Clinical trials

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.



RECENTLY COMPLETED STUDIES 

ECU-MG-301 A randomized, double-blind, placebo-controlled, multi-center study to evaluate the safety and efficacy of Eculizumab in subjects with refractory generalized Myasthenia Gravis (gMG). Sponsor: Alexion Pharmaceuticals Inc. Clinicaltrials.gov identifier NCT01997229.

CBVS857X2202  A two-part placebo-controlled study to evaluate the safety, tolerability and preliminary efficacy of BVS857 in patients with spinal and bulbar muscular atrophy (SBMA). Sponsor: Novartis. Clinicaltrials.gov identifier NCT02024932.

CBYM338B2203 A randomized, double-blind, placebo-controlled, multicenter, parallel group, dose-finding, pivotal, phase 2b/3 study to evaluate the efficacy, safety and tolerability of intravenous administration in 52 weeks on physical function, muscle strength, and mobility and additional long-term safety up to 2 years in patients with sporadic inclusion body myositis. Sponsor: Novartis. Clinicaltrials.gov identifier NCT01925209.

ATYR1940-c-004 An open-label, interpatient dose escalation study to evaluate the safety, tolerability, immunogenicity, and biological activity of ATYR1940 in patients with Limb Girdle and Facioscapulohumeral Muscular Dystrophies. Sponsor: aTYR Pharma Inc. Clinicaltrials.gov identifier NCT02579239.

NeoGAA TDR12857 An open-label, multicenter, multinational, ascending dose study of the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of repeated biweekly infusion og neoGAA in naïve and alglucosidase alfa treated late-onset Pompe disease patients. Sponsor: Sanofi/Genzyme. Clinicaltrials.gov identifier NCT01898364.

 

ONGOING STUDIES

ECU-MG-302 A phase 3, open-label, extension trial of ECU-MG_301 to evaluate the safety and efficacy of Eculizumab in subjects with refractory generalized Myasthenia Gravis (gMG). Sponsor: Alexion Pharmaceuticals, Inc. Clinicaltrials.gov identifier NCT02301624.

CBYM338B2203E1 Extension of the CBYM338B2203 phase 2b/3 study to evaluate the long-term efficacy, safety and tolerability of intravenous BYM338 in patients with sporadic inclusion body myositis. Sponsor: Novartis. Clinicaltrials.gov identifier NCT02573467.

CBYM338E2202 A 28-week, randomized, double-blind, placebo-controlled, multi-center, parallel group dose range finding study to assess the effect of monthly doses og bimagrumab 70, 210, and 700 mg on skeletal muscle strength and function in older adults with Sarcopenia. Sponsor: Novartis. Clinicaltrials.gov identifier NCT02333331.

CCFZ533X2204 A multi-center, randomized, double-blind, placebo-controlled, parallel group study to preliminarily evaluate the safety, tolerability, pharmacokinetics and efficacy of CFZ533 in patients with moderate to severe Myasthenia Gravis. Sponsor: Novartis. Clinicaltrials.gov identifier NCT02565576.

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.

ATYR1940-c-006 An open-label extension study to evaluate the long-term safety, tolerability, and biological activity of ATYR1940 in patients with Limb Girdle and Facioscapulohumeral Muscular Dystrophy. Sponsor: aTYR Pharma Inc. Clinicaltrials.gov identifier NCT02836418.

IG1104 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 Syndrom. Sponsor: Grifols. Clinicaltrials.gov identifier NCT02176863.

HyQvia A randomized, single-blinded, cross-over study investigating the non-inferiority of efficacy and safety in comparison with conventional subcutaneous Ig therapy in Multifocal Motor Neuropathy. Sponsor: Baxalta/Shire. Clinicaltrials.gov identifier NCT02556437.

408-C-1403 A phase 2 study of the safety, efficacy, and pharmacodynamics of RTA-408 in the treatment of mitochondrial myopathy. Sponsor: Reata Pharmaceuticals. Clinicaltrials.gov identifier NCT02255422.

C15-54 (UX007-IST062) The effect of Triheptanoin in adults with McArdle Disease (Glycogen Storage Disease Type V). Sponsor: Ultragenyx Pharmaceuticals. Clinicaltrials.gov identifier NCT02919631.

 

SCHEDULED UPCOMING STUDIES

SPIMM-300 A prospective observational study of patients with primary mitochondrial disease. Sponsor: Stealth Bio Therapeutics Inc. 

MG002 A multicenter, randomized, investigator-and subject-blind, placebo-controlled, treatment sequence study evaluating the safety, tolerability, and efficacy of UCB7665 in subjects with moderate to severe Myasthenia Gravis. Sponsor: UCB Biopharma SPRL.