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Vatiquinone for Friedreich Ataxia (MOVE-FA Trial)

Phase 2 & 3
Waitlist Available
Research Sponsored by PTC Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Difference in the mFARS at screening and baseline of no more than 4 points.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, week 72
Awards & highlights

MOVE-FA Trial Summary

This trial is testing a drug called vatiquinone to see if it can help improve symptoms of Friedreich ataxia, a rare neurological disorder.

Eligible Conditions
  • Friedreich Ataxia

MOVE-FA Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

MOVE-FA Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, week 72
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 72 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) Score at Week 72
Secondary outcome measures
Change From Baseline in 1-Minute Walk Test (1MWT) at Week 72
Change From Baseline in Friedreich Ataxia Rating Scale Activities of Daily Living (FARS-ADL) Score at Week 72
Accidental Falls

MOVE-FA Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: VatiquinoneExperimental Treatment1 Intervention
Participants will receive vatiquinone capsule at a dose of either 200 milligrams (mg) orally 3 times a day (TID) if ˂12 years of age and weighing ˂25 kilograms (kg) or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 72 weeks during the placebo-controlled phase and for 24 weeks during the open-label extension phase.
Group II: PlaceboPlacebo Group2 Interventions
Participants will receive placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the placebo-controlled phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label extension phase.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Vatiquinone
2020
Completed Phase 3
~210

Find a Location

Who is running the clinical trial?

PTC TherapeuticsLead Sponsor
74 Previous Clinical Trials
6,084 Total Patients Enrolled
3 Trials studying Friedreich Ataxia
205 Patients Enrolled for Friedreich Ataxia

Media Library

Vatiquinone (Other) Clinical Trial Eligibility Overview. Trial Name: NCT04577352 — Phase 2 & 3
Friedreich Ataxia Research Study Groups: Placebo, Vatiquinone
Friedreich Ataxia Clinical Trial 2023: Vatiquinone Highlights & Side Effects. Trial Name: NCT04577352 — Phase 2 & 3
Vatiquinone (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04577352 — Phase 2 & 3
Friedreich Ataxia Patient Testimony for trial: Trial Name: NCT04577352 — Phase 2 & 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is it currently possible to enroll in this clinical trial?

"As of right now, this particular trial has wrapped up recruiting patients. The listing for this trial was first posted on December 17th, 2020 and updated most recently on November 4th, 2022. However, there are still other ongoing trials that may be of interest. Out of the 48 trials currently looking for participants, 4 trials involve Vatiquinone."

Answered by AI

Are there a lot of research facilities testing this in Canada right now?

"If you are interested in enrolling in this trial, please note that there are 7 sites recruiting patients. They are situated in Tampa, Los Angeles, Montreal, and 7 other cities. To reduce stress, please try to enroll at the site closest to your location."

Answered by AI

Can you give me some other examples of Vatiquinone's usage?

"Vatiquinone was first used in a clinical setting in 2010 at CHOC Children's Clinic. Since that time, there have been a total of 18248 completed clinical trials. At the moment, there are 4 active clinical trials, with a few of these trials based in Tampa, California."

Answered by AI

How many patients will be enrolling in this clinical trial?

"This particular trial is not currently admitting patients. However, it was first posted on December 17th, 2020 and was last updated on November 4th, 2022. For individuals who are looking for other studies, there are 48 clinical trials for friedreich ataxia and 4 for Vatiquinone that are currently admitting patients."

Answered by AI

Does this clinical trial explore new territory?

"4 clinical trials for Vatiquinone are ongoing in 27 cities and 13 nations. The first study of Vatiquinone was in 2010. PTC Therapeutics sponsored the initial Phase 2 drug approval trial that included 94 patients. Since then, 18248 more trials have concluded."

Answered by AI

Who else is applying?

What site did they apply to?
The Children's Hospital of Philadelphia
What portion of applicants met pre-screening criteria?
Did not meet criteria
How many prior treatments have patients received?
0

Why did patients apply to this trial?

I have not tried any drugs yet.
PatientReceived 2+ prior treatments
~34 spots leftby Mar 2025