A study to determine if Vatiquinone, the study drug, is safe and effective to treat a neurological condition called Friedreich ataxia
- Conditions
- Friedreich Ataxia (FA)MedDRA version: 20.0Level: PTClassification code 10017374Term: Friedreich's ataxiaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2020-002812-36-DE
- Lead Sponsor
- PTC Therapeutics, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 126
1. mFARS =20 to =70 at baseline
2. Minimum age 7 years at the time of Screening Visit
3. Must be able to ambulate at least 10 feet in one minute with or without assistance (non-wheelchair)
4. Friedreich ataxia diagnosis (homozygous for guanine-adenine-adenine [GAA] repeat expansion in intron-1 of frataxin gene), confirmed by clinical genetic testing (Note: size of GAA repeat is not required for eligibility)
5. Consent to comply with study procedures. For subjects under the age of 18 (or age of consent), parent(s)/legal guardian(s) of the subject must agree to comply with the requirements of the study, including the need for frequent and prolonged follow-up; parent(s)/legal guardian(s) with custody of the subject must give their consent for subject to enroll in the study.
6. Difference in the mFARS between screening and baseline of no more than 4 points
7. Must be able to abstain from anticoagulants and any aspirin (including 81 mg) for 30 days prior to the Baseline Visit and for the duration of the study; any possible discontinuation of anticoagulants should be monitored and indicated by a specialist (eg, cardiologist, neurologist, or hematologist), and discontinuation will be noted by the prescribing physician (see Appendix 1)
8. Must be able to abstain from potent cytochrome P450 (CYP) 3A4 inducers/inhibitors (eg, ketoconazole, rifampin, St. John’s wort, grapefruit juice or any grapefruit product) for at least 30 days prior to enrollment (see Appendix 1)
9. Must be able to swallow capsules
10. Males and females of childbearing potential must be willing to use an effective method of contraception as defined in protocol Section 7.5.10 from the time consent is signed until 30 days after the 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
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 36
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Individuals with clinical diagnosis of FA who have point mutations or deletions or other non-GAA expansion mutations
2. Previous treatment with vatiquinone
3. Allergy to vatiquinone, sesame oil, gelatin (bovine and/or porcine), titanium dioxide, or red iron oxide
4. Ejection fraction <50%
5. Uncontrolled diabetes (HbA1c >7.0%) at the time of screening
6. Has current suicidal ideation based on Columbia-Suicide Severity Rating Scale (C-SSRS) within 3 months prior to screening or between screening and baseline at the Baseline Visit or suicidal behavior within the last year at the Screening Visit or between screening and baseline at the Baseline Visit
7. Pregnant or lactating subjects or those sexually active subjects who are unwilling to comply with proper birth control methods; females of childbearing potential must have a negative pregnancy test at Screening and during the Baseline Visit
8. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) =2×ULN at time of screening
9. INR =1.5×ULN at time of screening or clinically significant (CS) bleeding, as determined by the investigator
10. Serum creatinine =1.5×ULN at time of screening
11. Comorbidities that may confound study results (eg, fat malabsorption syndrome, other mitochondrial disorder) in the opinion of the investigator
12. Participation in any other interventional clinical trial or received any investigational drug in any other clinical trial within 60 days prior to the Baseline Visit. Subjects may be rescreened after the exclusionary period of 60 days has passed.
13. Concomitant use of interventional CoQ10, vitamin E, or any approved or non-approved medication for FA within 30 days prior to the Screening Visit (Appendix 1). These prohibited medications can be discontinued at the Screening Visit; if this is the case, the mFARS assessment must be repeated to confirm inclusion eligibility after a minimum of 30 days post-discontinuation and there must be no more than a 4-point difference in mFARS assessed from the Post-Discontinuation Visit to the Baseline Visit.
14. Illicit drug use 30 days prior to screening and during the study is prohibited.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method