This study is in progress, not accepting new patients

A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

a study on Cerebellar Ataxia Friedreich's Ataxia

Summary

Location
at UCLA
Dates
study started
completion around
Principal Investigator
by Susan L. Perlman, MD (ucla)
Headshot of Susan L. Perlman
Susan L. Perlman

Description

Summary

The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone.

Official Title

Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia

Details

This long-term, open-label study will serve as a continued access study for participants with FA who have previously participated in a vatiquinone study. The purpose of this study is to assess continued safety and efficacy of vatiquinone dosing in previously treated participants. This study addresses the medical need for participants to continue vatiquinone with a planned study duration of 3 years.

Keywords

Friedreich Ataxia, Ataxia, Cerebellar Ataxia, Vatiquinone

Eligibility

You can join if…

  • Participants with FA who completed and directly rolled over from a previous vatiquinone clinical study.
  • Males and females of childbearing potential must be willing to use an effective method of contraception (for example, implants, injectables, transdermal patches, combined oral contraceptives, barrier methods, and intrauterine devices) from the time consent is signed until 30 days after the last dose of study drug or Early Termination Visit.

You CAN'T join if...

  • Current participation in any other interventional study
  • Pregnancy or breastfeeding

Locations

  • UCLA
    Los Angeles California 90095 United States
  • University of Iowa
    Iowa City Iowa 52242 United States

Lead Scientist at University of California Health

  • Susan L. Perlman, MD (ucla)
    HS Clinical Professor, Neurology, Medicine. Authored (or co-authored) 182 research publications

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
PTC Therapeutics
ID
NCT05515536
Phase
Phase 3 research study
Study Type
Interventional
Participants
About 130 people participating
Last Updated