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

Last updated: July 29, 2024
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France
Overall Status: Completed

Phase

1/2

Condition

Friedreich's Ataxia

Dyskinesias

Spinocerebellar Disorders

Treatment

Artesunate Oral Product

Clinical Study ID

NCT04921930
C20-54
  • Ages 16-65
  • Male

Study 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

Eligibility Criteria

Inclusion

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 andup to 91 days after the last dose of the study treatment

  • Affiliation to an Health Insurance Scheme of beneficiary of such a scheme

Exclusion

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 ofartesunate

  • 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 orconduction 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

Study Design

Total Participants: 20
Treatment Group(s): 1
Primary Treatment: Artesunate Oral Product
Phase: 1/2
Study Start date:
May 06, 2022
Estimated Completion Date:
April 17, 2024

Connect with a study center

  • Centre d'Investigation Clinique, hôpital Necker Enfants Malades

    Paris,
    France

    Site Not Available

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