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

Part of paid clinical trials in Dallas, Texas.

Sponsor
Elpida Therapeutics SPC
Study ID
NCT05518188
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

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

Eligibility Criteria

Sex
ALL
Age
4 Months - 10 Years
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
Feb 15, 2023
Status verified
Nov 2023
Primary completion
Oct 1, 2028
Completion
Oct 1, 2030

Study Design

Enrollment
4 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Treatment Arm
    MELPIDA, a gene therapy product

Primary Outcome Measure

Incidence of unanticipated treatment-related toxicities, Grade 3 or higher in participants with SPG50 [ Time Frame: 60 months ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Children's Medical Center DallasDallasTexas75235
SUSAN IANNACCONE, MD
[email protected]
214/456-5220

Find similar trials in Dallas, TX

By research site
Children's Medical Center Dallas· Dallas, TX

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