臨床研究等提出・公開システム

Japanese

Date of registration	Nov. 05, 2023
Last modified on	July. 26, 2024
Trial ID	jRCT2011230045

Scientific Title	An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease
Public Title	A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

Contact for Scientific Queries	Name	Vinay Penematsa
	Affiliation	PTC Therapeutics, Inc.
	Address	500 Warren Corporate Center Drive Warren, N.J. 07059 USA
	Telephone	1-866-562-4620
	E-mail	medinfo@ptcbio.com
Contact for Public Queries	Name	Rosario Chikako
	Affiliation	Parexel International Inc.
	Address	Kayabacho Tower, 1-21-2, Shinkawa, Chuo-ku, Tokyo
	Telephone	+81-80-8929-3137
	E-mail	Clinicaltrial-registration@parexel.com

Recruitment status	Not Recruiting

Anticipated date of first enrollment		Nov. 01, 2023
Actual date of first enrollment		Oct. 31, 2023
Target sample size		10
Study Type		Interventional
Study Design	allocation	single arm study
	masking	open(masking not used)
	control	uncontrolled control
	assignment	single assignment
	purpose	treatment purpose
Key inclusion & exclusion 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.
	Age Minimum	No limit
	Age Maximum	No limit
	Gender	Both
Health Condition(s) or Problem(s) Studied		Inherited Mitochondrial Disease
Intervention(s)		Vatiquinone (PTC743): Participants will receive vatiquinone oral solution (100 mg/mL), up to 400 mg, administered orally or via feeding tube 3 times daily (TID).
Health Condition(s) Keyword
Intervention(s) Keyword
Health Condition(s) Code
Intervention(s) Code
Primary Outcome(s)		Number of Participants With Adverse Events (AEs) [Time Frame: Baseline (Day 1) up to end of study (up to approximately 3 years)]
Secondary Outcome(s)		N/A

Primary Sponsor	PTC Therapeutics, Inc.

Source of Monetary Support / Secondary Sponsor
Secondary Sponsor

Source of Monetary Support
Secondary Sponsor

Name of Certified Review Board	Hokkaido University Hospital Institutional Review Board
Address	Kita 14, Nishi 5, Kita-ku, Sapporo, Hokkaido, 060-8648, Japan, Hokkaido
Telephone	+81-11-706-7061
E-Mail	tiken@med.hokudai.ac.jp
Approval Status	Approval
Date of approval	Aug. 15, 2023

Plan to share IPD	No
Plan description

Secondary ID(s)	NCT05218655
Issuing Authority	ClinicalTrials.gov

Secondary ID(s)	2022-000375-39
Issuing Authority	EudraCT

Countries of Recruitment（Except Japan）	US/UK/Italy/Spain/France/Poland

History of Changes

No	Publication date
4	July. 26, 2024	(this page)	Changes
3	Feb. 27, 2024	Detail	Changes
2	Jan. 23, 2024	Detail	Changes
1	Nov. 05, 2023	Detail

List of change history