New gene therapy for ALS enters first human safety trial

NCT ID NCT07401121

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage study tests a single dose of a gene therapy called CTx1000 in 15 people with ALS. The main goal is to see if it is safe and tolerable. Participants must have had ALS for 2 years or less and be on stable standard medications. This is not a cure; it aims to control the disease.

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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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Conditions

The condition(s) this trial relates to.

amyotrophic lateral sclerosis motor neuron disorder

As listed by the trial registrant

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

Contacts and locations

Locations

  • Macquarie University Hospital

    RECRUITING

    Sydney, New South Wales, 2109, Australia

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