Clinical trial NCT04921930: Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA)
Official title: Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA) Phase I-II Efficacy-Toxicity of Artesunate in Friedreich Ataxia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Friedreich Ataxia
Lead sponsor: Institut National de la Santé Et de la Recherche Médicale, France
Sponsor class: OTHER_GOV
Collaborators: name: Imagine Institute
First posted: 2021-06-10
Enrollment: 20
Sex: MALE
Minimum age (years): 16
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


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

Primary outcomes:
measure: 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)
timeFrame: at Day 7 (last day of drug intake)
Secondary outcomes:
measure: Incidence of Adverse Events with Artesunate in FA patients
timeFrame: From first intake to 30 days after last intake of study drug
measure: Type of Adverse Events with Artesunate in FA patients
timeFrame: From first intake to 30 days after last intake of study drug
measure: Impact of stopping an effective dose of artesunate on the regulation of iron homeostasis and TfR1 immunofluorescence
timeFrame: At Day 14 (7 days after the last drug intake)
Other outcomes:
Locations:
city: Paris
country: France
facility: Centre d'Investigation Clinique, hôpital Necker Enfants Malades
Study officials:
affiliation: Institut National de la Santé Et de la Recherche Médicale, France
name: Arnold Munnich, MD
role: PRINCIPAL_INVESTIGATOR
References: