hereditary spastic paraplegia 50
hereditary spastic paraplegia 50
Explore linked clinical trials. Standard reference: MONDO:00130484 results ยท Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt) | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Malformations of Cortical Development, Group I, Muscle Hypertonia, Muscle Spasticity, Musculoskeletal Abnormalities, Neurologic Manifestations, Neuromuscular Manifestations, Paraplegia, Spastic, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, central nervous system malformation, cerebral cortical dysplasia, hereditary spastic paraplegia 50, microcephaly, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, palsy, paraplegia | Recruiting | Phase 1 / Phase 2 | 2026-07-08 | NCT05518188 | |
| Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls. | Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, hereditary spastic paraplegia 50, nervous system disorder, palsy, paraplegia | Recruiting | Phase 3 | 2026-04-20 | NCT06692712 | |
| Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, central nervous system malformation, hereditary disease, hereditary motor and sensory neuropathy, hereditary spastic paraplegia, hereditary spastic paraplegia 11, hereditary spastic paraplegia 15, hereditary spastic paraplegia 3A, hereditary spastic paraplegia 4, hereditary spastic paraplegia 47, hereditary spastic paraplegia 50, hereditary spastic paraplegia 51, hereditary spastic paraplegia 52, inherited neurodegenerative disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, palsy, paraplegia, peripheral nervous system disorder, polyneuropathy | Recruiting | Not stated | 2026-03-18 | NCT04712812 | |
| 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 |