Vatiquinone Safety for Mitochondrial Disease

Recruiting in Palo Alto (17 mi)

+31 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: PTC Therapeutics

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial is testing the safety of vatiquinone, a medication that helps cells produce energy, in people with inherited mitochondrial disease. These participants have already used vatiquinone in previous studies. The study will continue for an extended period. Vatiquinone, also known as EPI-743, is a new oral antioxidant that has been evaluated for mitochondrial diseases.

Eligibility Criteria

This trial is for people with inherited mitochondrial diseases who have previously taken vatiquinone in a study or treatment plan. Participants must not be pregnant, breastfeeding, or involved in another interventional study. Women of childbearing age and fertile men must agree to use effective contraception during the study.

Inclusion Criteria

You are a woman of childbearing potential who has a negative pregnancy test at screening/baseline and agrees to abstinence or the use of at least one of the highly effective forms of contraception as specified in the protocol (with a failure rate of <1% per year when used consistently and correctly)

I agree to use birth control during and up to 30 days after the study if I am a fertile man not having had a vasectomy.

I agree to use birth control during and up to 50 days after the study if I am a fertile man not vasectomized.

+2 more

Exclusion Criteria

Current participation in any other interventional study.

Pregnancy or breast feeding.

Participant Groups

The safety of vatiquinone is being tested in individuals with inherited mitochondrial disease who have used it before. The trial will monitor participants until vatiquinone is available commercially or the program ends.

1Treatment groups

Experimental Treatment

Group I: VatiquinoneExperimental Treatment1 Intervention

Participants will receive vatiquinone oral solution (100 milligrams \[mg\]/milliliter \[mL\]), up to 400 mg, administered orally or via feeding tube 3 times daily (TID).