Clinical trial - phase II - to test safety and efficay of Etravirine's treatment in Friedreich

Phase 1

• Conditions: Friedreich's ataxia; MedDRA version: 20.0Level: PTClassification code 10017374Term: Friedreich's ataxiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2019-002618-38-IT
• Lead Sponsor: ASSOCIAZIONE LA NOSTRA FAMIGLIA - SEZIONE SCIENTIFICA I.R.C.C.S. E.MEDEA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-06-01
• Last Update Posted: 12 March 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 35

Inclusion Criteria

• FRDA molecularly defined (expansion in at least one of the alleles of the FXN gene). • Absence of HIV infection (anti-HIV research negativity) • Ability to complete the cycle ergometer test with legs or arms to measure maximum exercise (the patient must be able to pedal at least 60 rpm without additional resistance for at least 3 minutes). • Willingness to participate in the study and sign the informed consent form. • Age range 10-40 years
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 18
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

• Pregnancy or breastfeeding in place. • Known sensitivity to the ETR or to one of its excipients. • Exposure to ETR or other experimental therapies used in FRDA (eg IFNy, erythropoietin, nicotinamide, idebenone, deferiprone, etc.). • Any previous treatments with ETR should have been interrupted at least 4 months before entering the study. • Serious medical conditions that can interfere with the absorption and distribution of the drug (liver or kidney failure, severe metabolic imbalance), major heart disease (ejection fraction <40%). • Ongoing treatment with carbamazepine, phenobarbital, phenytoin, rifampicin, rifapentine, clopidogrel, hypericum. • Positivity to the HIV test

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified