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

ACTIVE_NOT_RECRUITINGPHASE3

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

An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease

NCT03911505•Amicus Therapeutics

View on ClinicalTrials.gov

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

Eligibility

Age

0 - 17 years

Sex

ALL

Healthy Volunteers

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
+1 more criteria

Locations (17)

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

Key Dates

Start Date

February 13, 2020

Primary Completion Date

June 1, 2026

Completion Date

June 1, 2026

Last Updated

October 27, 2025

Enrollment

ACTUAL participants

Conditions

Pompe Disease (Late-onset)

Interventions

Cipaglucosidase Alfa

BIOLOGICAL

Miglustat

DRUG

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

NCT03694561

Pompe DiseasePompe Disease (Late-onset)+1 more

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ACTIVE_NOT_RECRUITINGPHASE1/PHASE2

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

NCT04093349

Pompe DiseasePompe Disease (Late-onset)+5 more

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 27, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

Developing a Management Approach for Patients With "Late-Onset" Pompe Disease

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)