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Long-term safety and effectiveness study of vatiquinone (oral capsules) in patients with Friedreich ataxia who have previously received this treatment

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on Friedreich ataxia, a rare genetic disorder that affects the nervous system and causes progressive damage to the spinal cord and nerves. The study will evaluate a medication called vatiquinone (also known as PTC743), which is being developed to treat conditions affecting cellular energy production.

The purpose of this research is to assess the long-term safety of vatiquinone in people with Friedreich ataxia who have previously received this medication in other studies. The medication comes in the form of capsules that are taken by mouth. Participants will receive vatiquinone for up to 36 months, with a maximum daily dose of 200 mg.

During the study, participants will be monitored for any health changes and will undergo various medical assessments. These assessments will include checking their ability to maintain balance while standing, movement of arms and legs, and speech function. The study will track how these abilities may change over time while taking the medication.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial medication administration

    You will receive vatiquinone in the form of oral capsules

    This medication is intended for patients with Friedreich ataxia who have previously taken vatiquinone in another clinical study

  2. Step 2

    Contraception requirements

    If you are able to have children, you must use effective contraception methods from the start of the study until 30 days after the last dose

    Acceptable methods include: implants, injections, patches, oral contraceptives, barrier methods, or intrauterine devices

    Male participants must not donate sperm during the study and for 30 days after the last dose

    If no other contraception method is used, a double-barrier method (condom with spermicide) is required

  3. Step 3

    Safety monitoring

    Your health will be monitored through regular checks of:

    - Adverse events and serious adverse events

    - Laboratory tests

    - Vital signs

    - Heart recordings (ECGs)

  4. Step 4

    Assessment of condition

    Your condition will be evaluated using the mFARS scale, which measures:

    - Ability to maintain upright position

    - Upper arm and hand function

    - Lower limb function

    - Speech and swallowing function

  5. Step 5

    Study duration

    The study continues until December 31, 2027

    Your participation may end earlier if needed

Who can join the trial?

13 criteria

  • Must have previously participated in and completed a clinical study using vatiquinone (an investigational medication)
  • Must be willing to provide informed consent to participate in the study
  • If you are able to have children (both men and women), you must agree to use effective birth control methods such as:
    • Implants
    • Injectable contraceptives
    • Birth control patch
    • Birth control pills
    • Barrier methods (like condoms)
    • Intrauterine devices (IUD)
    • Must use birth control from the time of signing consent until 30 days after the last dose of study medication
    • Male participants must agree not to donate sperm during the study and for 30 days after the last dose
    • If no other birth control method is being used, participants must use a double-barrier method (condom with spermicide)
    • Both men and women can participate in this study
    • The study includes adults and adolescents

Who cannot join the trial?

10 criteria

  • Having any medical condition that could interfere with the safe conduct of the study, as determined by the study doctor
  • History of significant allergic reactions or hypersensitivity to vatiquinone or similar medications
  • Pregnancy or breastfeeding
  • Participation in another clinical trial within the past 30 days
  • Inability to comply with study procedures and follow-up visits
  • Having any unstable medical conditions that require immediate treatment
  • Use of prohibited medications that might interact with the study drug
  • Significant abnormalities in blood tests or other laboratory results that could affect safety
  • History of substance abuse within the past year
  • Mental conditions that could affect the ability to provide informed consent or follow study instructions
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Investigated drugs

Vatiquinone is a medication being studied for treating Friedreich Ataxia, a rare genetic disease that affects the nervous system and heart. This drug is designed to help protect cells from damage caused by the disease. It works by supporting the function of mitochondria (the energy-producing parts of cells) and may help reduce oxidative stress, which is thought to play a role in the progression of Friedreich Ataxia.

What is already known about the treatment

Vatiquinone - An investigational drug administered orally that is being studied for the treatment of Friedreich ataxia, a rare genetic neurodegenerative disorder. This medication belongs to the class of quinone compounds and works by targeting cellular energy production in mitochondria, potentially helping to protect nerve cells from damage. Currently undergoing clinical trials to evaluate its long-term safety and effectiveness, vatiquinone represents a promising therapeutic approach for patients with Friedreich ataxia, though it is not yet approved for general medical use.

Investigated diseases

Friedreich ataxia - A genetic neurological disorder that affects the nervous system and causes progressive damage to the spinal cord and nerves. The condition typically begins in childhood or adolescence and causes problems with movement and coordination. People with this condition experience progressive difficulty with balance, walking, and speech clarity. The disorder also commonly affects fine motor skills in the hands and arms. Friedreich ataxia can lead to changes in heart function and may cause curvature of the spine.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-516505-23-00Protocol codePTC743-NEU-003e-FAEstimated enrolment134 patientsSponsorPTC Therapeutics Inc.

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