Klinische Studien

Duchenne Muskeldystrophie

• PTC PASS
  Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care (Anwendungsbeobachtungsstudie PTC124-GD-025o-DMD)
• SRP 4045-301
  Study of SRP-4045 and SRP-4053 in DMD Patients (ESSENCE) A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy (EudraCT: 2015-002069-52)
• SRP 4045-302
  Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen (EudraCT:2017004625-32)
• TAM DMD
  • Tamoxifen in Duchenne Muscular Dystrophy: A multicenter, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial
  • Tamoxifen in Duchenne Muscular Dystrophy: A 48-week open label extension of a multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy trial (EudraCT: 2017-004554-42)
• DSC/142357/48
  Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy (EudraCT: 2016-000401-36)
• DSC/142357/51
  Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies. (EudraCT: 2017-000397-10)

Centronukleäre Myopathien

• NatHis CNM
  Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)
• DYN101-C101
  A Phase 1/2 trial on the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of DYN101 in patients ≥ 16 years of age with centronuclear myopathies caused by mutations in DNM2 or MTM1. (EudraCT: 2018-004089-33)

Spinale Muskelatrophie

• ISIS 396443-CS11
  An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443 (EudraCT 2015-001870-16)
• Jewelfish BP 39054
  An open label study to investiagte the safety, tolerability, and pharmacokinetics/pharmacoynamics of Risdiplam RO7034067 in adult and pediatric patients with spinal muscular atrophy (EudraCT 2016-004184)

In Vorbereitung

• C3391003
  A phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne Muscular Dystrophy
• SRP-9001-301
  A Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy Who Are Aged 4-7 Years
• SRP 5051-201
  A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (EudraCT: 2019-000601-77)
• SRP 4658-402
  A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping (EudraCT 2018-001762-42)
• ARGX-113-2006
  Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis 2020-005841-18
• SRP-LGMD-501-NHS
  Journey: A Global, Multicenter, Longitudinal Study of the Natural History of Subjects with Limb Girdle Muscular Dystrophy (LGMD) Type 2E (LGMD2E/R4), Type 2D (LGMD2D/R3), and Type 2C (LGMD2C/R5)