臨床研究等提出・公開システム

Date of registration	Dec. 04, 2021
Last modified on	July. 29, 2025
Trial ID	jRCT2061210056

Scientific Title	An Expanded Access Study for Cipaglucosidase Alfa in Conjunction with Miglustat for the Treatment of Adult Subjects with Late-Onset Pompe Disease in Japan
Public Title	ATB200/AT2221 Expanded Access Study in Adult Pompe disease Subject

Contact for Scientific Queries	Name	Wright Jacquelyn
	Affiliation	Amicus Therapeutics, Inc.
	Address	47 Hulfish Street, Princeton, NJ
	Telephone	1-609-662-5085
	E-mail	PompeSiteInfo@amicusrx.com
Contact for Public Queries	Name	HARIMA Akiko
	Affiliation	CMIC Co., Ltd.
	Address	Hamamatsucho Building, 1-1-1, Shibaura, Minato-ku,Tokyo
	Telephone	+81-3-6779-8000
	E-mail	ClinicalTrialInformation@cmic.co.jp

Recruitment status	Not Recruiting

Anticipated date of first enrollment		Dec. 22, 2021
Actual date of first enrollment		Dec. 22, 2021
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	1. Male or female, 18 years old and above 2. A diagnosis of LOPD based on documented deficiency of acid alpha-glucosidase (GAA) enzyme activity or GAA genotyping 3. Female subjects of childbearing potential and male subjects if they are using appropriate, reliable contraception
	Exclusion Criteria	1. Subject has a hypersensitivity to any of the excipients in cipaglucosidase alfa or miglustat 2. Subject has a medical condition or any other extenuating circumstance that may pose an undue safety risk to the patient 3. Subject, if female, is pregnant or breastfeeding 4. Subject, whether male or female, is planning to conceive a child during the study 5. Subject has received treatment with prohibited medications within 30 days of screening
	Age Minimum	18age old over
	Age Maximum	No limit
	Gender	Both
Health Condition(s) or Problem(s) Studied		Pompe disease
Intervention(s)		ATB200 IV infusion over a 4-hour duration 1 hour after AT2221 administration every 2 weeks
Primary Outcome(s)		To evaluate the safety of ATB200/AT2221 co-administration

Primary Sponsor	Amicus Therapeutics, Inc.

Name of Certified Review Board	Tottori University Hospital Institutional Review Board
Address	36-1 Nishi-cho, Yonago, Tottori
Telephone	+81-859-38-6946
E-Mail	toridai-chiken@ml.med.tottori-u.ac.jp
Approval Status	Approval
Date of approval	Nov. 29, 2021

Plan to share IPD	No

Countries of Recruitment（Except Japan）	none

History of Changes

No	Publication date
4	July. 29, 2025	(this page)	Changes
3	May. 21, 2025	Detail	Changes
2	July. 23, 2024	Detail	Changes
1	Dec. 04, 2021	Detail

List of change history