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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT05485987
Other study ID # PTC743-NEU-005-FA
Secondary ID
Status Active, not recruiting
Phase Phase 2
First received
Last updated
Start date October 13, 2022
Est. completion date October 5, 2024

Study information

Verified date May 2024
Source PTC Therapeutics
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 5
Est. completion date October 5, 2024
Est. primary completion date October 5, 2024
Accepts healthy volunteers No
Gender All
Age group N/A to 6 Years
Eligibility 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. Note: Other inclusion and exclusion criteria may apply.

Study Design


Related Conditions & MeSH terms


Intervention

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

Locations

Country Name City State
United States The Children's Hospital of Philadelphia Philadelphia Pennsylvania

Sponsors (1)

Lead Sponsor Collaborator
PTC Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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
Primary 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
Primary Number of Participants With Adverse Events Baseline up to Week 76
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