Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 T… (NCT05518188) | Clinical Trial Compass
RecruitingPhase 1/2
Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
United States4 participantsStarted 2023-02-15
Plain-language 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.
Who can participate
Age range4 Months – 10 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Exclusion criteria
✕. Inability to participate in study procedures (as determined by the site investigator)
✕
What they're measuring
1
Incidence of unanticipated treatment-related toxicities, Grade 3 or higher in participants with SPG50
. 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