臨床研究等提出・公開システム

Japanese

Date of registration	July. 21, 2023
Last modified on	July. 21, 2023
Trial ID	jRCT2031230248

Scientific Title	A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated With Danicopan in an Alexion-sponsored Clinical Study
Public Title	A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated With Danicopan in an Alexion-sponsored Clinical Study

Contact for Scientific Queries	Name	Sugita Yuko
	Affiliation	Alexion Pharma GK
	Address	Tamachi Station Tower N, 3-1-1 Shibaura, Minato-ku, Tokyo
	Telephone	+81-3-3457-9559
	E-mail	yuko.sugita@alexion.com
Contact for Public Queries	Name	Sugita Yuko
	Affiliation	Alexion Pharma GK
	Address	Tamachi Station Tower N, 3-1-1 Shibaura, Minato-ku, Tokyo
	Telephone	+81-3-3457-9559
	E-mail	yuko.sugita@alexion.com

Recruitment status	Recruiting

Anticipated date of first enrollment		July. 21, 2023
Actual date of first enrollment
Target sample size		100
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	- All participants who completed their participation in an Alexion sponsored clinical study with danicopan as an add on to a C5i treatment. - Patient is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol. - Documentation of vaccination for Neisseria meningitidis: All participants must be revaccinated as per national vaccination guidelines or local practice for vaccination use with complement inhibitors.
	Exclusion Criteria	- Any medical condition (for example, cardiac, pulmonary, renal, oncologic, or psychiatric) that, in the opinion of the Investigator, might interfere with participation in the study, pose any added risk to the participant, or confound the assessment of the participant. - Patient has been permanently discontinued from danicopan in the parent study for any reason other than enrollment into this LTE study. - Female participants who are pregnant, breastfeeding, or intending to conceive during the course of the study.
	Age Minimum	18age old over
	Age Maximum	No limit
	Gender	Both
Health Condition(s) or Problem(s) Studied		Paroxysmal Nocturnal Hemoglobinuria (PNH)
Intervention(s)		Drug: Danicopan
Health Condition(s) Keyword
Intervention(s) Keyword
Health Condition(s) Code
Intervention(s) Code
Primary Outcome(s)		Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs
Secondary Outcome(s)		Change In Hemoglobin Values

Primary Sponsor	Alexion Pharma GK

Source of Monetary Support / Secondary Sponsor
Secondary Sponsor

Source of Monetary Support
Secondary Sponsor

Name of Certified Review Board	NIPPON MEDICAL SCHOOL HOSPITAL IRB
Address	1-1-5 Sendagi, Bunkyo-ku, Tokyo, Tokyo
Telephone
E-Mail
Approval Status	Approval
Date of approval	Jan. 27, 2023

Plan to share IPD	No
Plan description

Secondary ID(s)
Issuing Authority

Countries of Recruitment（Except Japan）	United States/Italy/United Kingdom