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

• Register: ClinicalTrials.gov
• Last refreshed on: 26 September 2023
• Main ID: NCT03911505
• Date of registration: 09/04/2019
• Prospective Registration: Yes
• Primary sponsor: Amicus Therapeutics
• 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
• 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
• Date of first enrolment: February 13, 2020
• Target sample size: 22
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03911505
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Canada, Japan, Taiwan, United States

Contacts

• Name: For Site

Key inclusion & exclusion criteria

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)



Age minimum: 0 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Pompe Disease (Late-onset)

Intervention(s)

Biological: Cipaglucosidase Alfa

Drug: Miglustat

Primary Outcome(s)

Incidence of treatment-emergent adverse events (TEAEs) from baseline [Time Frame: 52 weeks]

Secondary Outcome(s)

Assessment of pharmacokinetic parameters [Time Frame: 52 weeks]

Secondary ID(s)

ATB200-04

Source(s) of Monetary Support

Please refer to primary and secondary sponsors