Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Not yet recruiting · NCT06948019 · Registry updated 2025-04-28Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47) | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscle Hypertonia, Muscle Spasticity, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, central nervous system disorder, central nervous system malformation, hereditary disease, hereditary motor and sensory neuropathy, hereditary spastic paraplegia, hereditary spastic paraplegia 47, hereditary spastic paraplegia 50, hereditary spastic paraplegia 51, hereditary spastic paraplegia 52, inherited neurodegenerative disorder, movement disorder, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, polyneuropathy | Not yet recruiting | Phase 1 / Phase 2 | 2025-04-28 | NCT06948019 |
2 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| BlackfinBio Ltd | 1 |
| Boston Children's Hospital | 238 |
1 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Boston Children's Hospital | Boston | United States | 343 |