A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

JRCT ID: jRCT2011230045

Registered date:05/11/2023

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Inherited Mitochondrial Disease
• Date of first enrollment: 31/10/2023
• Target sample size: 10
• Countries of recruitment: US,Japan,UK,Japan,Italy,Japan,Spain,Japan,France,Japan,Poland,Japan
• Study type: Interventional

Intervention(s)

Vatiquinone (PTC743):
Participants will receive vatiquinone oral solution (100 mg/mL), up to 400 mg, administered orally or via feeding tube 3 times daily (TID).

Outcome(s)

• Primary Outcome: Number of Participants With Adverse Events (AEs) [Time Frame: Baseline (Day 1) up to end of study (up to approximately 3 years)]
• Secondary Outcome: N/A

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: Not applicable
• Gender: Both

Include criteria

- Participants 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.
- Women of childbearing potential must have a negative pregnancy test at screening/baseline and agree to abstinence or the use of at least 1 of the highly effective forms of contraception as specified in the protocol (with a failure rate of <1% per year when used consistently and correctly). Highly effective contraception or abstinence must be continued for the duration of the study, and for up to 30 days after the last dose of study drug.
- Fertile men who are sexually active with women of childbearing potential and who have not had a vasectomy, must agree to use a barrier method of birth control during the study and for up to 30 days after the last dose of study drug.

Exclude criteria

- Current participation in any other interventional study.
- Pregnancy or breast feeding.

Related Information

• Primary Sponsor: Vinay Penematsa
• Secondary ID(s): NCT05218655,2022-000375-39