We are enthusiastic about the merging treatment options for neuromuscular disease, which are indicated by the growing number of multicenter clinical trials set up or sponsored by the pharmaceutical industry. We are currently conducting a number of trials listed below in our neuromuscular clinical trial unit. The unit consists of 3 project coordinators/research nurses, 1 research secretary, and 0.35 medical doctor, who are fully dedicated to work in the trial unit. In addition, 5 other doctors, 2 lab technicians and 2 physiotherapists also participate in the work of the trial unit.
ONGOING STUDIES
NeoGAA LTS13769 An open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusion og neoGAA in patients with Pompe disease. Sponsor: Sanofi/Genzyme. Clinicaltrials.gov identifier NCT02032524.
ARGX-113 A safety and tolerability study of ARGX-113 in patients with myasthenia gravis who have generalized muscle weakness. Sponsor: Argenx. Clinicaltrials.gov identifier NCT03770403.
GNT-015-FKRP Natural history study of patients with limb-girdle muscular dystrophy 2I. Sponsor: Genethon. Clinicaltrials.gov identifier NCT03842878.
Alexion 1210 (Ravulizumab) Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis. Sponsor: Alexion. Clinicaltrials.gov identifier NCT03920293.
DYN101 Early phase human drug trial to investigate dynamin 101 (DYN101) in patients ≥ 16 years with centronuclear myopathies (Unite-CNM). Sponsor: Dynacure. Clinicaltrials.gov identifier NCT04033159.
MG0003 (Rozanolixizumab/UCB7665) A study to test efficacy and safety of Rozanolixizumab in adult patients with generalized myasthenia gravis. Sponsor: UCB Biopharma. Clinicaltrials.gov identifier NCT039771422.
MG0007 (Rozanolixizumab/UCB7665) A study to evaluate Rozanolixizumab in study participants with generalized myasthenia gravis. Sponsor: UCB Biopharma. Clinicaltrials.gov identifier NCT04650854.
CIDP04 (Rozanolixizumab/UCB7665) A study to assess long-term safety, tolerability and efficacy of Rozanolixizumab in subjects with chronic inflammatory demyelinating polyradiculoneuropathy. Sponsor: UCB . Clinicaltrials.gov identifier NCT04051944.
REN001-201 A study of the efficacy and safety of 24 week treatment with REN001 in patients with primary mitochondrial myopathy (STRIDE). Sponsor: Reneo Pharmaceuticals. Clinicaltrials.gov identifier NCT04535609.
REN001-903 A prospective study of the disease characteristics of adult patients with long-chain fatty acid oxidation disorders (FORWARD). Sponsor: Reneo Pharmaceuticals. Clinicaltrials.gov identifier NCT04482049.
COS2 Natural history study of patients with limb-girdle muscular dystrophy 2B. Sponsor: Jain Foundation.
MLB-01-001 Biomarker development in LGMD2I. Sponsor: Virginia Commonwealth University/ML BioSolutions. Clinicaltrials.gov identifier NCT04202627.
Inebilizumab (VIB0551) Myasthenia Gravis Inebilizumab Trial (MINT). Sponsor: Viela Bio. Clinicaltrials.gov identifier NCT04524273.
PXT3003-06 Phase III trial assessing the efficacy and safety of PXT3003 in CMT01A patients (PREMIER). Sponsor: Pharnext. Clinicaltrials.gov identifier NCT04762758.
Satralizumab (WN42636) A phase III, randomized, double-blind, placebo-controlled, multicenter study to evaluate efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab in patients with generalized myasthenia gravis. Sponsor: Roche. EU Clinical Trials Register EudraCT# 2020-004436-21.
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