NCT06948019 - Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47) | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

August 31, 2025

Primary Completion Date

August 31, 2030

Study Completion Date

August 31, 2032

Conditions

HSPHereditary Spastic ParaplegiaHereditary Spastic ParaparesisHereditary Spastic Paraplegia Type 50Hereditary Spastic Paraplegia Type 47Hereditary Spastic Paraplegia Type 51Hereditary Spastic Paraplegia Type 52SPG47AP4B1Neurogenetic DisordersNeurodevelopmental ConditionsMovement DisordersGene 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.

Trial Locations (1)

02115

Boston Children's Hospital, Boston