Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)

Part of paid clinical trials in Los Angeles, California.

Sponsor
PTC Therapeutics
Study ID
NCT07681713
Phase
PHASE3
Status
Not Yet Recruiting

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Conditions

  • Friedreich's Ataxia

Eligibility Criteria

Sex
ALL
Age
7 Years - 21 Years
Healthy Volunteers
Not accepted

Interventions

  • Vatiquinone — DRUG
    Vatiquinone will be administered per dose and schedule specified in the arm.

Study Details

The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.

Key Dates

First listed
Jul 2, 2026
Start date
Aug 15, 2026
Status verified
Aug 2026
Primary completion
Mar 15, 2029
Completion
Mar 15, 2029

Study Design

Enrollment
120 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Vatiquinone
    Participants will receive vatiquinone capsule at a dose of either 200 milligrams (mg) orally 3 times a day (TID) if weighing ˂25 kilograms (kg) or 400 mg orally TID if weighing ≥25 kg for 24 months.

Primary Outcome Measure

Change From Baseline in Total Modified Friedreich's Ataxia Rating Scale (mFARS) Score at Month 24 [ Time Frame: Baseline, Month 24 ]

Central Contacts

Locations (4)

FacilityCityStateZIPSite coordinators
University of California Los AngelesLos AngelesCalifornia90095
Susan Perlman (PRINCIPAL_INVESTIGATOR)
University of South FloridaTampaFlorida33612
Theresa Zesiewicz (PRINCIPAL_INVESTIGATOR)
University of IowaIowa CityIowa52242
Katherine Mathews (PRINCIPAL_INVESTIGATOR)
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
David Lynch (PRINCIPAL_INVESTIGATOR)

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University of California Los Angeles· Los Angeles, CAUniversity of South Florida· Tampa, FLUniversity of Iowa· Iowa City, IAChildren's Hospital of Philadelphia· Philadelphia, PA

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