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

Sponsor

PTC Therapeutics (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05515536

Collaborator

(none)

140

Enrollment

Arm

Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

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 in Study PTC743-NEU-003-FA (NCT04577352) or Study PTC743-NEU-005-FA.

Condition or Disease	Intervention/Treatment	Phase
Friedreich Ataxia	Drug: Vatiquinone	Phase 3

Detailed Description

Treatment will continue until the participant withdraw consent, regulatory authority and/or PTC discontinue the study, participant's condition substantially worsens after initiating vatiquinone treatment in this study, investigator withdraws the participant from vatiquinone treatment, if, in the investigator's clinical judgment, it is not in the participant's best interest to continue, or vatiquinone becomes commercially available in a participant's country.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

140 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia

Anticipated Study Start Date :

Nov 1, 2022

Anticipated Primary Completion Date :

Dec 31, 2027

Anticipated Study Completion Date :

Dec 31, 2027

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Vatiquinone 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 (PTC743-NEU-003-FA or PTC743-NEU-005-FA) 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.	Drug: Vatiquinone Vatiquinone will be administered per dose and schedule specified in the arm.

Arm

Intervention/Treatment

Experimental: Vatiquinone

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 (PTC743-NEU-003-FA or PTC743-NEU-005-FA) 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.

Drug: Vatiquinone

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

Outcome Measures

Primary Outcome Measures

Number of Participants With Adverse Events (AEs) [Baseline up to approximately 5 years]

Secondary Outcome Measures

Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) at Year 5 [Baseline, Year 5]

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Participants with FA who completed and directly rolled over from a previous vatiquinone clinical study (PTC743-NEU-003-FA [that is, MOVE-FA] or PTC743-NEU-005-FA).
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

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

PTC Therapeutics

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

PTC Therapeutics

ClinicalTrials.gov Identifier:

NCT05515536

Other Study ID Numbers:

PTC743-NEU-003e-FA

First Posted:

Aug 25, 2022

Last Update Posted:

Aug 25, 2022

Last Verified:

Aug 1, 2022

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Ataxia

Cerebellar Ataxia

Friedreich Ataxia

Study Results

No Results Posted as of Aug 25, 2022