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Quinone Analog

Vatiquinone Safety for Mitochondrial Disease

Phase 3
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline (day 1) up to end of study (up to approximately 3 years)
Awards & highlights

Study Summary

This trial is testing the safety of a drug for people with mitochondrial disease who have already taken the drug. The study will continue until the drug is available to the public or the program is stopped.

Who is the study for?
This trial is for people with inherited mitochondrial diseases who have previously taken vatiquinone in a study or treatment plan. Participants must not be pregnant, breastfeeding, or involved in another interventional study. Women of childbearing age and fertile men must agree to use effective contraception during the study.Check my eligibility
What is being tested?
The safety of vatiquinone is being tested in individuals with inherited mitochondrial disease who have used it before. The trial will monitor participants until vatiquinone is available commercially or the program ends.See study design
What are the potential side effects?
Specific side effects are not listed here, but as this is a safety study for vatiquinone, all potential side effects experienced by participants will be closely monitored and recorded throughout the duration of the trial.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline (day 1) up to end of study (up to approximately 3 years)
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline (day 1) up to end of study (up to approximately 3 years) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Participants With Adverse Events (AEs)

Trial Design

1Treatment groups
Experimental Treatment
Group I: VatiquinoneExperimental Treatment1 Intervention
Participants will receive vatiquinone oral solution (100 milligrams [mg]/milliliter [mL]), up to 400 mg, administered orally or via feeding tube 3 times daily (TID).
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
6,130 Total Patients Enrolled
Vinay Penematsa, MDStudy DirectorPTC Therapeutics
3 Previous Clinical Trials
690 Total Patients Enrolled

Media Library

Vatiquinone (Quinone Analog) Clinical Trial Eligibility Overview. Trial Name: NCT05218655 — Phase 3
Mitochondrial Disease Research Study Groups: Vatiquinone
Mitochondrial Disease Clinical Trial 2023: Vatiquinone Highlights & Side Effects. Trial Name: NCT05218655 — Phase 3
Vatiquinone (Quinone Analog) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05218655 — Phase 3

Frequently Asked Questions

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

Can people with the target condition participate in this clinical trial at this time?

"The clinical trial in question is no longer actively recruiting patients, as noted by the study's last update on October 21st, 2022. However, it is worth mentioning that there are 19 other trials currently enrolling individuals."

Answered by AI

Has Vatiquinone been given the okay by the FDA?

"Vatiquinone is classified as a 3 on Power's safety scale. This means that, while Phase 3 trials have yet to confirm efficacy, there is some data supporting Vatiquinone's safety from previous clinical studies."

Answered by AI

Is this trial widely available in city?

"In total, 18 hospitals are running this trial. Some of the notable locations include Nemours Children's Health in Jacksonville, Cleveland Clinic in Cleveland, and Children's National in Washington."

Answered by AI

Who else is applying?

How old are they?
< 18
What site did they apply to?
Medical University of South Carolina - MUSC
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
2022. "A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05218655.
All > Mitochondrial Disease
~33 spots leftby Mar 2025
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