- Conditions
- HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50, Hereditary Spastic Paraplegia Type 47, Hereditary Spastic Paraplegia Type 51, Hereditary Spastic Paraplegia Type 52, SPG47, AP4B1, Neurogenetic Disorders, Neurodevelopmental Conditions, Movement Disorders, Gene Therapy
- Interventions
- BFB-101 (AAV9-CBh-AP4B1)
- Biological
- Lead sponsor
- BlackfinBio Ltd
- Industry
- Eligibility
- 12 Months to 60 Months
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
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- Conditions
- Hereditary Spastic Paraplegia Type 50
- Interventions
- MELPIDA
- Genetic
- Lead sponsor
- Elpida Therapeutics SPC
- Industry
- Eligibility
- 4 Months to 72 Months
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2032
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Spasticity, Muscle, Microcephaly, Intellectual Deficiency, Growth Retardation, SPG50, Spastic Paraplegia
- Interventions
- MELPIDA
- Biological
- Lead sponsor
- Elpida Therapeutics SPC
- Industry
- Eligibility
- 4 Months to 10 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2030
- U.S. locations
- 1
- States / cities
- Dallas, Texas