Drugs / MELPIDA
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT06069687 | Mar 2022 → Mar 2027 expected | hereditary spastic paraplegia | The Hospital for Sick Children | Active not recruiting | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT05518188 | Feb 2023 → Oct 2028 expected | hereditary spastic paraplegia, intellectual disability, microcephaly | Elpida Therapeutics SPC | Recruiting | No outcome recorded |
| Phase 31 trial | ||||||
| Phase 3 | NCT06692712 | Apr 2026 → Feb 2032 expected | hereditary spastic paraplegia 50 | Elpida Therapeutics SPC | Recruiting | No outcome recorded |
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | MELPIDA | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05518188 ↗ |
| Known as | AAV-AP4M1 | “MELPIDA (AAV-AP4M1) represents one of the clinical programs in its developmental pipeline.” PMID 38942994 ↗ Jun 2024 |
| Action | Restore | “to deliver a fully functional human AP4M1 cDNA copy via intrathecal injection to counter the associated neuronal loss” NCT05518188 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “a recombinant serotype 9 adeno-associated virus (AAV) encoding a codon-optimized human AP4M1 transgene” NCT05518188 ↗ |
| Route | Intrathecal | “via intrathecal injection” NCT05518188 ↗ |
| Target | AP4M1 | “encoding a codon-optimized human AP4M1 transgene” NCT06069687 ↗ |