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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03911505
Registration number
NCT03911505
Ethics application status
Date submitted
9/04/2019
Date registered
11/04/2019
Date last updated
5/06/2024
Titles & IDs
Public title
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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Scientific 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
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Secondary ID [1]
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ATB200-04
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Pompe Disease (Late-onset)
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Condition category
Condition code
Metabolic and Endocrine
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Metabolic disorders
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Metabolic and Endocrine
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Other metabolic disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - Cipaglucosidase Alfa
Treatment: Drugs - Miglustat
Experimental: Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221) - Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsule
Other interventions: Cipaglucosidase Alfa
Enzyme Replacement Therapy via intravenous infusion
Treatment: Drugs: Miglustat
Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat(AT2221)
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Intervention code [1]
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Other interventions
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Intervention code [2]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incidence of treatment-emergent adverse events (TEAEs) from baseline
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Assessment method [1]
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Timepoint [1]
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52 weeks
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Secondary outcome [1]
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Assessment of pharmacokinetic parameters
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Assessment method [1]
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ATB200 and AT2221 concentrations in plasma
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Timepoint [1]
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52 weeks
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Eligibility
Key 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
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Minimum age
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Years
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Maximum age
17
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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)
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
13/02/2020
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/06/2026
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Actual
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Sample size
Target
22
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
SA
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Recruitment hospital [1]
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Women's and Children's Hospital - North Adelaide
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Recruitment postcode(s) [1]
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5006 - North Adelaide
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Recruitment outside Australia
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United States of America
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Arizona
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Arkansas
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California
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Florida
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United States of America
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Georgia
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United States of America
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Michigan
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United States of America
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Minnesota
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Missouri
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North Carolina
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Virginia
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Canada
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Alberta
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Japan
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State/province [12]
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Izumi-Shi
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Japan
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State/province [13]
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Gunma
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Japan
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State/province [14]
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Miyagi
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Japan
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Tokyo
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Taiwan
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State/province [16]
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Taipei
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Amicus Therapeutics
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Address
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Ethics approval
Ethics application status
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Summary
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
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03911505
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Email
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Contact person for public queries
Name
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For Site
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Address
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Phone
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215-921-7600
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03911505
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