Vatiquinone for Friedreich Ataxia

Phase-Based Progress Estimates
The Children's Hospital of Philadelphia, Philadelphia, PA
Friedreich Ataxia+2 More
Vatiquinone - Drug
< 18
All Sexes
What conditions do you have?

Study 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.

Eligible Conditions

  • Friedreich Ataxia

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Friedreich Ataxia

Study Objectives

3 Primary · 0 Secondary · Reporting Duration: 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

Week 76
Number of Participants With Adverse Events
Week 4
Area Under the Curve (AUC) of Vatiquinone
Plasma Concentration of Vatiquinone

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Friedreich Ataxia

Trial Design

1 Treatment Group

1 of 1
Experimental Treatment

5 Total Participants · 1 Treatment Group

Primary Treatment: Vatiquinone · No Placebo Group · Phase 2

Experimental Group · 1 Intervention: Vatiquinone · Intervention Types: Drug

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 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
Closest Location: The Children's Hospital of Philadelphia · Philadelphia, PA
Photo of Philadelphia  1Photo of Philadelphia  2Photo of Philadelphia  3
2005First Recorded Clinical Trial
7 TrialsResearching Friedreich Ataxia
206 CompletedClinical Trials

Eligibility Criteria

Age < 18 · All Participants · 4 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You are able to take vatiquinone oral solution with food.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.