Condition: Spasticity, Muscle · Microcephaly · Intellectual Deficiency · Sponsor: Elpida Therapeutics SPC
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.
This description comes directly from the study's public registry record.
Elaine Most, MS · 214-456-2463 · elaine.most@utsouthwestern.edu
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| Children's Medical Center Dallas | Dallas, Texas, United States | Recruiting |
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Source record: clinicaltrials.gov/study/NCT05518188