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Phase 1
A Phase 1/2 Open-label Intrathecal Administration of MELPIDA to Determine Its Safety and Efficacy for Patients with Spastic Paraplegia Type 50 (SPG50) Caused by Mutation in the AP4M1 Gene.
MELPIDA is proposed for the treatment of subjects with SPG50 and targets neuronal cells to deliver a fully functional human AP4M1 cDNA copy via intrathecal inj…
Spasticity, MuscleMicrocephalyIntellectual Deficiency+3 more
Elpida Therapeutics SPCNCT05518188
Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP)
The Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP) is focused on gathering longitudinal clinical data as well as biolog…
Hereditary Spastic ParaplegiaSPG47SPG50+8 more
Boston Children's HospitalNCT04712812