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

NCT ID: NCT04921930

Last Updated: 2024-07-30

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-05-06
• Study Completion Date: 2024-04-17

Brief 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

Conditions

Friedreich Ataxia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Artesunate

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)

Group Type: EXPERIMENTAL

Artesunate Oral Product

Intervention Type: DRUG

Dose escalation intake of artesunate

Interventions

Artesunate Oral Product

Dose escalation intake of artesunate

Intervention Type: DRUG

Eligibility 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

• Minimum Eligible Age: 16 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Imagine Institute

OTHER

Sponsor Role: collaborator

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

OTHER_GOV

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Arnold Munnich, MD

Role: PRINCIPAL_INVESTIGATOR

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

Locations

Centre d'Investigation Clinique, hôpital Necker Enfants Malades

Paris, , France

Countries

France

Other Identifiers

C20-54

Identifier Type: -

Identifier Source: org_study_id