ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

NCT ID: NCT03911505

Last Updated: 2025-10-27

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-02-13
• Study Completion Date: 2026-06-30

Brief Summary

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to < 18 years

Conditions

Pompe Disease (Late-onset)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221)

Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsule

Group Type: EXPERIMENTAL

Cipaglucosidase Alfa

Intervention Type: BIOLOGICAL

Enzyme Replacement Therapy via intravenous infusion

Miglustat

Intervention Type: DRUG

Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat(AT2221)

Interventions

Cipaglucosidase Alfa

Enzyme Replacement Therapy via intravenous infusion

Intervention Type: BIOLOGICAL

Miglustat

Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat(AT2221)

Intervention Type: DRUG

Other Intervention Names

ATB200; AT2221

Eligibility Criteria

Inclusion Criteria

1. Male or female subjects (ERT-naïve [have never received a dose of rhGAA] or ERT-experienced [have received rhGAA every 2 weeks for at least 6 months immediately before enrollment, and if ERT dosage has been modified, must have been on the modified dosage for at least 3 months before enrollment]) diagnosed with LOPD who are aged 12 to <18 years at screening (Cohort 1 only) or aged 0 months to < 12 years at screening (Cohort 2 only)

2. Subject weighs ≤ 115 kg. (Cohort 1 Only)

3. Subject must have a diagnosis of LOPD based on documentation as defined in study protocol

4. If of reproductive potential and if sexually active, female and male subjects agree to use a highly effective method of contraception throughout the duration of the study and for up to 90 days after their last dose of Cipaglucosidase Alfa/Miglustat

5. Subject has a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy Adolescents at screening (Cohort 1 only)

6. Subject (aged 12 to <18 years; Cohort 1) performs one 6-Minute Walk Test (6MWT) (≥ 75 meters) at screening that is valid, as determined by the clinical evaluator, or subject (aged ≥ 5 to < 12 years; Cohort 2) performs one 6MWT (≥ 40 meters) at screening that is valid, as determined by the clinical evaluator

Exclusion Criteria

1. Subject has received any investigational/experimental drug, oral anabolic steroid or derivative, biologic, or device within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before screening

2. Subject has received treatment with prohibited medications within 30 days of screening

3. Subject has received any gene therapy at any time

4. Subject has any intercurrent illness or condition at screening or baseline that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator and/or the medical monitor that the potential subject may have an unacceptable risk by participating in this study

5. Subject has a hypersensitivity to any of the excipients in ATB200, approved rhGAA, or AT2221

6. Female subject is pregnant or breast-feeding at screening

7. Subject requires the use of ventilation support for > 6 hours per day while awake

8. Subject has evidence of moderate to severe hypertrophic cardiomyopathy aligning with classic IOPD

9. In the opinion of the investigator, the parent or legally authorized representative is unlikely or unable to comply with the study requirements

10. Subject has any prior history of illness or condition known to affect motor function, such as, but not limited to, Guillain-Barre syndrome, cerebral palsy, etc

11. Subject who is diagnosed with Pompe disease via newborn screening and is asymptomatic (ie, showing no signs and symptoms of Pompe disease (Cohort 2 Only)

• Minimum Eligible Age: 0 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Amicus Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

University of Florida Clinical Research Center

Gainesville, Florida, United States

Wolfson Children's Hospital

Jacksonville, Florida, United States

Woodruff Memorial Research Building

Atlanta, Georgia, United States

St. Louis Children's Hospital

St Louis, Missouri, United States

Duke University Medical Center

Durham, North Carolina, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

University of Utah, Clinical and Translational Sciences Institute

Salt Lake City, Utah, United States

Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Fairfax, Virginia, United States

Women's and Children's Hospital

North Adelaide, South Australia, Australia

University of Calgary

Calgary, Alberta, Canada

SphinCS GmbH Clinical Science for LSD

Hochheim am Main, Hesse, Germany

San Gerardo Hospital

Monza, , Italy

Izumi City General Hospital

Osaka, Izumi-Shi, Japan

Gunma University Hospital

Gunma, , Japan

Tohoku University Hospital

Miyagi, , Japan

Tokyo Women's Medical University

Tokyo, , Japan

Countries

United States; Australia; Canada; Germany; Italy; Japan

Other Identifiers

ATB200-04

Identifier Type: -

Identifier Source: org_study_id