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NCT05518188 · ClinicalTrials.gov registry record · Phase 1
Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
A Phase 1 study of Spasticity, Muscle and Microcephaly, sponsored by Elpida Therapeutics SPC.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 4
- Enrollment target
- 1
- Study location
NCT05518188: Recruiting Phase 1 study of Spasticity, Muscle and Microcephaly, sponsored by Elpida Therapeutics SPC.
NCT05518188 is a Phase 1 study of Spasticity, Muscle and Microcephaly that is actively recruiting participants, run by Elpida Therapeutics SPC. The registered enrollment target is 4 participants, below the 80-participant average among 11 other Spasticity, Muscle trials with a reported enrollment target (95% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05518188, a Phase 1 study of Spasticity, Muscle and Microcephaly, is actively recruiting participants, sponsored by Elpida Therapeutics SPC.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 4 participants
- Enrollment target
- 1
- Study location
Study 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.
Primary Outcome
Incidence of unanticipated treatment-related toxicities, Grade 3 or higher, in participants with SPG50 will be determined from the collection of occurrence and severity of serious adverse events (SAEs). Adverse events will be determined according to Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
Conditions Studied
Interventions
- BIOLOGICAL MELPIDA
Study Locations (1)
Texas
- Children's Medical Center Dallas - Dallas
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2023-02-15 |
| Est. Completion | 2030-10-01 |
| Phase | Phase 1 |
What NCT05518188 shows while recruiting
NCT05518188 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 4 participants, a relatively small participant target, below the 80-participant average among 11 other Spasticity, Muscle trials with a reported enrollment target (95% lower).
The record links to 6 conditions, with Spasticity, Muscle appearing as the primary indexed condition, and to 1 intervention - of which MELPIDA is the first listed.
NCT05518188 reports a single indexed study location in Texas.
Frequently Asked Questions
What is clinical trial NCT05518188 about?
NCT05518188 is a clinical study titled "Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)". 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...
What is the current status of trial NCT05518188?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 4 participants. The study started on 2023-02-15. Estimated completion is 2030-10-01.
What conditions does trial NCT05518188 study?
This clinical trial studies the following conditions: Spasticity, Muscle, Microcephaly, Spastic Paraplegia, SPG50, Growth Retardation.
What interventions are being tested in trial NCT05518188?
The interventions under investigation include: MELPIDA (BIOLOGICAL).
Who is sponsoring clinical trial NCT05518188?
This trial is sponsored by Elpida Therapeutics SPC, which has 4 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05518188 being conducted?
This trial has 1 study location across Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT05518188's enrollment target sits among peer trials
4 12th of 11 the lowest of 11 other Spasticity, Muscle trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Spasticity, Muscle trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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