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Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)

NCT05518188 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2024-10-08

No results posted yet for this 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.

Conditions

  • Spasticity, Muscle
  • Microcephaly
  • Intellectual Deficiency
  • Growth Retardation
  • SPG50
  • Spastic Paraplegia

Interventions

BIOLOGICAL

MELPIDA

MELPIDA, a recombinant serotype 9 adeno-associated virus (AAV) encoding a codon-optimized human AP4M1 transgene

Sponsors & Collaborators

  • University of Texas Southwestern Medical Center

    collaborator OTHER

  • Elpida Therapeutics SPC

    lead INDUSTRY

Principal Investigators

  • Susan T. Iannaccone, MD, FAAN · UT Southwestern Medical Center

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Months
Max Age
10 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-02-15
Primary Completion
2028-10-01
Completion
2030-10-01
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05518188 on ClinicalTrials.gov