Myotubular Myopathy

  • NatHis-MTM: Prospective Natural History Study of Patients With Myotubular Myopathy and Other CentroNuclear Myopathies
  • INCEPTUS: A Prospective, Non-Interventional Clinical Assessment Study in X Linked Myotubular Myopathy (XLMTM) Subjects Aged 3 Years and Younger
  • ASPIRO: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients
  • Dyn-101: 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

 

Acronym

NatHis-MTM

INCEPTUS

ASPIRO

Dyn-101

Study Type RBM-HPS RBM-HPS RBM
Intervention AT132 DYN101
Trial type International multicenter trial International multicenter trial International multicenter trial
Principal investigators
Andreea Seferian Andreea Seferian Andreea Seferian Anthony Béhin
Sponsor Valerion and AIM Audentes Audentes Dynacure
Fundings Valerion Audentes Audentes
Study status Ongoing Closed Ongoing In preparation
Recruitment status Closed Closed Closed
Population Adult / Child Child Child Adult
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