NCT05518188

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

Recruiting now Phase 1, PHASE2 Last updated 8 October 2024

What this trial tests

Phase 1, PHASE2 trial testing MELPIDA in Spasticity, Muscle in 4 participants. Currently enrolling.

Timeline

15 February 2023

Primary endpoint
1 October 2028

1 October 2030

Quick facts

Lead sponsor	Elpida Therapeutics SPC
Phase	Phase 1, PHASE2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	4
Start date	15 February 2023
Primary completion	1 October 2028
Estimated completion	1 October 2030
Sites	1 location across United States

Drugs / interventions tested

• MELPIDA — full drug profile →

Conditions studied

• Spasticity, Muscle — all drugs for Spasticity, Muscle →
• Microcephaly — all drugs for Microcephaly →
• Intellectual Deficiency — all drugs for Intellectual Deficiency →
• Growth Retardation — all drugs for Growth Retardation →

Sponsor

Elpida Therapeutics SPC — full company profile →

Who can join

Adults 4 Months to 10, any sex, with Spasticity, Muscle or Microcephaly. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies.
   Chen X, Dong T, Hu Y, De Pace R, et al · · 2023 · cited 32× · PMID 36951961 · DOI 10.1172/jci164575
2. AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patient.
   Dowling JJ, Pirovolakis T, Devakandan K, Stosic A, et al · · 2024 · cited 21× · PMID 38942994 · DOI 10.1038/s41591-024-03078-4
3. Recombinant Adeno-Associated Virus Vectors for Gene Therapy of the Central Nervous System: Delivery Routes and Clinical Aspects.
   Słyk Ż, Stachowiak N, Małecki M. · · 2024 · cited 14× · PMID 39062095 · DOI 10.3390/biomedicines12071523
4. Expression and distribution of rAAV9 intrathecally administered in juvenile to adolescent mice.
   Garza IT, Eller MM, Holmes SK, Schackmuth MK, et al · · 2025 · cited 7× · PMID 39501094 · DOI 10.1038/s41434-024-00498-2
5. Current trends in gene therapy to treat inherited disorders of the brain.
   Matuszek Z, Brown BL, Yrigollen CM, Keiser MS, et al · · 2025 · cited 6× · PMID 40181540 · DOI 10.1016/j.ymthe.2025.03.057
6. Emerging therapies for childhood-onset movement disorders.
   Vogt L, Quiroz V, Ebrahimi-Fakhari D. · · 2024 · cited 6× · PMID 38655812 · DOI 10.1097/mop.0000000000001354
7. Restoring adapter protein complex 4 function with small molecules: an in silico approach to spastic paraplegia 50.
   Francisco S, Lamacchia L, Turco A, Ermondi G, et al · · 2025 · cited 5× · PMID 39723768 · DOI 10.1002/pro.70006
8. Patient-Relevant Digital-Motor Outcomes for Clinical Trials in Hereditary Spastic Paraplegia Type 7: A Multicenter PROSPAX Study.
   Beichert L, Seemann J, Kessler C, Traschütz A, et al · · 2024 · cited 5× · PMID 39621946 · DOI 10.1212/wnl.0000000000209887