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

PTC Therapeutics

Status and phase: Active, not recruiting; Phase 3

Conditions: Friedreich Ataxia

Treatments: Drug: Vatiquinone

Study type: Interventional

Funder types: Industry

Identifiers

NCT05515536

2022-002668-65 (EudraCT Number)

2024-516505-23-00 (Other Identifier)

PTC743-NEU-003e-FA

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 130 patients

Sex: All

Volunteers: No Healthy Volunteers

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 design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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130 participants in 1 patient group

Vatiquinone

Experimental group

Description:

Participants with FA who were previously treated with vatiquinone at an investigational site and completed participation in a prior PTC Therapeutics (PTC)-sponsored clinical study will continue to receive the same dose/formulation of vatiquinone (unless there has been a change in age and/or weight that meets the criteria for a different dose/formulation as described below; dose/formulation will also be changed if participants meet the criteria for a different dose/formulation during study treatment): If \<7 years of age, participants will receive an oral solution (100 milligrams \[mg\]/milliliter \[mL\]) 3 times a day (TID) at one of the following doses: 15 mg/kilogram (kg) if body weight \<13 kg, or 200 mg if body weight ≥13 kg. If ≥7 years of age, participants will receive a capsule formulation (200 mg) orally TID at one of the following doses: 200 mg if body weight ˂25 kg, or 400 mg if body weight ≥25 kg.

Treatment:

Drug: Vatiquinone