Back to resultsA Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
Primary Purpose
Inherited Mitochondrial Disease
Status
Enrolling by invitation
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Vatiquinone
Sponsored by
About this trial
This is an interventional treatment trial for Inherited Mitochondrial Disease focused on measuring Leigh syndrome, Alpers Syndrome, mitochondrial encephalomyopathy, MELAS, MERRF, PCH6, refractory epilepsy
Eligibility Criteria
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.
Sites / Locations
- University of California, San Diego Altman Clinical and Translational Research Institute
- Stanford University
- Yale Medicine
- Children's National
- Child Neurology Center of Northwest Florida
- The Johns Hopkins University
- Columbia University Medical Center - CUMC
- Akron Children's Hospital
- Cleveland Clinic
- Children's Hospital of Philadelphia - CHOP
- Medical University of South Carolina - MUSC
- Baylor College of Medicine
- UT Health The University of Texas
- Seattle Children Hospital
- Ospedale Pediatrico Bambino Gesù
- Hospital Sant Joan de Déu
- Hospital Universitario 12 de Octubre
- Hospital Ruber Internacional
- Great Ormond Street Hospital for Children NHS Foundation Trust
- The Newcastle Upon Tyne Hospitals NHS Foundation Trust
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Vatiquinone
Arm 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).
Outcomes
Primary Outcome Measures
Number of Participants With Adverse Events (AEs)
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05218655
Brief Title
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
Official Title
An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
June 21, 2022 (Actual)
Primary Completion Date
March 31, 2025 (Anticipated)
Study Completion Date
November 15, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
PTC Therapeutics
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Inherited Mitochondrial Disease
Keywords
Leigh syndrome, Alpers Syndrome, mitochondrial encephalomyopathy, MELAS, MERRF, PCH6, refractory epilepsy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
200 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Vatiquinone
Arm Type
Experimental
Arm 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).
Intervention Type
Drug
Intervention Name(s)
Vatiquinone
Other Intervention Name(s)
PTC743
Intervention Description
Vatiquinone will be administered per dose and schedule specified in the arm description.
Primary Outcome Measure Information:
Title
Number of Participants With Adverse Events (AEs)
Time Frame
Baseline (Day 1) up to end of study (up to approximately 3 years)
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Vinay Penematsa, MD
Organizational Affiliation
PTC Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
University of California, San Diego Altman Clinical and Translational Research Institute
City
La Jolla
State/Province
California
ZIP/Postal Code
92037
Country
United States
Facility Name
Stanford University
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Yale Medicine
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06511
Country
United States
Facility Name
Children's National
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Child Neurology Center of Northwest Florida
City
Gulf Breeze
State/Province
Florida
ZIP/Postal Code
32561
Country
United States
Facility Name
The Johns Hopkins University
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Columbia University Medical Center - CUMC
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Akron Children's Hospital
City
Akron
State/Province
Ohio
ZIP/Postal Code
44308
Country
United States
Facility Name
Cleveland Clinic
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Children's Hospital of Philadelphia - CHOP
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Medical University of South Carolina - MUSC
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Baylor College of Medicine
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
UT Health The University of Texas
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Seattle Children Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
Ospedale Pediatrico Bambino Gesù
City
Roma
ZIP/Postal Code
00165
Country
Italy
Facility Name
Hospital Sant Joan de Déu
City
Barcelona
ZIP/Postal Code
08950
Country
Spain
Facility Name
Hospital Universitario 12 de Octubre
City
Madrid
ZIP/Postal Code
28003
Country
Spain
Facility Name
Hospital Ruber Internacional
City
Madrid
ZIP/Postal Code
28034
Country
Spain
Facility Name
Great Ormond Street Hospital for Children NHS Foundation Trust
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
Facility Name
The Newcastle Upon Tyne Hospitals NHS Foundation Trust
City
Newcastle Upon Tyne
ZIP/Postal Code
NE1 4LP
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
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