Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)

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 injection to counter the associated neuronal loss. Outcomes will evaluate the safety and tolerability of a single dose of MELPIDA, which will be measured by the treatment-associated adverse events (AEs) and serious adverse events (SAEs). Secondarily, the trial will explore efficacy in terms of disease burden assessments.

NCT Number	NCT05518188
Lead Sponsor	Elpida Therapeutics SPC Collaborators: University of Texas Southwestern Medical Center
Conditions	Spasticity, Muscle, Microcephaly, Intellectual Deficiency, Growth Retardation, SPG50, Spastic Paraplegia
Enrollment	4 participants
Start Date	2023-02-15
Primary Completion	2028-10-01 (estimated)
Study Completion	2030-10-01 (estimated)
Updated on ClinicalTrials.gov	2026-07-08