Centre d'Investigation Clinique, hôpital Necker Enfants Malades
Paris, France
NCT Number: NCT04921930
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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Notify Me16 year–65 year
Male
Interventional
Phase 1 / Phase 2
Paris, France
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Inclusion criteria
Exclusion criteria
Dose escalation intake of artesunate
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.
Time frame: From first intake to 30 days after last intake of study drug
Rate of side effects according artesunate doses
Time frame: From first intake to 30 days after last intake of study drug
Desciption of side effects according artesunate doses
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
Institut National de la Santé Et de la Recherche Médicale, France
Other Gov
Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA) Phase I-II Efficacy-Toxicity of Artesunate in Friedreich Ataxia
Acronym: ARTEMIS
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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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