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Gene therapy breakthrough? new trial targets rare childhood paralysis

NCT ID NCT06692712

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

First seen Jan 21, 2026 · Last updated May 21, 2026 · Updated 13 times

Summary

This study tests a one-time gene therapy called MELPIDA for children aged 4 months to 6 years with a rare genetic disease called SPG50, which causes progressive paralysis and developmental delays. The treatment is given as a single injection into the spinal fluid. The goal is to see if it helps children gain or maintain motor skills like sitting, crawling, and walking compared to untreated children. While promising, this is not a cure—children may still need ongoing care and therapy.

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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

Explore the condition pages connected to this study.

HEREDITARY SPASTIC PARAPLEGIA TYPE 50 HEREDITARY SPASTIC PARAPLEGIA TYPE 50