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

Sponsor
PTC Therapeutics (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05218655
Collaborator
(none)
200
Enrollment
16
Locations
1
Arm
30.4
Anticipated Duration (Months)
12.5
Patients Per Site
0.4
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
200 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease
Actual Study Start Date :
Jun 21, 2022
Anticipated Primary Completion Date :
Dec 31, 2023
Anticipated Study Completion Date :
Dec 31, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: Vatiquinone

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).

Drug: Vatiquinone
Vatiquinone will be administered per dose and schedule specified in the arm description.
Other Names:
  • PTC743

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Number of Participants With Adverse Events (AEs) [Baseline (Day 1) up to end of study (up to approximately 3 years)]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    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 50 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 50 days after the last dose of study drug.

    Exclusion Criteria:

    • Current participation in any other interventional study.

    • Pregnancy or breast feeding.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 University of California, San Diego Altman Clinical and Translational Research Institute La Jolla California United States 92037
    2 Stanford University Stanford California United States 94305
    3 Yale Medicine New Haven Connecticut United States 06511
    4 Children's National Washington District of Columbia United States 20010
    5 The Johns Hopkins University Baltimore Maryland United States 21205
    6 Massachusetts General Hospital - MGH Boston Massachusetts United States 02114
    7 Boston Children Hospital Boston Massachusetts United States 02115
    8 Children's of Minnesota Minneapolis Minnesota United States 55404
    9 Columbia University Medical Center - CUMC New York New York United States 10032
    10 Akron Children's Hospital Akron Ohio United States 44308
    11 Cleveland Clinic Cleveland Ohio United States 44195
    12 Children's Hospital of Philadelphia - CHOP Philadelphia Pennsylvania United States 19104
    13 Medical University of South Carolina - MUSC Charleston South Carolina United States 29425
    14 Baylor College of Medicine Houston Texas United States 77030
    15 UT Health The University of Texas Houston Texas United States 77030
    16 Seattle Children Hospital Seattle Washington United States 98105

    Sponsors and Collaborators

    • PTC Therapeutics

    Investigators

    • Study Director: Vinay Penematsa, MD, PTC Therapeutics

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    PTC Therapeutics
    ClinicalTrials.gov Identifier:
    NCT05218655
    Other Study ID Numbers:
    • PTC743-CNS-005-LSEP
    First Posted:
    Feb 1, 2022
    Last Update Posted:
    Jun 29, 2022
    Last Verified:
    Jun 1, 2022
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by PTC Therapeutics
    Leigh syndrome
    Alpers Syndrome
    mitochondrial encephalomyopathy
    MELAS
    MERRF
    PCH6
    refractory epilepsy
    Additional relevant MeSH terms:
    Mitochondrial Diseases

    Study Results

    No Results Posted as of Jun 29, 2022