Melpida is named as an intervention in 2 current clinical trials in our USA and Canada registers. 1 is recruiting patients now. The most common phase is Phase 1/2. The sponsor with the most trials is Elpida Therapeutics SPC (1). The most-studied condition is Spasticity, Muscle. The EU clinical trials register (CTIS) separately lists 2 authorised or ongoing trials.
| Study | Phase | Status | Lead sponsor | Registers |
|---|---|---|---|---|
|
Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
NCT05518188 · ClinicalTrials.gov ↗ · Spasticity, Muscle, Microcephaly, Intellectual Deficiency
|
Phase 1/2 | Recruiting | Elpida Therapeutics SPC | USA |
|
Administration of MELPIDA to Determine the Safety and Efficacy for Patients With Spastic Paraplegia Type 50
NCT06069687 · ClinicalTrials.gov ↗ · Spastic Paraplegia Type 50
|
Phase 1 | Active, closed | The Hospital for Sick Children | Canada |
Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.
Authorised or ongoing trials in the EU Clinical Trials Information System that list a product containing Melpida. Many trials are registered in both CTIS and ClinicalTrials.gov, so these are shown separately rather than added to the count above. Trials with a site in Ireland are listed first.
| Study | Phase | Status | Sponsor | Countries |
|---|---|---|---|---|
|
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)
2025-524576-28-00 · Congenital, Hereditary, and Neonatal Diseases and Abnormalities
|
Phase 3 | Authorised | Elpida Therapeutics SPC | Spain |
|
A Phase IIb, Non-Profit, Open-label Trial for the Intrathecal Administration of AAV9/AP4M1 for Hereditary Spastic Paraplegia Type 50 (SPG50).
2025-524481-15-00 · CTIS ↗ · Nervous System Diseases
|
Phase 2 | Authorised | Rigshospitalet, Elpida Therapeutics SPC | Denmark |
| Lead sponsor | Trials |
|---|---|
| Elpida Therapeutics SPC | 1 |
| The Hospital for Sick Children | 1 |
| Register | Trials | Recruiting |
|---|---|---|
| Clinical trials in USA | 1 | 1 |
| Clinical trials in Canada | 1 | 0 |
| EU register (CTIS) | 2 | — |
A trial with sites in more than one country appears in each of those registers. CTIS does not publish a recruiting flag; its trials may also appear in the registers above.
2 current trials in our USA and Canada registers name Melpida as a drug or biological intervention, as of the registry sync on 26 September 2026. Completed and withdrawn studies are not counted.
Yes. 1 of the 2 trials is recruiting: 1 with sites in USA. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Elpida Therapeutics SPC (1) and The Hospital for Sick Children (1). The number in brackets is how many trials each organisation leads.
Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Melpida works or whether it is effective, and it is not medical advice.
Everything on this page is copied from public ClinicalTrials.gov registry records — it is not written from memory, and nothing here describes how Melpida works or whether it is effective. Each trial links to its own record so you can check it yourself.
This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Melpida (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 26 Sep 2026.