Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA) Phase I-II Efficacy-Toxicity of Artesunate in Friedreich Ataxia
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- 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)
研究概览
简要总结
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
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
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)
干预措施: Artesunate Oral Product (Drug)
结局指标
主要结局
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)
时间窗: at Day 7 (last day of drug intake)
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.
次要结局
- 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))