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)
