Trials / Completed
CompletedNCT05485987
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
An Open-Label Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Vatiquinone in Children With Friedreich Ataxia Younger Than 7 Years of Age
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 5 (actual)
- Sponsor
- PTC Therapeutics · Industry
- Sex
- All
- Age
- 6 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Vatiquinone | Vatiquinone will be administered per dose and schedule specified in the arm. |
Timeline
- Start date
- 2022-10-12
- Primary completion
- 2024-08-29
- Completion
- 2024-08-29
- First posted
- 2022-08-03
- Last updated
- 2025-08-22
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05485987. Inclusion in this directory is not an endorsement.