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
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Dose escalation intake of artesunate
Centre d'Investigation Clinique, hôpital Necker Enfants Malades
Paris, France
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)
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