A Phase 1/2 interventional study of Artesunate Oral Product in Friedreich Ataxia, sponsored by Institut National de la Santé Et de la Recherche Médicale, France. Completed at 1 site in France. Open to male participants aged 16 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-05-20.
Sponsored by Institut National de la Santé Et de la Recherche Médicale, France · Phase 1/2, Interventional, and Treatment
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
107 studies on the registry are indexed under Friedreich Ataxia; 25 are open to participants now.
This study's enrollment of 20 is below the median of 30 across 74 interventional studies indexed under Friedreich Ataxia.
Browse Friedreich Ataxia studies →Institut National de la Santé Et de la Recherche Médicale, France is the lead sponsor of 375 studies on the registry; 82 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Dose escalation of oral artesunate: Step 1: 25 mg daily (1 tablet) during one week Step 2: 50 mg daily (2 tablets) during one week (if no effect on biomarker and no adverse reaction at step 1) Step 3: 75 mg daily (3 tablets) during one week (if no effect on biomarker and no adverse reaction at step 2) Step 4: 100 mg daily (4 tablets) duing one week (if no efficacy and no adverse reaction at step 3)
Drug: Artesunate Oral Product
Dose escalation intake of artesunate
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)
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.
Time frame: at Day 7 (last day of drug intake)
Incidence of Adverse Events with Artesunate in FA patients
Rate of side effects according artesunate doses
Time frame: From first intake to 30 days after last intake of study drug
Type of Adverse Events with Artesunate in FA patients
Desciption of side effects according artesunate doses
Time frame: From first intake to 30 days after last intake of study drug
Impact of stopping an effective dose of artesunate on the regulation of iron homeostasis and TfR1 immunofluorescence
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
Time frame: At Day 14 (7 days after the last drug intake)
This study is completed, as verified in Jul 2024. You cannot join it, but the record below documents what was studied.
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Institut National de la Santé Et de la Recherche Médicale, France