CURRENT RECRUITING TRIALS
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.
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.
SPINRAZA Washington University Observational
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.
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.
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.
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.
UCBMG0003 Mg CarinG
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).
RAISE Ra Pharma Phase 3
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.
ACTIVE (NOT RECRUITING) TRIALS
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.
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.
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.