.gov:

The purpose of the current study is to assess safety/tolerability and key pharmacodynamic (PD) effects that are considered to be associated with clinical benefit (reduction of total IgG and anti-AChR-IgG) in Myasthenia Gravis patients following treatment with RVT-1401 (also known as IMVT-1401) compared to placebo.

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A Phase 2a, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of RVT-1401 in Myasthenia Gravis Patients

From clinicaltrials.gov:

This study is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, multicenter study designed to evaluate the efficacy and safety of losmapimod in treating patients with Facioscapulohumeral Muscular Dystrophy (FSHD) over 24 weeks. Patients will participate in this study for approximately 29 weeks. This will include a 4-week screening period, a 24-week, placebo-controlled treatment period and a 7 day safety follow-up period. Patients must have a confirmed diagnosis of FSHD1 and genetic confirmation must be obtained prior to the screening MRI and baseline muscle biopsy. Patients will be randomized to receive 15 mg of losmapimod or placebo twice daily given as two 7.5 mg tablets per dose by mouth; for a total of 4 pills or 30 mg daily for 24 weeks. All patients will be asked to attend the study clinic for each scheduled visit.

The primary endpoint of the study is to evaluate the efficacy of losmapimod in inhibiting or reducing expression of DUX4, as measured by a subset of DUX4-regulated gene transcripts in skeletal muscle biopsies of FSHD patients. Secondary endpoints include evaluation of the safety and tolerability of losmapimod, the plasma concentrations of losmapimod, levels of losmapimod in skeletal muscle and losmapimod target engagement in blood and skeletal muscle in FSHD patients.

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Active Studies (not recruiting)

A Phase 2, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of RA101495 in Subjects with Generalized Myasthenia Gravis

From clinicaltrials.gov:

The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with generalized Myasthenia Gravis (gMG). Subjects will be randomized in a 1:1:1 ratio to receive daily SC doses of 0.1 mg/kg RA101495, 0.3 mg/kg RA101495, or matching placebo for 12 weeks.

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A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase 3 Trial to Evaluate the Efficacy, Safety and Tolerability of ARGX-113 in Patients with Myasthenia Gravis Having Generalized Muscle Weakness

From clinicaltrials.gov:

This is a randomized, double-blind, placebo controlled, multicenter Phase 3 trial to evaluate the efficacy, safety, tolerability, quality of life and impact on normal daily activities of ARGX-113 in patients with gMG.

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The A phase 3 randomized, multicenter, multinational, double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late-onset Pompe disease

From clinicaltrials.gov:

The duration of the study per patient will be up to 3 years that will consist of a 14- day screening period (may be extended up to 8 weeks in pre-specified situations), a 49-week blinded treatment period, a 96-week open-label treatment period, and a 4-week post-treatment observation period.

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