Overview

A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

Status:
Recruiting
Trial end date:
2023-07-06
Target enrollment:
0
Participant gender:
All
Summary
The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale [mFARS]) and safety of vatiquinone in participants with Friedreich ataxia (FA).
Phase:
Phase 2/Phase 3
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
PTC Therapeutics
Criteria
Inclusion Criteria:

- mFARS ≥20 to ≤70 at baseline

- Must be able to ambulate at least 10 feet in 1 minute with or without assistance
(non-wheelchair)

- Friedreich ataxia diagnosis (homozygous for guanine-adenine-adenine [GAA] repeat
expansion in intron-1 of frataxin [FXN] gene), confirmed by clinical testing (Note:
size of GAA repeat is not required for eligibility)

- Consent to comply with study procedures. For participants under the age of 18 (or age
of consent), parent(s)/legal guardian(s) of the participant 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 participant must give their
consent for participant to enroll in the study.

- Difference in the mFARS at screening and baseline of no more than 4 points.

- 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
(for example, cardiologist, neurologist, or hematologist) and discontinuation will be
noted by the prescribing physician.

- Must be able to abstain from potent cytochrome P450 (CYP) 3A4 inducers/inhibitors (for
example, ketoconazole, rifampin, St. John's wort, grapefruit juice or any grapefruit
product) for at least 30 days prior to enrollment

- Must be able to swallow capsules

- Males and females of childbearing potential must be willing to use an effective method
of contraception from the time consent is signed until 30 days after the last dose of
study drug or early termination visit. Male participants 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.

Exclusion Criteria:

- Individuals with clinical diagnosis of FA who have point mutations or deletions or
other non-GAA expansion mutations

- Previous treatment with vatiquinone

- Allergy to vatiquinone, sesame oil, gelatin (bovine and/or porcine), titanium dioxide,
or red iron oxide

- Ejection fraction <50%

- Uncontrolled diabetes (glycated hemoglobin [HbA1c] >7.0%) at the time of screening

- 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

- Pregnant or lactating participants or those sexually active participants 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

- Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≥2 * upper limit of
normal (ULN) at time of screening

- International normalized ratio (INR) ≥1.5 * ULN at time of screening or clinically
significant (CS) bleeding, as determined by the investigator

- Serum creatinine ≥1.5 * ULN at time of screening

- Comorbidities that may confound study results (for example, fat malabsorption
syndrome, other mitochondrial disorder) in the opinion of the investigator

- 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. Participants may be rescreened after the exclusionary period of 60 days has
passed.

- Concomitant use of interventional coenzyme Q10 (CoQ10), vitamin E, or any approved or
non-approved medication for FA within 30 days prior to the screening visit. 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.

- Illicit drug use 30 days prior to screening and during the study is prohibited.