ATB200/AT2221 Expanded Access Study in Adult Pompe disease Subject

An Expanded Access Study for Cipaglucosidase Alfa in Conjunction with Miglustat for the Treatment of Adult Subjects with Late-Onset Pompe Disease in Japan

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2061210056

Enrollment

Registered

2021-12-04

Start date

2021-12-22

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pompe disease

Interventions

ATB200 IV infusion over a 4-hour duration 1 hour after AT2221 administration every 2 weeks

Eligibility

Sex/Gender

All

Inclusion 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

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

Design outcomes

Primary

Measure	Time frame
To evaluate the safety of ATB200/AT2221 co-administration	—

Contacts

Public ContactAkiko HARIMA

CMIC Co., Ltd.

ClinicalTrialInformation@cmic.co.jp+81-3-6779-8000

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026