MedTrace Logo

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

NCT06948019 · Status: NOT_YET_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2025-04-28

No results posted yet for this study

Summary

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

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

BIOLOGICAL

BFB-101 (AAV9-CBh-AP4B1)

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.

Sponsors & Collaborators

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Months
Max Age
60 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-08-31
Primary Completion
2030-08-31
Completion
2032-08-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06948019 on ClinicalTrials.gov