AN OPEN-LABEL, SAFETY STUDY FOR PREVIOUSLY TREATED VATIQUINONE (PTC743) SUBJECTS WITH INHERITED MITOCHONDRIAL DISEASE

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2022-000375-39-FR

Enrollment

200

Registered

2022-12-22

Start date

2023-06-27

Completion date

Unknown

Last updated

2025-09-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Inherited mitochondrial disease MedDRA version: 20.0 Level: HLT Classification code 10052637 Term: Genetic mitochondrial abnormalities NEC System Organ Class: 100000004850

Interventions

Product Name: Vatiquinone Product Code: PTC743 Pharmaceutical Form: Oral solution INN or Proposed INN: Vatiquinone CAS Number: 1213269-98-7 Current Sponsor code: PTC743 Other descriptive name: EPI743

alpha-tocotrienol quinone Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100-

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Subjects with inherited mitochondrial disease including Leigh syndrome, Alpers Syndrome, mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), myoclonic epilepsy with ragged-red fibers (MERRF), pontocerebellar hypoplasia type 6 (PCH6), or other mitochondrial disease who participated in a previous vatiquinone clinical study or treatment plan. 2. Women of childbearing potential, as defined in (CTFG 2020), must have a negative pregnancy test at screening/baseline and agree to abstinence or the use of at least one of the following highly effective forms of contraception (with a failure rate of =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. Current participation in any other interventional study. 2. Pregnancy or breast feeding.

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the safety of vatiquinone in subjects with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron-sponsored (previously Edison) clinical study or treatment plan ;Secondary Objective: N/A ;Primary end point(s): Adverse events (AEs)/SAEs, ECGs, vital signs, and laboratory data (hematology, biochemistry, and urine data) ;Timepoint(s) of evaluation of this end point: throughout study	—

Measure

Time frame

Main Objective: To assess the safety of vatiquinone in subjects with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron-sponsored (previously Edison) clinical study or treatment plan ;Secondary Objective: N/A ;Primary end point(s): Adverse events (AEs)/SAEs, ECGs, vital signs, and laboratory data (hematology, biochemistry, and urine data) ;Timepoint(s) of evaluation of this end point: throughout study

—

Secondary

Measure	Time frame
Secondary end point(s): N/A ;Timepoint(s) of evaluation of this end point: N/A	—

Countries

France, Italy, Japan, Poland, Spain, Sweden, United Kingdom, United States

Contacts

Public ContactPatient Advocacy

PTC THERAPEUTICS, INC.

medinfo@ptcbio.com+19082227000

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026