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Hope for kids with rare paralysis: gene therapy trial launches

NCT ID NCT06692712

Recruiting now Disease control Sponsor: Elpida Therapeutics SPC Source: ClinicalTrials.gov ↗

First seen Jan 21, 2026 · Last updated Jun 06, 2026 · Updated 17 times

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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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Sant Joan de Deu

    ACTIVE_NOT_RECRUITING

    Barcelona, 08950, Spain

  • University of Texas Southwestern Medical Center

    RECRUITING

    Dallas, Texas, 75025, United States

Conditions

The condition(s) this trial relates to.

paraplegia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HEREDITARY SPASTIC PARAPLEGIA TYPE 50 HEREDITARY SPASTIC PARAPLEGIA TYPE 50