# Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)

> **NCT06948019** · PHASE1,PHASE2 · NOT_YET_RECRUITING · sponsor: **BlackfinBio Ltd** · enrollment: 5 (estimated)

## Conditions studied

- 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

- **BIOLOGICAL:** BFB-101 (AAV9-CBh-AP4B1)

## Key facts

- **NCT ID:** NCT06948019
- **Lead sponsor:** BlackfinBio Ltd
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** NOT_YET_RECRUITING
- **Start date:** 2025-08
- **Primary completion:** 2030-08
- **Final completion:** 2032-08
- **Target enrollment:** 5 (ESTIMATED)
- **Last updated:** 2025-04-28

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT06948019

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT06948019, "Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)". Retrieved via AI Analytics 2026-06-16 from https://api.ai-analytics.org/clinical/NCT06948019. Licensed CC0.

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