臨床研究等提出・公開システム

Date of registration	Aug. 02, 2023
Last modified on	Dec. 11, 2023
Trial ID	jRCT2031230275

Scientific Title	Special Drug Use-Results Survey (All-Case Surveillance) to Investigate Safety and Effectiveness in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Received Empaveli in the Post-Marketing Clinical Use in Japan (PEG_PNH JPN PMS)
Public Title	Special Drug Use-Results Survey (All-Case Surveillance) to Investigate Safety and Effectiveness in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Received Empaveli in the Post-Marketing Clinical Use in Japan (PEG_PNH JPN PMS)

Contact for Scientific Queries	Name	Shimmoto Naoki
	Affiliation	Swedish Orphan Biovitrum Japan Co., Ltd.
	Address	GINZA SIX 13F, 6-10-1, Ginza, Chuo-ku, Tokyo Japan
	Telephone	+81-3-4400-6872
	E-mail	SobiJapanContact@sobi.com
Contact for Public Queries	Name	Clinical Study Manager
	Affiliation	Swedish Orphan Biovitrum Japan Co., Ltd.
	Address	GINZA SIX 13F, 6-10-1, Ginza, Chuo-ku, Tokyo Japan
	Telephone	+81-3-4400-6872
	E-mail	SobiJapanContact@sobi.com

Recruitment status	Recruiting

Anticipated date of first enrollment		Sept. 04, 2023
Actual date of first enrollment		Nov. 06, 2023
Target sample size		100
Study Type		Observational
Key inclusion & exclusion criteria	Inclusion Criteria	All patients with PNH who received Empaveli in Japan from the date of product launch to 31 May 2028 or until the approval conditions are lifted.
	Exclusion Criteria	Not applicable.
	Age Minimum	No limit
	Age Maximum	No limit
	Gender	Both
Health Condition(s) or Problem(s) Studied		Paroxysmal nocturnal hemoglobinuria
Primary Outcome(s)		Occurrence of the following adverse reactions: Hypersensitivity, Meningococcal infection, Infections (except meningococci), Serious hemolysis due to discontinuation of Empaveli.

Primary Sponsor	Swedish Orphan Biovitrum Japan Co., Ltd.

Name of Certified Review Board	Not applicable.
Address	Not applicable., Tokyo

Plan to share IPD	No

Countries of Recruitment（Except Japan）	none

History of Changes

No	Publication date
4	Dec. 11, 2023	(this page)	Changes
3	Sept. 06, 2023	Detail	Changes
2	Sept. 04, 2023	Detail	Changes
1	Aug. 02, 2023	Detail

List of change history