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

NCT03911505 | Active Not Recruiting Phase 3 FDA Drug

• 1 other identifier: ATB200-04
• Study Type: interventional
• Target: 21
• Locations: 6 countries
• Sites: 17

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)

Keywords

Pompe; rhGAA

Outcome Measures

Primary Outcomes (1)

• Incidence of treatment-emergent adverse events (TEAEs) from baseline

  52 weeks

Secondary Outcomes (1)

• Assessment of pharmacokinetic parameters

  52 weeks

Study Arms (1)

Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221)

EXPERIMENTAL

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

Biological: Cipaglucosidase Alfa; Drug: Miglustat

Interventions

Cipaglucosidase Alfa BIOLOGICAL

Enzyme Replacement Therapy via intravenous infusion

Also known as: ATB200

Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221)

Miglustat DRUG

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

Also known as: AT2221

Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221)

Eligibility Criteria

• Age: 0 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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)
• Subject weighs ≤ 115 kg. (Cohort 1 Only)
• Subject must have a diagnosis of LOPD based on documentation as defined in study protocol
• 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
• Subject has a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy Adolescents at screening (Cohort 1 only)
• 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

You may not qualify if:

• 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
• Subject has received treatment with prohibited medications within 30 days of screening
• Subject has received any gene therapy at any time
• 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
• Subject has a hypersensitivity to any of the excipients in ATB200, approved rhGAA, or AT2221
• Female subject is pregnant or breast-feeding at screening
• Subject requires the use of ventilation support for \> 6 hours per day while awake
• Subject has evidence of moderate to severe hypertrophic cardiomyopathy aligning with classic IOPD
• In the opinion of the investigator, the parent or legally authorized representative is unlikely or unable to comply with the study requirements
• 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
• 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)

Sponsors & Collaborators

• Amicus Therapeutics: lead

Study Sites (17)

University of Florida Clinical Research Center

Gainesville, Florida, 32610, United States

Location

Wolfson Children's Hospital

Jacksonville, Florida, 32207, United States

Location

Woodruff Memorial Research Building

Atlanta, Georgia, 30322, United States

Location

St. Louis Children's Hospital

St Louis, Missouri, 63110, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

University of Utah, Clinical and Translational Sciences Institute

Salt Lake City, Utah, 84108, United States

Location

Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Fairfax, Virginia, 22030, United States

Location

Women's and Children's Hospital

North Adelaide, South Australia, 5006, Australia

Location

University of Calgary

Calgary, Alberta, T3B 6A8, Canada

Location

SphinCS GmbH Clinical Science for LSD

Hochheim am Main, Hesse, 65239, Germany

Location

San Gerardo Hospital

Monza, 20900, Italy

Location

Izumi City General Hospital

Osaka, Izumi-Shi, 594-0073, Japan

Location

Gunma University Hospital

Gunma, 371-8511, Japan

Location

Tohoku University Hospital

Miyagi, 980-8574, Japan

Location

Tokyo Women's Medical University

Tokyo, 162-8666, Japan

Location

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Interventions

miglustat

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: April 9, 2019
• First Posted: April 11, 2019
• Study Start: February 13, 2020
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: October 27, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1); AU (1); US (9); DE (1); IT (1); JP (4)