Clinical trial NCT05218655: 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
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Inherited Mitochondrial Disease
Keywords: Leigh syndrome
Alpers Syndrome
mitochondrial encephalomyopathy
MELAS
MERRF
PCH6
refractory epilepsy
Lead sponsor: PTC Therapeutics
Sponsor class: INDUSTRY
First posted: 2022-02-01
Enrollment: 102
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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 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.

Primary outcomes:
measure: Number of Participants With Adverse Events (AEs)
timeFrame: Baseline (Day 1) up to end of study (up to approximately 3 years)
Secondary outcomes:
Other outcomes:
Locations:
city: La Jolla
country: United States
facility: University of California, San Diego Altman Clinical and Translational Research Institute
state: California
city: Stanford
country: United States
facility: Stanford University
state: California
city: New Haven
country: United States
facility: Yale Medicine
state: Connecticut
city: Washington
country: United States
facility: Children's National
state: District of Columbia
city: Gulf Breeze
country: United States
facility: Child Neurology Center of Northwest Florida
state: Florida
city: Baltimore
country: United States
facility: The Johns Hopkins University
state: Maryland
city: New York
country: United States
facility: Columbia University Medical Center - CUMC
state: New York
city: Akron
country: United States
facility: Akron Children's Hospital
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia - CHOP
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina - MUSC
state: South Carolina
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Houston
country: United States
facility: UT Health The University of Texas
state: Texas
city: Seattle
country: United States
facility: Seattle Children Hospital
state: Washington
city: Angers Cedex 9
country: France
facility: Chu Angers
city: Montpellier Cedex 5
country: France
facility: CHU Montpellier - Hopital Saint-Eloi
city: Paris
country: France
facility: Hopital Necker-Enfants Malades
city: Strasbourg
country: France
facility: Hôpital de Hautepierre - Hôpitaux Universitaires de Strasbourg
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambino Gesù
city: Japanese City
country: Japan
facility: PTC Clinical Site
city: Warszawa
country: Poland
facility: Instytut Pomnik - Centrum Zdrowia Dziecka
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Déu
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Madrid
country: Spain
facility: Hospital Ruber Internacional
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children NHS Foundation Trust
city: Newcastle Upon Tyne
country: United Kingdom
facility: The Newcastle Upon Tyne Hospitals NHS Foundation Trust
Study officials:
affiliation: PTC Therapeutics
name: Vinay Penematsa, MD
role: STUDY_DIRECTOR
References: