Klinische Studien

Duchenne Muskeldystrophie

• EMBARK SRP-9001-301 Eudra CT 2019-003374-91
  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

• MISSION SRP 4658-402  EudraCT 2018-001762-42
  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

• MOMENTUM SRP 5051-201 EudraCT: 2019-000601-77
  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

• ESSENCE SRP 4045-301 EUDRA CT NUMMER:2015-002069 5
  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 - Study of SRP-4045 and SRP-4053  in DMD Patients                                           

• SRP 4045-302 EUDRACT Number: 2017-004625-32
  Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen

• DSC/142357/51 EUDRACT: 2017-000397-10
  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.

• PTC PASS
  Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care (Anwendungsbeobachtungsstudie PTC124-GD-025o-DMD)

Spinale Muskelatrophie

• Biogen 232SM303 ASCEND EUDRA CT NUMBER: 2021-001294-23
  A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients with Spinal Muscular Atrophy Previously Treated with Risdiplam

• Sapphire - SRK-015-003 EudraCT Number: 2021-005314-34
  Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy

• SHINE ISIS 396443-CS11 EudraCT No: 2015-001870-16
  An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443

Myasthenia gravis

• ARGX-113-2006 EudraCT: 2020-005841-18
  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

Gliedergürteldystrophie

• SRP-LGMD-501-NH
  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)

 In Vorbereitung

DMD

• PFIZER C3391003 Eudra CT 2019-002921-31
  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

• BN 43881 Envol   EudraCT 2022-000691-19
  A TWO-PART, OPEN-LABEL SYSTEMIC GENE DELIVERY STUDY TO EVALUATE THE SAFETY AND EXPRESSION OF RO7494222 (SRP-9001) IN SUBJECTS UNDER THE AGE OF FOUR WITH DUCHENNE MUSCULAR DYSTROPHY

• SRP 9001-303 Envison EudraCT 2020-002372-13
  A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy

LGMD

• MLB-01-005 LGMD  IND Number: 145713
  Protocol Title: A Phase 3 Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of BBP-418 (ribitol) in Patients with Limb Girdle Muscular Dystrophy 2I (LGMD2I)

SMA

• BN 42644 Manatee  2021-003417-19
  A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY,   TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN    AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY
• BHV-200-301 RESILIENT Eudra CT 2022-000193-25
  A Randomized, Double-Blind, PlaceboControlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension