RecruitingPhase 1Phase 2NCT05518188

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

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.

Sponsor

Elpida Therapeutics SPC

Enrollment

4 participants

Start Date

Feb 15, 2023

Study Type

INTERVENTIONAL

Conditions

Spasticity, Muscle Spastic Paraplegia Microcephaly SPG50 Growth Retardation Intellectual Deficiency

Summary

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.

Eligibility

Min Age: 4 MonthsMax Age: 10 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a biological treatment called MELPIDA for people with growth retardation, intellectual deficiency, and other related conditions. The study is currently recruiting participants at 1 location. People eligible for this study include aged 4 Months to 10 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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