Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Public ClinicalTrials.gov record NCT06948019. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Safety and Efficacy of AAV9/AP4B1 For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration. The goal of this clinical trial is to evaluate whether a gene therapy can safely treat children with SPG47, a rare genetic condition that causes progressive spasticity and developmental delays. The main questions it aims to answer are: * Is the gene therapy safe and well tolerated? * Does the gene therapy improve motor function and developmental outcomes? Participants will: * Undergo screening assessments to confirm eligibility * Receive a single dose of the gene therapy vector * Attend follow-up visits for safety monitoring and developmental assessments over the course of five years
Study identification
- NCT ID
- NCT06948019
- Recruitment status
- Not yet recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 5 participants
Conditions and interventions
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
Eligibility (public fields only)
- Age range
- 12 Months to 60 Months
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Aug 2025 (Estimated)
- Primary completion
- Aug 2030 (Estimated)
- Completion
- Aug 2032 (Estimated)
- Last update posted
- Apr 28, 2025
2025 – 2032
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06948019, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 28, 2025 · Synced Oct 21, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT06948019 live on ClinicalTrials.gov.