Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human
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 tre
| Condition(s) | Spasticity, Muscle, Microcephaly, Intellectual Deficiency, Growth Retardation, SPG50, Spastic Paraplegia |
|---|---|
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Study type | Interventional |
| 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. |
| Who can participate | Inclusion Criteria: 1. Age 4 months-10 years old 2. Confirmed diagnosis of SPG50 disease by: 1. Genomic DNA mutation analysis demonstrating homozygous or compound heterozygous, confirmed pathogenic variants in the AP4M1 gene 2. Clinical history or examination features consistent with SPG50 and that include neurologic dysfunction 3. Parent/legal guardian willing to provide written informed consent for their child prior to participation in the study 4. Subject able to comply with all protocol requirements and procedures 5. Ability to stand for more than 5 seconds OR 6. Ability to take 5 steps independently or with a walker OR 7. Modified Ashworth Scale score 2 or below (Ankles). Exclusion Criteria: 1. Inability to participate in study procedures (as determined by the site investigator) 2. Pr |
| Ages | 4 Months to 10 Years |
| Sex | All |
| Lead sponsor | Elpida Therapeutics SPC |
| Locations | Dallas, Texas, United States |
| Start date | 2023-02-15 |
| NCT ID | NCT05518188 |
| Official listing | https://clinicaltrials.gov/study/NCT05518188 |