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

Study Overview
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Brief Summary
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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

Official Title
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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

Conditions
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Pompe Disease (Late-onset)

Intervention / Treatment
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* Biological: Cipaglucosidase Alfa
* Drug: Miglustat


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Exclusion Criteria: 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)

Ages Eligible for Study
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Minimum Age: 0 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221)<br>Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsule | Biological: Cipaglucosidase Alfa<br>* Enzyme Replacement Therapy via intravenous infusion<br>* Other names: ATB200;Drug: Miglustat<br>* Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat(AT2221)<br>* Other names: AT2221;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of treatment-emergent adverse events (TEAEs) from baseline |  | 52 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Assessment of pharmacokinetic parameters | ATB200 and AT2221 concentrations in plasma | 52 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pompe, rhGAA