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Clinical trial

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.

NCT05518188 is registered in ClinicalTrials.gov with Elpida Therapeutics SPC as lead sponsor.

NCT NCT05518188
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Registry record

As filed in ClinicalTrials.gov.

Title
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.
Lead sponsor
Elpida Therapeutics SPC
Phase
Phase 1/2
Overall status
Recruiting
Start date
February 15, 2023
Completion date
October 1, 2030
Enrollment
4 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

growth retardation, intellectual deficiency, microcephaly, spastic paraplegia, spasticity, muscle, spg50