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Malaria drug repurposed to fight rare nerve disease

NCT ID NCT04921930

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

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

20 people

The number who actually took part.

Started

May 2022

Finished

Apr 2024

Lead sponsor

A government agency

The lead sponsor is a government body.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

16 to 65 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Patients with FA confirmed by genetic analysis * Weight of at least 50 kg * Compliant patient agreeing to come to all protocol visits * Signature of consent form by patient or parents of minor patient * Patients with no treatment during 30 days prior to the first intake of study drug, except cardiac, diabetes and spasticity treatments * Patients agreeing to use effective contraception for the duration of the study and up to 91 days after the last dose of the study treatment * Affiliation to an Health Insurance Scheme of beneficiary of such a scheme Exclusion Criteria: * Patient under justice protection * Female patients * Abnormal biological values of renal and liver functions and cell blood count (CBC) * Progressive associated disease * Treatment interfering with iron transport within 30 days before first intake of artesunate * Participation to another clinical trial * Hypersensitivity to artesunate or to any component of the drug * Blood potassium lower than normal value * QT / QTc interval \> 450 ms on the ECG performed at inclusion * Congenital long QT syndrome * Family history of sudden cardiac death before the age of 50 * Heart disease: ischemia or myocardial infarction, congestive heart failure or conduction disorder in the 6 months preceding inclusion * History of arrhythmia * Electrolyte imbalances: hypomagnesemia, hypocalcemia * Bradycardia (\<50 beats per minute) * Acute neurological events within 6 months prior to inclusion

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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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