A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

NCT ID: NCT05485987

Last Updated: 2025-08-22

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 5 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-10-12
• Study Completion Date: 2024-08-29

Brief Summary

The primary objective of the study is to assess the pharmacokinetics (PK) and safety of vatiquinone administered in participants with Friedreich ataxia (FA) younger than 7 years.

Conditions

Friedreich Ataxia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Vatiquinone

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.

Group Type: EXPERIMENTAL

Vatiquinone

Intervention Type: DRUG

Vatiquinone will be administered per dose and schedule specified in the arm.

Interventions

Vatiquinone

Vatiquinone will be administered per dose and schedule specified in the arm.

Intervention Type: DRUG

Other Intervention Names

PTC743; EPI-743

Eligibility Criteria

Inclusion Criteria

• Friedreich ataxia diagnosis (homozygous for GAA repeat expansion in intron-1 of FXN gene), confirmed by clinical genetic testing.
• Must be able to abstain from anticoagulants and any aspirin (including 81 mg) for 30 days before 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 strong 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 and for the duration of the study.
• Must be able to take vatiquinone oral solution with food.

Exclusion Criteria

• Previous treatment with vatiquinone.
• Allergy to vatiquinone or sesame oil.
• Ejection fraction <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.
• Concomitant use of interventional CoQ10, vitamin E, or any approved or non-approved medication for FA within 30 days prior to the Baseline Visit. These prohibited medications can be discontinued at the Screening Visit.

• Maximum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

PTC Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Countries

United States

Other Identifiers

PTC743-NEU-005-FA

Identifier Type: -

Identifier Source: org_study_id