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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

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ClinicalTrials.gov Identifier: NCT03911505
Recruitment Status : Recruiting
First Posted : April 11, 2019
Last Update Posted : July 18, 2022
Information provided by (Responsible Party):
Amicus Therapeutics

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

Condition or disease Intervention/treatment Phase
Pompe Disease (Late-onset) Biological: Cipaglucosidase Alfa Drug: Miglustat Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 22 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : February 13, 2020
Estimated Primary Completion Date : June 2026
Estimated Study Completion Date : June 2026

Arm Intervention/treatment
Experimental: Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221)
Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsule
Biological: Cipaglucosidase Alfa
Enzyme Replacement Therapy via intravenous infusion
Other Name: ATB200

Drug: Miglustat
Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat(AT2221)
Other Name: AT2221

Primary Outcome Measures :
  1. Incidence of treatment-emergent adverse events (TEAEs) from baseline [ Time Frame: 52 weeks ]

Secondary Outcome Measures :
  1. Assessment of pharmacokinetic parameters [ Time Frame: 52 weeks ]
    ATB200 and AT2221 concentrations in plasma

Information from the National Library of Medicine

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Ages Eligible for Study:   0 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03911505

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Contact: For Site 215-921-7600 PompeSiteInfo@amicusrx.com
Contact: For Patient 215-921-7600 patientadvocacy@amicusrx.com

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United States, Arizona
Neuromuscular Research Center Recruiting
Phoenix, Arizona, United States, 85028
United States, California
UCSF Benioff Children's Hospital Recruiting
Oakland, California, United States, 94609
United States, Florida
University of Florida Clinical Research Center Recruiting
Gainesville, Florida, United States, 32610
United States, Michigan
Infusion Associates Recruiting
Grand Rapids, Michigan, United States, 49525
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
United States, Missouri
St. Louis Children's Hospital Recruiting
Saint Louis, Missouri, United States, 63110
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27710
United States, Virginia
Lysosomal and Rare Disorders Research and Treatment Center, Inc. Recruiting
Fairfax, Virginia, United States, 22030
Australia, South Australia
Women's and Children's Hospital Not yet recruiting
North Adelaide, South Australia, Australia, 5006
Canada, Alberta
University of Calgary Recruiting
Calgary, Alberta, Canada, T3B 6A8
Gunma University Hospital Recruiting
Gunma, Japan, 371-8511
Tohoku University Hospital Recruiting
Miyagi, Japan, 980-8574
Tokyo Women's Medical University Recruiting
Tokyo, Japan, 162-8666
National Taiwan University Hospital Recruiting
Taipei, Taiwan, 100
Sponsors and Collaborators
Amicus Therapeutics
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Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT03911505    
Other Study ID Numbers: ATB200-04
First Posted: April 11, 2019    Key Record Dates
Last Update Posted: July 18, 2022
Last Verified: July 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Amicus Therapeutics:
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
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
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents
Anti-Infective Agents
Glycoside Hydrolase Inhibitors
Hypoglycemic Agents
Physiological Effects of Drugs