Friedreich AtaxiaPhase 1/2ARTEMISNCT04921930
Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA)
A Phase 1/2 study testing Artesunate Oral Product in people with Friedreich Ataxia, run by Institut National de la Santé Et de la Recherche Médicale, France at 1 site in France.
Trial 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 Read more about this study
- Who is it for?
- Ages 16 to 65 years, men only
- Conditions the study is for
- Friedreich Ataxia
- What is being tested?
- Artesunate Oral Product
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteFrance
- Study dates
- 6 May 2022to 17 Apr 2024
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 16Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
France1 site
Centre d'Investigation Clinique, hôpital Necker Enfants Malades
Paris
About this study
Official title: Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA) Phase I-II Efficacy-Toxicity of Artesunate in Friedreich Ataxia
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
- Being tested
- Artesunate Oral Product
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Artesunate Oral ProductDrug
Dose escalation intake of artesunate
| Study group | Role | What they receive |
|---|---|---|
| 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) Receives: Drug: Artesunate Oral Product |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: at Day 7 (last day of drug intake)
Secondary outcomes (3)
Incidence of Adverse Events with Artesunate in FA patients
Rate of side effects according artesunate doses
Measured: From first intake to 30 days after last intake of study drug
Type of Adverse Events with Artesunate in FA patients
Desciption of side effects according artesunate doses
Measured: 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
Measured: At Day 14 (7 days after the last drug intake)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2021
- Study start6 May 2022
- Primary completion17 Apr 2024
- Study completion17 Apr 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich AtaxiaStudies of the same condition, open and under way.
- More trials run by Institut National de la Santé Et de la Recherche Médicale, FranceThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Sponsor type
- Other government
- Collaborators
- Imagine Institute
- Responsible party
- Sponsor
- Investigators
- Arnold Munnich, MD, Principal investigator, Institut National de la Santé Et de la Recherche Médicale, France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04921930
- Sponsor's protocol ID
- C20-54
- Other IDs
- 2021-000171-36
- Acronym
- ARTEMIS
- Status last verified
- Jul 2024
- First submitted
- 31 May 2021
- Last updated
- 20 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA) Phase I-II Efficacy-Toxicity of Artesunate in Friedreich Ataxia
- ClinicalTrials.gov ID
- NCT04921930
- Acronym / sponsor ID
- ARTEMIS / C20-54
- Conditions
- Friedreich Ataxia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Institut National de la Santé Et de la Recherche Médicale, FranceOther government
- Collaborators
- Imagine Institute
- Study start
- 6 May 2022 (actual)
- Primary completion
- 17 Apr 2024 (actual)
- Study completion
- 17 Apr 2024 (actual)
- First posted
- 10 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Artesunate | Experimental | Drug: Artesunate Oral ProductDose 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) |
Who may qualify
- Ages
- 16 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (16 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Artesunate Oral ProductDose escalation intake of artesunate | Drug | Artesunate |
Primary outcome
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)
Time frame: 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.
3 secondary outcomes
Incidence of Adverse Events with Artesunate in FA patients
Time frame: From first intake to 30 days after last intake of study drug
Type of Adverse Events with Artesunate in FA patients
Time frame: 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
Time frame: At Day 14 (7 days after the last drug intake)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Arnold Munnich, MD principal investigator
Institut National de la Santé Et de la Recherche Médicale, France
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Artesunate Oral Product in people with Friedreich Ataxia, run by Institut National de la Santé Et de la Recherche Médicale, France at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome 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) (time frame: at Day 7 (last day of drug intake)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 May 2026.