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

Institut National de la Santé Et de la Recherche Médicale, France

Status and phase: Completed; Phase 2; Phase 1

Conditions: Friedreich Ataxia

Treatments: Drug: Artesunate Oral Product

Study type: Interventional

Funder types: Other

Identifiers

NCT04921930

C20-54

Details and patient eligibility

About

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

Enrollment: 20 patients

Sex: Male

Ages: 16 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

Artesunate

Experimental group

Description:

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)

Treatment:

Drug: Artesunate Oral Product