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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 below
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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.
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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 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
This trial's timeline: 3 weeks for screening, Varies for treatment, and 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 for reporting.

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

Vatiquinone (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05485987 — Phase 2
Friedreich Ataxia Research Study Groups: Vatiquinone
Friedreich Ataxia Clinical Trial 2023: Vatiquinone Highlights & Side Effects. Trial Name: NCT05485987 — Phase 2
Vatiquinone (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05485987 — Phase 2
Friedreich Ataxia Patient Testimony for trial: Trial Name: NCT05485987 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has Vatiquinone met the FDA's criteria for safety and efficacy?

"Vatiquinone has not been proven effective in any clinical trials, however, it did receive a score of 2."

Answered by AI

How many test subjects are involved in this experiment?

"The listed clinical trial on clinicaltrials.gov is currently looking for 5 patients from 1 centre. The study was initially posted on August 15th, 2022 and last updated on November 7th, 202202."

Answered by AI

Is this experiment looking for more test subjects at the moment?

"The clinical trial is currently recruiting, as indicated by the most recent update on November 7th, 2020. The study requires 5 participants and was first posted on August 15th, 2020."

Answered by AI

Who else is applying?

What state do they live in?
Georgia
What site did they apply to?
The Children's Hospital of Philadelphia
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
How many prior treatments have patients received?
0

Why did patients apply to this trial?

I havent tried any drug. We are willing to do and try anything to help find a cure for Friedreich's Ataxia!
PatientReceived no prior treatments
Recent research and studies
clinicaltrials.govStudy
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
2022. "A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05485987.
All > Cerebellar Ataxia
~1 spots leftby Oct 2024
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