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.
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.
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.
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.
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.