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| ID | Type | Description | Link |
|---|---|---|---|
| 2021-000171-36 | EudraCT Number |
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| Name | Class |
|---|---|
| Imagine Institute | OTHER |
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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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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) |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Artesunate Oral Product | Drug | Dose escalation intake of artesunate |
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| Measure | Description | Time Frame |
|---|---|---|
| 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) |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Adverse Events with Artesunate in FA patients | Rate of side effects according artesunate doses | From first intake to 30 days after last intake of study drug |
| Type of Adverse Events with Artesunate in FA patients |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Arnold Munnich, MD | Institut National de la Santé Et de la Recherche Médicale, France | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Centre d'Investigation Clinique, hôpital Necker Enfants Malades | Paris | France |
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| ID | Term |
|---|---|
| D005621 | Friedreich Ataxia |
| ID | Term |
|---|---|
| D013132 | Spinocerebellar Degenerations |
| D002526 | Cerebellar Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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Desciption of side effects according artesunate doses
| 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 | Evolution of the response to treatment after one week without treatment in patients who presented a positive response: Comparison of the results of intracellular iron concentration in in vitro PBMCs obtained with the sample at the end of the week without treatment with those obtained at the end of the week under treatment at a given dose | At Day 14 (7 days after the last drug intake) |
| D009422 | Nervous System Diseases |
| D013118 | Spinal Cord Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D028361 | Mitochondrial Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |