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

PTC Therapeutics

Status and phase: Completed; Phase 3

Conditions: Inherited Mitochondrial Disease

Treatments: Drug: Vatiquinone

Study type: Interventional

Funder types: Industry

Identifiers

NCT05218655

PTC743-CNS-005-LSEP

2022-000375-39 (EudraCT Number)

Details and patient eligibility

About

The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical study or treatment plan.

The study will continue until vatiquinone becomes commercially available or the program is terminated.

Enrollment: 101 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion 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.

Exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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101 participants in 1 patient group

Vatiquinone

Experimental group

Description:

Participants will receive vatiquinone oral solution (100 milligrams \[mg\]/milliliter \[mL\]), up to 400 mg, administered orally or via feeding tube 3 times daily (TID).

Treatment:

Drug: Vatiquinone