At a glance
ClinicalIndex Comparison Record- ✓Age 4 months to 10 years old
- ✓Confirmed homozygous or compound heterozygous pathogenic variants in AP4M1 gene
- ✓Clinical features consistent with SPG50 including neurologic dysfunction
- ✓Parent/legal guardian willing to provide written informed consent
- ✕Inability to participate in study procedures
- ✕Medical condition that precludes lumbar puncture or anesthetics
- ✕History of bleeding disorder or contraindication to lumbar puncture per institutional policy
- ✕Inability to be safely sedated per anesthesiologist
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Phase 1/2 Open-label Intrathecal Administration of MELPIDA to Determine Its Safety and Efficacy for Patients with Spastic Paraplegia Type 50 (SPG50) Caused by Mutation in the AP4M1 Gene.
In Brief
A Phase 2 clinical trial evaluating MELPIDA for Spasticity, Muscle and 5 related conditions. Currently recruiting, targeting 4 participants across 1 site.
Detailed Summary
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.
Study Details
Timeline
Interventions
MELPIDA, a recombinant serotype 9 adeno-associated virus (AAV) encoding a codon-optimized human AP4M1 transgene