Safety and Efficacy of Etravirine in Friedreich Ataxia Patients (FAEST1)
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ClinicalTrials.gov Identifier: NCT04273165 |
Recruitment Status :
Active, not recruiting
First Posted : February 17, 2020
Last Update Posted : March 31, 2022
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Condition or disease | Intervention/treatment | Phase |
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Friedreich Ataxia | Drug: Etravirine Tablets | Phase 2 |

Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 30 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Intervention Model Description: | dose comparison in open label pilot study |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 2 Clinical Trial to Test the Safety and Efficacy of Etravirine in Friedreich Ataxia Patients |
Actual Study Start Date : | September 17, 2020 |
Estimated Primary Completion Date : | October 31, 2022 |
Estimated Study Completion Date : | November 30, 2022 |

Arm | Intervention/treatment |
---|---|
Active Comparator: Etravirine Dose 1
Etravirine dose 200 mg per diem(100+100)
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Drug: Etravirine Tablets
Daily etravirine BID with 100 or 200 tablets for 4 months |
Active Comparator: Etravirine Dose 2
Etravirine dose 400 mg per diem (200+200)
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Drug: Etravirine Tablets
Daily etravirine BID with 100 or 200 tablets for 4 months |
- Adverse event number and severity [ Time Frame: 4 months ]description and count of AE and SAE to document the safety of etravirine treatment at 200 mg/die and 400mg/die in FA patients
- VO2 max increase [ Time Frame: 12 months ]efficacy of etravirine treatment in increasing aerobic capacity in FA patients comparing pre and post treatment periods
- Scale for the Assessment and Rating of Ataxia [ Time Frame: 12 months ]Scale for the Assessment and Rating of Ataxia (SARA) score change comparing pre-, during- and post- treatment evaluations. SARA scores ataxia on a 0-40 points scale where 0 means no ataxia and 40 most severe ataxia
- Cardiac wall thickness (in mm) [ Time Frame: 12 months ]cardiac wall thickness (in mm) as measured by standard Echo cardiography
- Frataxin measurement [ Time Frame: 12 months ]Frataxin protein content in peripheral blood mononuclear cells
- short form 36 scale for quality of life [ Time Frame: 12 months ]changes in QoL as measured by the short form 36 (SF36) scale and disability as measured with the World Health Organization Disability Assessment schedule 2.0 (WHO-DAS 2.0) during the study period. Both tools are graded on a 0-100 scale where 100 is best and 0 is worst.

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Ages Eligible for Study: | 10 Years to 40 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Molecularly defined FA diagnosis (at least one expanded FXN allele),
- ability to complete maximal exercise testing (being able to ride a recumbent exercise ergometer at 60 rpm with no added resistance for at least 3 minutes),
- willingness to participate, as shown by the signed consent form.
Exclusion Criteria:
- Known intolerance to any of the components of the drug preparation.
- Ongoing treatment with Etravirine or other experimental therapeutics under study for FA (e.g. IFNy, erythropoietin, nicotinamide, etc.).
- Previous treatment, if any, should have been stopped at least 4 weeks before study entry.
- Severe medical condition likely to interfere with drug absorbance and distribution (liver or kidney failure, severe metabolic unbalance), significant cardiac disease

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04273165
Italy | |
IRCCS Medea Scientific Institute, Pieve di Soligo Research Centre | |
Pieve Di Soligo, TV, Italy, 31953 |
Responsible Party: | IRCCS Eugenio Medea |
ClinicalTrials.gov Identifier: | NCT04273165 |
Other Study ID Numbers: |
2019-002618-38 2019-002618-38 ( EudraCT Number ) |
First Posted: | February 17, 2020 Key Record Dates |
Last Update Posted: | March 31, 2022 |
Last Verified: | March 2022 |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | No |
Ataxia Cerebellar Ataxia Friedreich Ataxia Dyskinesias Neurologic Manifestations Nervous System Diseases Cerebellar Diseases Brain Diseases Central Nervous System Diseases Spinocerebellar Degenerations Spinal Cord Diseases Heredodegenerative Disorders, Nervous System |
Neurodegenerative Diseases Genetic Diseases, Inborn Mitochondrial Diseases Metabolic Diseases Etravirine Reverse Transcriptase Inhibitors Nucleic Acid Synthesis Inhibitors Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action Antiviral Agents Anti-Infective Agents |