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Malaria drug repurposed to fight rare nerve disease

NCT ID NCT04921930

Completed Disease control Sponsor: Institut National de la Santé Et de la Recherche Médicale, France Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a malaria drug called artesunate in 20 people with Friedreich ataxia, a rare genetic disorder that affects movement and coordination. The goal was to find a safe dose that could help control iron levels in cells, which may slow the disease. Researchers started with a low dose and increased it until they saw a biological effect or side effects appeared.

What this could mean

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

Active substance
artesunate (an oral malaria drug repurposed for Friedreich ataxia)
What this could lead to
If it works, this could point toward a treatment that helps manage Friedreich ataxia by improving iron balance in cells.
What could go wrong
This is a very early, small trial (20 people) testing a repurposed drug. It may not show clear benefit, and side effects are possible at higher doses.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Friedreich ataxia

As listed by the trial registrant

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

FRIEDREICH ATAXIA

Contacts and locations

Locations

  • Centre d'Investigation Clinique, hôpital Necker Enfants Malades

    Paris, France

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