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

C20-54

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

2022-05-06

2023-12-05

2023-12-05

Recruiting

Early phase I

20

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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 20, 'type': 'ESTIMATED'}}

2023-07-12