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AAV9-IGHMBP2 Gene Therapy for IGHMBP2-Related Diseases
NCT05152823Phase 2Enrolling by invitation
- Start
- Nov 2021
- Primary completion
- Jul 2028
- Modality
- Gene Editing, Gene Silencing, Viral Vectors
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of ACTX-401 in Spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot-Marie-Tooth disease type 2S (CMT2S). Primary endpoint: Unacceptable toxicity at 3 years. Started Nov 2021; status: enrolling by invitation.
02
Drugs
ACTX-401
03
Conditions
Spinal muscular atrophy with respiratory distress type 1 (SMARD1)Charcot-Marie-Tooth disease type 2S (CMT2S)
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