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Gene therapy aims to halt rare childhood paralysis disorder

NCT ID NCT05518188

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

First seen Jul 08, 2026 · Last updated Jul 09, 2026 · Updated 1 time

Summary

This trial tests a single injection of MELPIDA, a gene therapy that delivers a working copy of the AP4M1 gene directly into the spinal fluid of children with SPG50. SPG50 is a rare genetic condition that causes muscle stiffness, intellectual disability, and developmental delays. The study will first check if the treatment is safe, and then look for signs that it improves muscle control and overall disease symptoms.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
MELPIDA (gene therapy using a harmless virus to deliver a working copy of the AP4M1 gene)
What this could lead to
If successful, this could provide a one-time treatment that slows or stops the progression of SPG50, a severe neurological disorder in children.
What could go wrong
This is an early-phase trial with only 4 participants, so safety and effectiveness are not yet proven. Gene therapies can cause immune reactions or other side effects.

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Conditions

The condition(s) this trial relates to.

hereditary spastic paraplegia hereditary spastic paraplegia 50 intellectual disability microcephaly Muscle Spasticity paraplegia

As listed by the trial registrant

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

GROWTH RETARDATION INTELLECTUAL DEFICIENCY MICROCEPHALY SPASTIC PARAPLEGIA SPASTICITY, MUSCLE SPG50

Contacts and locations

Study contacts

  • Contact

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

Locations

  • Children's Medical Center Dallas

    RECRUITING

    Dallas, Texas, 75235, United States

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

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