Trial Profile

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

Status: Not yet recruiting

Phase of Trial: Phase I/II

Latest Information Update: 26 Oct 2025

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At a glance

05 May 2025 New trial record
28 Apr 2025 Status changed from planning to not yet recruiting.
28 Apr 2025 According to a BlackfinBio media release, the trial will be conducted in the U.S. at Boston Children Hospital and is expected to commence recruiting by the end of 2025.

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