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Clinical Trials/NCT05218655
NCT05218655CompletedPhase 3

An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease

PTC Therapeutics26 sites in 7 countries101 target enrollmentStarted: June 22, 2022Last updated:
Conditions
Interventions
Drugs

Trial Snapshot

Phase
Phase 3
Status
Completed
Enrollment
101
Locations
26
Primary Endpoint
Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Study Overview

Brief Summary

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.

Study Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Sex
All
Accepts Healthy Volunteers
No

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.

Arms & Interventions

Vatiquinone

Experimental

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).

Intervention: Vatiquinone (Drug)

Outcomes

Primary Outcomes

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Time Frame: Baseline (Day 1) up to 30 days after last dose of study drug (956 days)

An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs included both serious adverse events (SAEs) and non-serious AEs. A TEAE was defined as an AE that had an onset date or date of worsening on or after the first dose of study drug and within 30 days of the date of the last dose of treatment. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.

Secondary Outcomes

No secondary outcomes reported

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (26)

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