Vatiquinone for Friedreich Ataxia

Overseen By Patient Advocacy

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: PTC Therapeutics

Must not be taking: Anticoagulants, Aspirin, CYP3A4 inducers

Disqualifiers: Previous vatiquinone, Allergy, Ejection fraction <50%, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 72 weeks

5 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial is testing a medication called vatiquinone to see how it behaves in the bodies of children under 7 years old who have a genetic disorder called Friedreich ataxia. The goal is to understand if the medication is safe and how it is processed by their bodies.

Will I have to stop taking my current medications?

You will need to stop taking anticoagulants, aspirin, and strong CYP3A4 inducers/inhibitors at least 30 days before the study and during the study. Also, you cannot use interventional CoQ10, vitamin E, or any medication for Friedreich ataxia 30 days before the study.

Are You a Good Fit for This Trial?

This trial is for children under 7 with Friedreich ataxia, confirmed by genetic testing. They must be able to take vatiquinone orally with food and avoid certain medications like anticoagulants, aspirin, and strong CYP3A4 inducers/inhibitors for specific periods before and during the study.

Inclusion Criteria

I can stop taking blood thinners and aspirin for 30 days before and during the study, as advised by my doctor.

I can avoid certain medications and products like grapefruit for 30 days before and during the study.

I can take vatiquinone oral solution with food.

Exclusion Criteria

I haven't taken CoQ10, vitamin E, or any FA medication in the last 30 days.

Your heart's pumping ability is less than 50%.

Participation in any other interventional clinical trial or receipt of any study 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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive vatiquinone orally 3 times a day for 72 weeks

72 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Vatiquinone

Trial Overview The trial is studying vatiquinone in young participants with Friedreich ataxia. It aims to understand how the body processes the drug (pharmacokinetics) and evaluate its safety when administered alongside a meal.

How Is the Trial Designed?

Group I: Vatiquinone Experimental Treatment 1 Intervention

Participants will receive an oral solution (100 milligrams \[mg\]/milliliter \[mL\]) of vatiquinone (15 mg/kilogram \[kg\] if body weight \<13 kg and 200 mg if body weight ≥13 kg) 3 times a day (TID) for 72 weeks.

Who Is Running the Clinical Trial?

PTC Therapeutics

Lead Sponsor

Trials

75

Recruited

6,300+

Dr. Matthew B. Klein

PTC Therapeutics

Chief Executive Officer since 2023

BA from the University of Pennsylvania, MD from Yale University School of Medicine, MS in Epidemiology from the University of Washington School of Public Health

Dr. Stuart W. Peltz

PTC Therapeutics

Chief Medical Officer since 2023

MD from Harvard Medical School