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

NCT05485987 | Completed Phase 2 FDA Drug

• 1 other identifier: PTC743-NEU-005-FA
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (3)

• Plasma Concentration of Vatiquinone

  Pre-morning dose (0 hour) at Week 4; 1 to 3 hours and 3 to 6 hours post-morning dose at Weeks 4, 12, and 24

• Area Under the Curve (AUC) of Vatiquinone

  Pre-morning dose (0 hour) at Week 4; 1 to 3 hours and 3 to 6 hours post-morning dose at Weeks 4, 12, and 24

• Number of Participants With Adverse Events

  Baseline up to Week 76

Study Arms (1)

Vatiquinone

EXPERIMENTAL

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.

Drug: Vatiquinone

Interventions

Vatiquinone DRUG

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

Also known as: PTC743, EPI-743

Vatiquinone

Eligibility Criteria

• Age: Up to 6 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• Previous treatment with vatiquinone.
• Allergy to vatiquinone or sesame oil.
• Ejection fraction \<50%.
• 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.

Sponsors & Collaborators

• PTC Therapeutics: lead

Study Sites (1)

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

alpha-tocotrienol quinone

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 1, 2022
• First Posted: August 3, 2022
• Study Start: October 12, 2022
• Primary Completion: August 29, 2024
• Study Completion: August 29, 2024
• Last Updated: August 22, 2025

Record last verified: 2025-08

Locations

US (1)