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

NCT04921930 | Completed Phase 1 DMC

• 2 other identifiers: C20-542021-000171-36
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (1)

• 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.

  at Day 7 (last day of drug intake)

Secondary Outcomes (3)

• Incidence of Adverse Events with Artesunate in FA patients

  From first intake to 30 days after last intake of study drug

• Type of Adverse Events with Artesunate in FA patients

  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

  At Day 14 (7 days after the last drug intake)

Study Arms (1)

Artesunate

EXPERIMENTAL

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)

Drug: Artesunate Oral Product

Interventions

Artesunate Oral Product DRUG

Dose escalation intake of artesunate

Artesunate

Eligibility Criteria

• Age: 16 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• Congenital long QT syndrome
• Family history of sudden cardiac death before the age of 50
• History of arrhythmia
• Electrolyte imbalances: hypomagnesemia, hypocalcemia
• Bradycardia (\<50 beats per minute)

Sponsors & Collaborators

• Institut National de la Santé Et de la Recherche Médicale, France: lead
• Imagine Institute: collaborator

Study Sites (1)

Centre d'Investigation Clinique, hôpital Necker Enfants Malades

Paris, France

Location

MeSH Terms

Conditions

Friedreich Ataxia

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Arnold Munnich, MD

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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 31, 2021
• First Posted: June 10, 2021
• Study Start: May 6, 2022
• Primary Completion: April 17, 2024
• Study Completion: April 17, 2024
• Last Updated: May 20, 2026

Record last verified: 2024-07

Locations

FR (1)