MELPIDA for Spastic Paraplegia
(SPG50 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
Phase 3, open-label study to assess the efficacy and safety of a single lumbar intrathecal administration of MELPIDA in individuals with Hereditary Spastic Paraplegia Type 50 (SPG50).
Are You a Good Fit for This Trial?
This trial is for children aged 23 to 72 months with Hereditary Spastic Paraplegia Type 50 (SPG50). They must be able to sit independently for a short time, have confirmed SPG50 due to specific gene mutations, and show neurological dysfunction. Stable medication use and caregiver involvement are required.Inclusion Criteria
What Are the Treatments Tested in This Trial?
Interventions
- MELPIDA
Trial Overview
The study tests the safety and effectiveness of MELPIDA, given as a single lumbar intrathecal injection, in treating SPG50. It's an open-label Phase 3 trial with concurrent controls comparing treated participants against those not receiving the intervention.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Active Control
Eligible subjects (N=8) will receive a single open-label intrathecal administration of MELPIDA and follow up to week 260.
Approximately 16 untreated age- and disease- matched controls with confirmed AP-4-related disease (SPG47, SPG50, or SPG52) will be enrolled and attend study visits concurrent with the MELPIDA treatment arm.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Elpida Therapeutics SPC
Lead Sponsor
University of Texas Southwestern Medical Center
Collaborator
Hospital Sant Joan de Deu
Collaborator
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