A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological condition called Friedreich ataxia

Phase 1

Conditions: Friedreich Ataxia (FA)
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: EUCTR2022-002668-65-DE

Lead Sponsor: PTC Therapeutics, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: A

Sex: All

Target Recruitment: 140

Inclusion Criteria

1. Subject provides consent to participate.

2. Subjects with FA who completed and directly rolled over from a previous vatiquinone clinical study.

3. Males and females of childbearing potential must be willing to use an effective method of contraception (eg, implants, injectables, transdermal patch, combined oral contraceptives, barrier methods, and intrauterine devices) from the time consent is signed until 30 days after last dose of study drug or Early Termination Visit. Male subjects must agree not to donate sperm during the study and for at least 30 days after the last dose of study drug or Early Termination Visit. Note: Double-barrier method (ie, condom with spermicide) is
required if no other methods of contraception are in use.
Are the trial subjects under 18? yes
Number of subjects for this age range: 90
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 45
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Current participation in any other interventional study

2. Pregnancy or breastfeeding

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the long-term safety of vatiquinone in subjects with FA previously exposed to vatiquinone;Secondary Objective: To assess the effects of vatiquinone on the Modified Friedreich Ataxia Rating Scale (mFARS) and its subscales in subjects with FA previously exposed to vatiquinone in Study PTC743-NEU-003-FA or Study PTC743-NEU-005-FA;Primary end point(s): Adverse events/serious adverse events (SAEs), laboratory abnormalities, vital signs, and electrocardiograms (ECGs);Timepoint(s) of evaluation of this end point: Frequencies of AEs will be tabulated by MedDRA System Organ Class, Preferred Term, severity, relationship to study drug, and seriousness.<br>Vital signs, laboratory data, and ECG will be summarized by visit. Changes from baseline will be summarized by visit, where appropriate.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Change from baseline in the mFARS and its 4 subscales (Upright Stability, Upper Limb, Lower Limb, Bulbar).;Timepoint(s) of evaluation of this end point: Modified Friedreich Ataxia Rating Scale scores and the changes from baseline will be summarized by visit based on the Safety Analysis Set.