Status: Recruiting
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.
Participants needed: 4
Trial details
Phase: Phase 1, Phase 2Age: 4-10Biological sex: AllType: InterventionalSponsor: Elpida Therapeutics SPCUpdated: Oct 8, 2024Locations: 1
Eligibility criteria
Age 4 months-10 years old [+7]
Inability to participate in study procedures (as determined by the site investig... [+13]