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

• Status: Not yet recruiting
• Trial end date: 2022-06-30
• Target enrollment: 0
• Participant gender: Male

Summary

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

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Institut National de la Santé Et de la Recherche Médicale, France
• Collaborator: Imagine Institute
• Treatments: Artesunate

Criteria

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