Trial Profile

A Phase 1/2, Multicenter, Open-label, Dose Escalation and Expansion Clinical Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of ASP2957 in Male Participants With Invasive Ventilator-dependent X-linked Myotubular Myopathy

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 01 Jun 2026

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At a glance

Drugs ASP 2957 (Primary) ; Methylprednisolone (Primary) ; Prednisolone (Primary) ; Sirolimus (Primary)
Indications Congenital structural myopathies
Focus Adverse reactions; First in man
Sponsors Astellas Gene Therapies

08 Oct 2025 Planned End Date changed from 31 Aug 2027 to 31 Oct 2027.
08 Oct 2025 Planned primary completion date changed from 31 Aug 2027 to 31 Oct 2027.
08 Oct 2025 Status changed from not yet recruiting to recruiting.

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