NIPH Clinical Trials Search

JRCT ID: jRCT2061210056

Registered date:04/12/2021

ATB200/AT2221 Expanded Access Study in Adult Pompe disease Subject

Basic Information

Recruitment status Recruiting
Health condition(s) or Problem(s) studiedPompe disease
Date of first enrollment22/12/2021
Target sample size10
Countries of recruitment
Study typeInterventional
Intervention(s)ATB200 IV infusion over a 4-hour duration 1 hour after AT2221 administration every 2 weeks


Primary OutcomeTo evaluate the safety of ATB200/AT2221 co-administration
Secondary Outcome

Key inclusion & exclusion criteria

Age minimum>= 18age old
Age maximumNot applicable
Include criteria1. 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
Exclude criteria1. 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

Related Information


Public contact
Name Akiko HARIMA
Address Hamamatsucho Building, 1-1-1, Shibaura, Minato-ku,Tokyo Tokyo Japan 105-0023
Telephone +81-3-6779-8000
Affiliation CMIC Co., Ltd.
Scientific contact
Name Jacquelyn Wright
Address 3675 Market Street, Philadelphia, PA Japan 08512
Telephone 1-609-662-5085
Affiliation Amicus Therapeutics, Inc.