Clinical trials: Hereditary Spastic Paraplegia Type 51

HSP, Hereditary Spastic Paraplegia

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

• Not yet recruiting
• Age: 12 to 60 months
• Phase 1/2

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.

Conditions

HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50

Trial name

NCT06948019

Testing

BFB-101 (AAV9-CBh-AP4B1)

Where

United States

Volunteers

5 people

Sponsor

BlackfinBio Ltd