Hope for kids with rare paralysis: gene therapy trial launches
NCT ID NCT06692712
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 3 trial tests a one-time gene therapy called MELPIDA for children with SPG50, a rare genetic disease that causes progressive paralysis and developmental delays. The study will give the treatment via a spinal injection to 24 children aged 4 months to 6 years and compare their motor skills to untreated children. The goal is to see if MELPIDA can help them achieve milestones like sitting, crawling, and walking.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Email: •••••@•••••
Locations
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Sant Joan de Deu
ACTIVE_NOT_RECRUITINGBarcelona, 08950, Spain
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University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75025, United States