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

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.

PTC Identifier:
PTC743-NEU-003e-FA
ClinicalTrials.gov Identifier:
NCT05515536
EudraCT Identifier:
2022-002668-65
EU CT ID:
2024-516505-23-00
Not Recruiting

Trial Purpose

Age
N/A
Sex
Both (Female and Male)
Healthy Volunteers
No

Key Participation Requirements

Inclusion Criteria:

  • 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.

Exclusion Criteria:

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

Trial Summary

Total Participants

This is the number of individuals who are taking part in the trial.

Total Participants
130

Trial Dates

These are the start and end dates for the trial. Note that future dates are estimates that may be updated before or during the trial.

Trial Dates
Dec 2022 - Dec 2027

Phase

These are the different steps that a treatment goes through during clinical development before it is approved for use.

Phase
Phase 3

Placebo Controlled

In this type of study, some participants receive a “placebo,” which looks like the study treatment, but does not actually contain the active treatment.

Placebo Controlled
No

Product

This is any type of drug, device, or other treatment that is being studied in the trial.

Product
N/A

Accepts Healthy Volunteers

Healthy volunteers are individuals who participate in the trial and do not have that specific disease or condition.

Accepts Healthy Volunteers
No
Discuss with the trial center for details

Trial Design

Trial Type

This is the general nature of the study.

Trial Type
Interventional

Interventional

Participants in these studies are subject to an intervention by researchers. These trials are used to understand the effects of a particular intervention.

Discuss with the trial center for details

Blinding

Study participants know which treatments they might get if they join the trial, however during a trial they may not be informed which treatment they are actually taking. The act of not disclosing this information to the participant is known as “blinding.”

Blinding
N/A
Discuss with the trial center for details

Intervention Type

This is a medicine, device, or other type of treatment that is being studied or is already approved for use. Interventions can also include behavior changes, like diet and exercise.

Intervention Type
Drug

Allocation

This determines how study participants are assigned their treatment.

Allocation
N/A

Trial Purpose

This is the rationale behind why the clinical trial is being performed.

Trial Purpose
Treatment

Trial Arms

These are the groups of participants in a clinical study. Each different “arm” is assigned a different treatment to use. Different types of trial arms in studies can include:

Trial Arms
Experimental

Experimental arm

Experimental arm: The group assigned to use the study treatment

Primary Outcome

Primary Outcome

This is the most important measurement of the study and is used to help researchers evaluate the effect of a treatment.

Number of Participants With Adverse Events (AEs)

Timeframe: Baseline up to 3 years

Secondary Outcome

Secondary Outcome

This measurement is a secondary measurement and is still important in helping researchers evaluate the effect of a treatment.

Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) and its 4 Subscales (Upright Stability, Upper Limb, Lower Limb, Bulbar) Scores at Year 3

Timeframe: Baseline, Year 3

Contact Information

Location
Status
Location
The Children's Hospital of Philadelphia
Philadelphia, PA, United States, 19104
Status
Enrolling by invitation
Location
University of Iowa
Iowa City, IA, United States, 52242
Status
Not yet recruiting
Location
University of South Florida
Tampa, FL, United States, 33612
Status
Not yet recruiting
Location
UCLA
Los Angeles, CA, United States, 90095
Status
Enrolling by invitation
Location
Murdoch Children’s Research Institute
Parkville, Victoria, Australia, 3052
Status
Enrolling by invitation
Location
Centre de Recherche du Centre Hospitalier de l’Université de Montreal (CRCHUM)
Montreal, Quebec, Canada, H2X 0A9
Status
Not yet recruiting
Go to page