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RecruitingPhase 1/Phase 2

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

Elpida Therapeutics SPC · with University of Texas Southwestern Medical Center · NCT05518188

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 4 mo–10 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 1, Phase 2
Target enrollment
4
US sites recruiting
1
Study start
February 15, 2023
Record last updated
July 8, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: MELPIDA

Conditions: Spasticity, Muscle; Microcephaly; Intellectual Deficiency; Growth Retardation; SPG50; Spastic Paraplegia

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

Inclusion Criteria

  • Age 4 months-10 years old
  • Confirmed diagnosis of SPG50 disease by:
  • Genomic DNA mutation analysis demonstrating homozygous or compound heterozygous, confirmed pathogenic variants in the AP4M1 gene
  • Clinical history or examination features consistent with SPG50 and that include neurologic dysfunction
  • Parent/legal guardian willing to provide written informed consent for their child prior to participation in the study
  • Subject able to comply with all protocol requirements and procedures
  • Ability to stand for more than 5 seconds OR
  • Ability to take 5 steps independently or with a walker OR
  • Modified Ashworth Scale score 2 or below (Ankles).

Exclusion Criteria

  • Inability to participate in study procedures (as determined by the site investigator)
  • Presence of a concomitant medical condition that precludes lumbar puncture (LP) or use of anesthetics
  • History of bleeding disorder or any other medical condition or circumstance in which lumbar puncture is contraindicated according to local institutional policy
  • Inability to be safely sedated in the opinion of the clinical anesthesiologist
  • Active infection, at the time of dosing, based on clinical observations
  • Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer
  • Inability of the patient to undergo MRI according to local institutional policy
  • Inability of the patient to undergo any other procedure required in this study
  • The presence of significant non-SPG50 related CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study
  • Have received an investigational drug within 30 days prior to screening or plan to receive an investigational drug (other than gene therapy) during the study.
  • Enrollment and participation in another interventional clinical trial
  • Contraindication to MELPIDA or any of its ingredients
  • Contraindication to any of the immune suppression medications used in this study
  • Clinically significant abnormal laboratory values (GGT, ALT, and AST, or total bilirubin \> 3 × ULN, creatinine ≥ 1.5 mg/dL, hemoglobin [Hgb] \< 6 or \> 20 g/dL; white blood cell [WBC] \> 20,000 per cmm) prior to gene replacement therapy.

Recruiting US locations

  • Children's Medical Center Dallas

    Dallas, TX 75235

    Site contact: SUSAN IANNACCONE, MD · 214/456-5220 · SUSAN.IANNACCONE@UTSouthwestern.edu

Study contacts (from the official record)

  • Elaine Most, MS · 214-456-2463 · elaine.most@utsouthwestern.edu
All phase 1 studies in Texas →
Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding… (NCT05518188) | Bored Studies