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Intrathecal AAV9/AP4M1 (Melpida) Gene Therapy for SPG50
2025-524481-15-00Phase 2Authorised
- Sponsor
- Rigshospitalet
- Start
- Oct 2026
- Modality
- Recombinant Proteins and Enzymes, Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of MELPIDA in Hereditary spastic paraplegia type 50 (SPG50), sponsored by Rigshospitalet. Primary endpoint: Change in total percent score of the eight Major Motor Milestones from GMFM-88 at Week 156. Planned start Oct 2026; status: authorised.
02
Drugs
MELPIDA
03
Conditions
Hereditary spastic paraplegia type 50 (SPG50)
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