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Completed

NCT Number: NCT04921930

Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA)

This dose-escalation study is aimed at investigating a novel application for artesunate in the treatment of Friedreich ataxia. It will evaluate this novel application of oral artesunate using a surrogate biological marker as primary endpoint in a phase I-II open trial

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Key information

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Artesunate Oral Product

Drug

Dose escalation intake of artesunate

Primary outcomes

  1. Search for the maximal tolerated and effective dose of oral artesunate to regulate iron homeostasis and Transferrin 1 receptor (TfR1) immunofluorescence in Peripheral Blood Mononuclear Cells (PBMCs)

    Time frame: at Day 7 (last day of drug intake)

    Evaluation of the biological efficacy on an ex vivo marker in the absence of observed side effects. This is a binary criterion established by comparison between compared measurements of the biomarker. If an effect on the biomarker is observed from the initial dose, the dose escalation will stop as the "ex-vivo" efficacy criterion is met. Otherwise the test will be repeated at an escalating dose. If an adverse effect is observed for a given dose, the maximal tolerated dose will be considered to be the immediately lower dose.

Secondary outcomes

  1. Incidence of Adverse Events with Artesunate in FA patients

    Time frame: From first intake to 30 days after last intake of study drug

    Rate of side effects according artesunate doses

  2. Type of Adverse Events with Artesunate in FA patients

    Time frame: From first intake to 30 days after last intake of study drug

    Desciption of side effects according artesunate doses

  3. Impact of stopping an effective dose of artesunate on the regulation of iron homeostasis and TfR1 immunofluorescence

    Time frame: At Day 14 (7 days after the last drug intake)

    Evolution of the response to treatment after one week without treatment in patients who presented a positive response: Comparison of the results of intracellular iron concentration in in vitro PBMCs obtained with the sample at the end of the week without treatment with those obtained at the end of the week under treatment at a given dose

Sponsors and collaborators

Lead sponsor

Institut National de la Santé Et de la Recherche Médicale, France

Other Gov

Collaborators

  • Imagine Institute

Registry information

Official study title

Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA) Phase I-II Efficacy-Toxicity of Artesunate in Friedreich Ataxia

Acronym: ARTEMIS

Important dates

Study start
2022
Primary completion
2024
Study completion
2024
First posted
Jun 10, 2021
Registry last updated
May 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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