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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05515536




Registration number
NCT05515536
Ethics application status
Date submitted
23/08/2022
Date registered
25/08/2022
Date last updated
19/11/2024

Titles & IDs
Public title
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
Scientific title
Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia
Secondary ID [1] 0 0
2022-002668-65
Secondary ID [2] 0 0
PTC743-NEU-003e-FA
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Friedreich Ataxia 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Neurodegenerative diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Vatiquinone

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 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: Drugs: Vatiquinone
Vatiquinone will be administered per dose and schedule specified in the arm.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Adverse Events (AEs)
Timepoint [1] 0 0
Baseline up to 3 years
Secondary outcome [1] 0 0
Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) and its 4 Subscales (Upright Stability, Upper Limb, Lower Limb, Bulbar) Scores at Year 3
Timepoint [1] 0 0
Baseline, Year 3

Eligibility
Key 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.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Current participation in any other interventional study
* Pregnancy or breastfeeding

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/12/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/12/2027
Actual
Sample size
Target
140
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Iowa
Country [4] 0 0
United States of America
State/province [4] 0 0
Pennsylvania
Country [5] 0 0
Brazil
State/province [5] 0 0
São Paulo
Country [6] 0 0
Canada
State/province [6] 0 0
Quebec
Country [7] 0 0
France
State/province [7] 0 0
PARIS cedex
Country [8] 0 0
Germany
State/province [8] 0 0
Tuebingen
Country [9] 0 0
Italy
State/province [9] 0 0
Roma
Country [10] 0 0
New Zealand
State/province [10] 0 0
Auckland
Country [11] 0 0
Spain
State/province [11] 0 0
Barcelona

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
PTC Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT05515536
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05515536