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

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
BlackfinBio Ltd
Study ID
NCT06948019
Phase
PHASE1/PHASE2
Status
Not Yet Recruiting

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Conditions

  • AP4B1
  • Gene Therapy
  • HSP
  • Hereditary Spastic Paraparesis
  • Hereditary Spastic Paraplegia
  • Hereditary Spastic Paraplegia Type 47
  • Hereditary Spastic Paraplegia Type 50
  • Hereditary Spastic Paraplegia Type 51
  • Hereditary Spastic Paraplegia Type 52
  • Movement Disorders
  • Neurodevelopmental Conditions
  • Neurogenetic Disorders
  • SPG47

Eligibility Criteria

Sex
ALL
Age
12 Months - 60 Months
Healthy Volunteers
Not accepted

Interventions

  • BFB-101 (AAV9-CBh-AP4B1) — BIOLOGICAL
    The AAV9-CBh-AP4B1 biological drug product is an aqueous suspension of a gene transfer vector intended for CSF injection. It consists of replication deficient adeno-associated virus (AAV) vector with the AAV serotype 9 capsid enclosing a single stranded DNA with an expression cassette of AP4B1 driven by CBh promoter.

Study Details

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

Key Dates

Start date
Aug 31, 2025
Status verified
Apr 2025
Primary completion
Aug 31, 2030
Completion
Aug 31, 2032

Study Design

Enrollment
5 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Treatment Arm
    BFB-101, a gene therapy product

Primary Outcome Measure

Incidence of unanticipated treatment-related toxicities, Grade 3 or higher in participants with SPG47 [ Time Frame: 60 months ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Boston Children's HospitalBostonMassachusetts02115
Darius Ebrahimi-Fakhari, MD, PhD
[email protected]
617-355-0097
Josh Rong, BS
[email protected]
617-355-0903
Darius Ebrahimi-Fakhari, MD, PhD (PRINCIPAL_INVESTIGATOR)

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By research site
Boston Children's Hospital· Boston, MA

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