Clinical Trials List
Protocol NumberATB200-04
NCT Number(ClinicalTrials.gov Identfier)NCT03911505
2019-10-01 - 2021-12-31
Phase III
Recruiting1
ICD-10E74.00
Glycogen storage disease, unspecified
ICD-10E74.01
von Gierke disease
ICD-10E74.02
Pompe disease
ICD-10E74.03
Cori disease
ICD-10E74.04
McArdle disease
ICD-10E74.09
Other glycogen storage disease
ICD-10E74.4
Disorders of pyruvate metabolism and gluconeogenesis
ICD-9271.0
Glycogenosis
An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of ATB200/AT2221 in Pediatric Subjects Aged 0 to < 18 Years With Pompe Disease
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Trial Applicant
MEDPACE TAIWAN LIMITED
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Sponsor
Amicus Therapeutics, Inc.
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
Co-Principal Investigator
- NI-CHUNG LEE Division of General Internal Medicine
- 陳俊安 Division of General Internal Medicine
- WUH-LIANG HWU Division of General Internal Medicine
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Pompe Disease
Objectives
Primary Objective
The primary objectives of the study are as follows:
•to characterize the PK of ATB200 and AT2221 and validate the pediatric doses determined by extrapolation of adult exposure to the children for the combination therapy
•to evaluate the safety and tolerability of ATB200/AT2221 co-administration
Secondary Objectives
The secondary objectives of the study are as follows:
•to evaluate the effect of ATB200/AT2221 on efficacy
•to evaluate the effect of ATB200/AT2221 on pharmacodynamics (PD)
•to evaluate the effect of ATB200 exposure on efficacy and PD endpoints (exposure-response relationships)
•to evaluate the effect of anti-rhGAA antibodies on ATB200 exposure
Test Drug
ATB200、AT2221
Active Ingredient
N-butyl-deoxynojirimycin (Miglustat)
recombinant human acid α-glucosidase (rhGAA)
recombinant human acid α-glucosidase (rhGAA)
Dosage Form
Lyophilized powder
Hard gelatin capsule
Hard gelatin capsule
Dosage
105
65
65
Endpoints
Primary Outcome Measures :
Incidence of treatment-emergent adverse events (TEAEs) from baseline [ Time Frame: 52 weeks ]
Secondary Outcome Measures :
Assessment of pharmacokinetic parameters [ Time Frame: 52 weeks ]
ATB200 and AT2221 concentrations in plasma
Incidence of treatment-emergent adverse events (TEAEs) from baseline [ Time Frame: 52 weeks ]
Secondary Outcome Measures :
Assessment of pharmacokinetic parameters [ Time Frame: 52 weeks ]
ATB200 and AT2221 concentrations in plasma
Inclution Criteria
1.Male or female subjects (ERT-naïve [have never received a dose of alglucosidase alfa] or ERT-experienced [have received at least 1 dose of alglucosidase alfa]), diagnosed with late-onset Pompe disease who are aged 12 to < 18 years at screening
2.Subject’s parent or legally authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable based on site and regulatory regulations
3.Subject must have a diagnosis of LOPD based on documentation of one of the following:
a.deficiency of GAA enzyme
b.GAA genotyping
4.If of reproductive potential, both male and female 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 ATB200/AT2221 (see Section 8.4 for more details)
5.Subject has a sitting FVC ≥ 30% of the predicted value for healthy adolescents (Global Lung Function Initiative [GLI]) at screening
6.Subject performs two 6MWTs at screening that are valid, as determined by the clinical evaluator, and that meet all of the following criteria:
a.both screening values of 6MWD are ≥ 75 meters
b.both screening values of 6MWD are ≤ 90% of the predicted value for healthy adolescents c.the lower value of 6MWD is within 20% of the higher value of 6MWD
2.Subject’s parent or legally authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable based on site and regulatory regulations
3.Subject must have a diagnosis of LOPD based on documentation of one of the following:
a.deficiency of GAA enzyme
b.GAA genotyping
4.If of reproductive potential, both male and female 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 ATB200/AT2221 (see Section 8.4 for more details)
5.Subject has a sitting FVC ≥ 30% of the predicted value for healthy adolescents (Global Lung Function Initiative [GLI]) at screening
6.Subject performs two 6MWTs at screening that are valid, as determined by the clinical evaluator, and that meet all of the following criteria:
a.both screening values of 6MWD are ≥ 75 meters
b.both screening values of 6MWD are ≤ 90% of the predicted value for healthy adolescents c.the lower value of 6MWD is within 20% of the higher value of 6MWD
Exclusion Criteria
1.Subject has received any investigational/experimental drug, 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 (see Section 9.2.2) 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, alglucosidase alfa, 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
2.Subject has received treatment with prohibited medications (see Section 9.2.2) 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, alglucosidase alfa, 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
The Estimated Number of Participants
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Taiwan
3 participants
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Global
12 participants
