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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

PTC Therapeutics Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

22 Mar 2023 → ongoing

Decision date (initial)

2024-10-25

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

PTC Therapeutics, Inc.

External identifiers

EU CT number

2024-516505-23-00

EudraCT number

2022-002668-65

ClinicalTrials.gov

NCT05515536

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To assess the long-term safety of vatiquinone in subjects with FA previously exposed to vatiquinone

Secondary objectives 1

1. To assess the effects of vatiquinone on the Modified Friedreich Ataxia Rating Scale (mFARS) and its subscales in subjects with FA previously exposed to vatiquinone in Study PTC743NEU-003-FA or Study PTC743-NEU005-FA

Conditions and MedDRA coding

Friedreich ataxia

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Subjects with FA who completed and directly rolled over from a previous vatiquinone clinical study.
2. Males and females of childbearing potential must be willing to use an effective method of contraception (eg, implants, injectables, transdermal patch, combined oral contraceptives, barrier methods, and intrauterine devices) from the time consent is signed until 30 days after last dose of study drug or Early Termination Visit. Male subjects must agree not to donate sperm during the study and for at least 30 days after the last dose of study drug or Early Termination Visit. Note: Double-barrier method (ie, condom with spermicide) is required if no other methods of contraception are in use.
3. Subject provides consent to participate.

Exclusion criteria 2

1. Current participation in any other interventional study
2. Pregnancy or breastfeeding

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Adverse events (AEs)/serious adverse events (SAEs), laboratory abnormalities, vital signs, and electrocardiograms (ECGs)

Secondary endpoints 1

1. Change from baseline in the mFARS and its 4 subscales (Upright Stability, Upper Limb, Lower Limb, Bulbar)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

PTC Therapeutics Inc.

Sponsor organisation

PTC Therapeutics Inc.

Address

500 Warren Corporate Center Drive

City

Warren

Postcode

07059

Country

United States

Scientific contact point

Organisation

PTC Therapeutics Inc.

Contact name

Mark Rance, MBBS BSc(Hons) MRCS(Eng)

Public contact point

Organisation

PTC Therapeutics Inc.

Contact name

Mark Rance, MBBS BSc(Hons) MRCS(Eng)

Third parties 8

Locations

4 EU/EEA countries · 4 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 41 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications