Intrathecal Administration of MELPIDA (AAV9/AP4M1) For Hereditary Spastic Paraplegia Type 50 (SPG50): A Phase 3, Open-Label Trial With Matched Prospective Concurrent Control Arm
Elpida Therapeutics SPC
Summary
Phase 3, open-label study to assess the efficacy and safety of a single lumbar intrathecal administration of MELPIDA in individuals with Hereditary Spastic Paraplegia Type 50 (SPG50).
Description
MELPIDA is an AAV9-based gene therapy vector that expresses the fully functional form of AP4M1 under the control of a synthetic promoter. MELPIDA will be delivered intrathecally and is designed to achieve stable, potentially life-long expression of AP4M1 in non-dividing cells. This clinical study is a pivotal open-label phase 3 study designed to assess safety and efficacy of MELPIDA in individuals with SPG50.
Eligibility
- Age range
- 0–6 years
- Sex
- All
- Healthy volunteers
- No
Inclusion: For the treatment group * Male and females between the ages of 4 months to 72 months at the time of screening. * Molecularly-confirmed diagnosis of SPG50 (confirmed by a CLIA certified, CE-marked, or equivalent lab): Genomic DNA mutation analysis demonstrating bi-allelic pathogenic or likely pathogenic variants in the AP4M1 gene. * Subjects must have features of neurologic dysfunction by clinical history and physical examination. * Stable doses of concomitant medications such as anti-spasticity medications, anti-seizure medications, behavioral management medications, sleep medicat…
Interventions
- GeneticMELPIDA
Gene Therapy agent
Locations (2)
- University of Texas Southwestern Medical CenterDallas, Texas
- Sant Joan de DeuBarcelona