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MELPIDA in Spastic Paraplegia Type 50 (SPG50)
NCT06069687Phase 1Active, not recruiting
- Sponsor
- SickKids
- Start
- Mar 2022
- Primary completion
- Mar 2027
- Modality
- Gene Editing, Gene Silencing, Viral Vectors
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of MELPIDA in Spastic Paraplegia Type 50 (SPG50), sponsored by SickKids. Primary endpoints: Incidence of unanticipated treatment-related toxicities, Grade 3 or higher at 5 years, Change from baseline in nerve conduction velocity at Month 60 and 2 more. Started Mar 2022; status: active, not recruiting.
02
Drugs
MELPIDA
03
Conditions
Spastic Paraplegia Type 50 (SPG50)
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