Back to search results

MELPIDA for SPG50

This study is testing a gene therapy called MELPIDA for people with SPG50 (Spastic Paraplegia Type 50), a rare genetic disorder. MELPIDA aims to deliver a working copy of the AP4M1 gene to brain cells to help with the disease. The main goal is to see how safe MELPIDA is by tracking any side effects over 60 months. The study is looking for 4 participants, aged 4 months to 10 years, who have a confirmed diagnosis of SPG50 with specific genetic changes in the AP4M1 gene and signs of neurological problems. This trial will also explore how well MELPIDA might improve disease symptoms.

Study design
This is an interventional study planning to enroll 4 participants. It is not specified if it is randomized, blinded, or its phase.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for 60 months to assess treatment-related toxicities.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05518188

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

Recruiting
PHASE1Ages 4–10InterventionalTreatment
Elpida Therapeutics SPC
~4 participants
Updated 2026-07-08 on ClinicalTrials.gov
What's tested:MELPIDA

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of unanticipated treatment-related toxicities, Grade 3 or higher in participants with SPG50
Measured over 60 months

Conditions

Spasticity, Muscle
Microcephaly
Intellectual Deficiency
Growth Retardation
SPG50
Spastic Paraplegia

Explore related trials

X-linked spastic paraplegia type 2AP4M1 Gene

NCT05518188

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Children's Medical Center Dallas

    Dallas, Texasstudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Susan T. Iannaccone, MD, FAAN · PRINCIPAL_INVESTIGATOR · University of Texas Southwestern Medical Center

Who to contact

Elaine Most, MS
[email protected]
214-456-2463
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

What this trial measures

  • Incidence of unanticipated treatment-related toxicities, Grade 3 or higher in participants with SPG5060 months

    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.