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Clinical trial

A Phase 1 Open-label Intrathecal Administration of MELPIDA to Determine the Safety and Efficacy for Patients With Spastic Paraplegia Type 50 (SPG50) Caused by a Mutation in the AP4M1 Gene

NCT06069687 is registered in ClinicalTrials.gov with The Hospital for Sick Children as lead sponsor.

NCT NCT06069687
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 1 Open-label Intrathecal Administration of MELPIDA to Determine the Safety and Efficacy for Patients With Spastic Paraplegia Type 50 (SPG50) Caused by a Mutation in the AP4M1 Gene
Lead sponsor
The Hospital for Sick Children
Phase
Phase 1
Overall status
Active, not recruiting
Start date
March 11, 2022
Completion date
March 31, 2027
Enrollment
1 (actual)
Sites
1

Conditions

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

spastic paraplegia type 50