CURRENT RECRUITING TRIALS

CRU-SCIG
Switch from intravenous to subcutaneous immunoglobulin in patients with myasthenia gravis (MG) and chronic inflammatory demyelinating polyneuropathy (CIDP): A single-arm, open-label, prospective pilot study to determine efficacy, treatment satisfaction, health care resource utilization and tolerability

The aim of this study will investigate efficacy, treatment satisfaction, health care resource utilization and tolerability of SCIg in MG and CIDP patients previously treated with IVIg. . The data gathered during the study may also be useful for medical professionals and researchers in the future.

Immunovant 1401-2002
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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SPINRAZA Washington University Observational
A prospective, multi-center, observational study of the safety, tolerability and effectiveness of SPINRAZA in adult patients with Spinal Muscular Atrophy

From clinicaltrials.gov:

This is a longitudinal, observational study of adult patients with genetically confirmed chromosome 5q SMA to examine the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) for up to 30 months.

This is a prospective, longitudinal, multi-center, observational study designed to evaluate the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) in ambulatory and non-ambulatory adult patients with SMA. Subjects with SMA II/III that are 18 years to 70 years of age who are planning to initiate treatment with SPINRAZA® (nusinersen) as part of their clinical care plan will be enrolled in this study. This study does not provide SPINRAZA® (nusinersen) or cover costs associated with standard clinical care.These patients will be treated by their respective physicians according to standard clinical practice. Study visits including standardized assessments of strength and function will occur at baseline, day 15 after treatment initiation, day 30, day 70, and then 4-month intervals through month 30.

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Fulcrum Gene Therapy

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD) with Open-Label Extension (OLE)

The purpose of the 48 week placebo-controlled treatment period of this study is is to determine whether losmapimod may slow down or stop the progression of the disease in individuals with FSHD by preventing or decreasing DUX4 expression in skeletal muscle and other affected organs. This study will also evaluate how you feel when you are taking the study drug and assess how you think the study drug may be affecting the progression of your disease.

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FORCE
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)

The aim of this research is to test how well a human immunoglobulin concentrate named Flebogamma® 5% DIF (hereafter referred to as Flebogamma) improves your PPS symptoms such as walking capacity, pain, overall quality of life, fatigue and muscle strength. Flebogamma is the investigational drug. Investigational means that is has not been approved by any health authority (government authorities) such as the US Food and Drug Administration (FDA) or Health Canada (HC) for improving your PPS symptoms effectively and safely. However, HC does not object to the use of Flebogamma in this study.

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Pompe Disease Registry
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)

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.

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

UPCOMING TRIALS

UCBMG0003 Mg CarinG
A phase 3, randomized, double-blind, placebo-controlled study evaluating efficacy and safety of rozanolixizumab in adult patients with generalized myasthenia gravis

From clinicaltrials.gov:

The purpose of the MycarinGstudy is to demonstrate the clinical efficacy and to assess safety and tolerability of rozanolixizumab in patients with generalized myasthenia gravis (MG).

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RAISE Ra Pharma Phase 3
A Phase 3, Multicenter, Randomized, Double- Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects with Generalized Myasthenia Gravis

From clinicaltrials.gov:

The RAISE study is a multicenter, randomized, double-blind, placebo controlled study to confirm the efficacy, safety, and tolerability of zilucoplan in subjects with generalized Myasthenia Gravis. Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg zilucoplan or placebo for 12 weeks.

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ACTIVE (NOT RECRUITING) TRIALS

RA101495-02.201
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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ARGX-113
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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EFC14028
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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