Melpida
Sponsors
Azienda Socio Sanitaria Territoriale Papa Giovanni Xxiii, Rigshospitalet, Elpida Therapeutics SPC, Elpida Therapeutics SPC, The Hospital for Sick Children
Conditions
Growth RetardationHereditary Spastic Paraplegia Type 50Hereditary Spastic Paraplegia Type 50 (SPG50)Heriditary spastic paraplegia (SPG50)Intellectual DeficiencyMicrocephalySPG50Spastic Paraplegia
Phase 1
Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
RecruitingNCT05518188
Start: 2023-02-15End: 2030-10-01Target: 4Updated: 2026-07-08
Administration of MELPIDA to Determine the Safety and Efficacy for Patients With Spastic Paraplegia Type 50
Active, not recruitingNCT06069687
Start: 2022-03-11End: 2027-03-31Updated: 2025-05-14
Phase 2
A Phase IIb, Monocentric, Non-Profit, Open-label Trial for the Intrathecal Administration of AAV9/AP4M1 for Hereditary Spastic Paraplegia Type 50 (SPG50)
Not yet recruitingCTIS2025-522603-15-00
Target: 5Updated: 2026-02-06
A Phase IIb, Non-Profit, Open-label Trial for the Intrathecal Administration of AAV9/AP4M1 for Hereditary Spastic Paraplegia Type 50 (SPG50).
Not yet recruitingCTIS2025-524481-15-00
Target: 2Updated: 2026-06-23
Phase 3
Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls.
RecruitingNCT06692712
Start: 2026-04-01End: 2032-06-01Target: 24Updated: 2026-04-20
Intrathecal Administration of MELPIDA For Hereditary Spastic Paraplegia Type 50 (SPG50): A multicenter Phase 3, Open-Label Trial with Matched Prospective Concurrent Control Arm (CT-MEL-03)
Not yet recruitingCTIS2025-524576-28-00
Target: 12Updated: 2026-07-17