Vatiquinone for Friedreich Ataxia
Phase-Based Progress Estimates
Effectiveness
Safety
The Children's Hospital of Philadelphia, Philadelphia, PA
Friedreich Ataxia+2 More
Vatiquinone - DrugEligibility
< 18
All Sexes
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Eligibility
< 18
All Sexes
What conditions do you have?
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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
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
This is further along than 68% of similar trials
Trial Design
1 Treatment Group
Vatiquinone
1 of 1
Experimental Treatment
5 Total Participants · 1 Treatment Group
Primary Treatment: Vatiquinone · No Placebo Group · Phase 2
Vatiquinone
Drug
Experimental Group · 1 Intervention: Vatiquinone · Intervention Types: DrugTrial 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
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 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.
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