Friedreich Ataxia Clinical Trial
— FAEST1Official title:
A Phase 2 Clinical Trial to Test the Safety and Efficacy of Etravirine in Friedreich Ataxia Patients
Verified date | March 2023 |
Source | IRCCS Eugenio Medea |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
A drug repositioning effort provided evidence supporting the possible use of Etravirine, a drug approved for the treatment of HIV infections in patients starting from 2 years of age, as a treatment for FA. We found that Etravirine is able to increase Frataxin protein both in vitro - in cells derived from FA patients - and in vivo - in the heart and skeletal muscle of Frataxin-deficient YG8 mice. Because of these findings, and since Etravirine displays a generally favorable safety profile, we plan to launch an open-label, phase 2 clinical trial aimed at assessing the safety and efficacy of Etravirine in FA patients. We aim at recruiting 30 FA patients. 15 will be treated with Etravirine for 4 months at 200 mcg/day and 15 will be treated with Etravirine for 4 months at 400 mg/day. Efficacy primary endpoint will be represented changes in peak VO2 as measured by incremental cycle ergometer exercise test. Secondary endpoints will include maximal workload, SARA score, cardiac measures, Frataxin protein levels in peripheral blood mononuclear cells and molecular analysis of Frataxin mRNA translation efficiency. Complete sets of data will be collected 4 months before the start of the treatment (T -4), at the start (T0), after 2 months (T2), at the end of the treatment (T4) and 4 months after the termination of the treatment (T8).
Status | Completed |
Enrollment | 30 |
Est. completion date | January 17, 2023 |
Est. primary completion date | January 17, 2023 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 10 Years to 40 Years |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
Italy | IRCCS Medea Scientific Institute, Pieve di Soligo Research Centre | Pieve Di Soligo | TV |
Lead Sponsor | Collaborator |
---|---|
IRCCS Eugenio Medea | University of Rome Tor Vergata |
Italy,
Alfedi G, Luffarelli R, Condo I, Pedini G, Mannucci L, Massaro DS, Benini M, Toschi N, Alaimo G, Panarello L, Pacini L, Fortuni S, Serio D, Malisan F, Testi R, Rufini A. Drug repositioning screening identifies etravirine as a potential therapeutic for friedreich's ataxia. Mov Disord. 2019 Mar;34(3):323-334. doi: 10.1002/mds.27604. Epub 2019 Jan 9. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Scale for the Assessment and Rating of Ataxia | 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 | 12 months | |
Other | Cardiac wall thickness (in mm) | cardiac wall thickness (in mm) as measured by standard Echo cardiography | 12 months | |
Other | Frataxin measurement | Frataxin protein content in peripheral blood mononuclear cells | 12 months | |
Other | short form 36 scale for quality of life | 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. | 12 months | |
Primary | Adverse event number and severity | description and count of AE and SAE to document the safety of etravirine treatment at 200 mg/die and 400mg/die in FA patients | 4 months | |
Secondary | VO2 max increase | efficacy of etravirine treatment in increasing aerobic capacity in FA patients comparing pre and post treatment periods | 12 months |
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