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Vatiquinone for Friedreich Ataxia
Phase 2
Waitlist Available
Research Sponsored by PTC Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
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.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 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
Awards & highlights
Study Summary
This trial will study the safety and how the body processes a new medication for young children with Friedreich ataxia.
Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
While not explicitly listed here, potential side effects may include reactions related to sesame oil (an ingredient), issues due to stopping anticoagulants or other drugs as required by the study protocol.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I can stop taking blood thinners and aspirin for 30 days before and during the study, as advised by my doctor.
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I can avoid certain medications and products like grapefruit for 30 days before and during the study.
Select...
I have been diagnosed with Friedreich ataxia through genetic testing.
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I can avoid certain medications and products like grapefruit for 30 days before and during the study.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 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
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~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
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Area Under the Curve (AUC) of Vatiquinone
Number of Participants With Adverse Events
Plasma Concentration of Vatiquinone
Trial Design
1Treatment groups
Experimental Treatment
Group I: VatiquinoneExperimental Treatment1 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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Vatiquinone
2020
Completed Phase 3
~210
Find a Location
Who is running the clinical trial?
PTC TherapeuticsLead Sponsor
74 Previous Clinical Trials
6,225 Total Patients Enrolled
3 Trials studying Friedreich Ataxia
346 Patients Enrolled for Friedreich Ataxia
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I haven't taken CoQ10, vitamin E, or any FA medication in the last 30 days.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.I can stop taking blood thinners and aspirin for 30 days before and during the study, as advised by my doctor.Your heart's pumping ability is less than 50%.I have been treated with vatiquinone before.I have been diagnosed with Friedreich ataxia through genetic testing.I can take vatiquinone oral solution with food.I can avoid certain medications and products like grapefruit for 30 days before and during the study.You are allergic to vatiquinone or sesame oil.
Research Study Groups:
This trial has the following groups:- Group 1: Vatiquinone
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Friedreich Ataxia Patient Testimony for trial: Trial Name: NCT05485987 — Phase 2
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