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Clinical trial

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

NCT06948019 is registered in ClinicalTrials.gov with BlackfinBio Ltd as lead sponsor.

NCT NCT06948019
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Registry record

As filed in ClinicalTrials.gov.

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
Lead sponsor
BlackfinBio Ltd
Phase
Phase 1/2
Overall status
Not yet recruiting
Start date
August 31, 2025
Completion date
August 31, 2032
Enrollment
5 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

ap4b1, gene therapy, 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, hsp, movement disorders, neurodevelopmental conditions, neurogenetic disorders, spg47