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

Pompe Disease (Late-onset) Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03911505
• Other study ID #: ATB200-04
• Status: Recruiting
• Phase: Phase 3
• Start date: February 13, 2020
• Est. completion date: June 2026

Study information

• Verified date: September 2023
• Source: Amicus Therapeutics
• Contact: For Site
• Phone: 215-921-7600
• Email: PompeSiteInfo[@]amicusrx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 22
• Est. completion date: June 2026
• Est. primary completion date: June 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years to 17 Years

Eligibility

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)

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Pompe Disease (Late-onset)

Intervention

Biological:
• Cipaglucosidase Alfa
Enzyme Replacement Therapy via intravenous infusion

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

Locations

Country	Name	City	State
Australia	Women's and Children's Hospital	North Adelaide	South Australia
Canada	University of Calgary	Calgary	Alberta
Japan	Gunma University Hospital	Gunma
Japan	Tohoku University Hospital	Miyagi
Japan	Izumi City General Hospital	Osaka	Izumi-Shi
Japan	Tokyo Women's Medical University	Tokyo
Taiwan	National Taiwan University Hospital	Taipei
United States	Woodruff Memorial Research Building	Atlanta	Georgia
United States	Duke University Medical Center	Durham	North Carolina
United States	Lysosomal and Rare Disorders Research and Treatment Center, Inc.	Fairfax	Virginia
United States	University of Florida Clinical Research Center	Gainesville	Florida
United States	Infusion Associates	Grand Rapids	Michigan
United States	Wolfson Children's Hospital	Jacksonville	Florida
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	UCSF Benioff Children's Hospital	Oakland	California
United States	Neuromuscular Research Center	Phoenix	Arizona
United States	Mayo Clinic	Rochester	Minnesota
United States	St. Louis Children's Hospital	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Amicus Therapeutics

Countries where clinical trial is conducted

United States,  Australia,  Canada,  Japan,  Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of treatment-emergent adverse events (TEAEs) from baseline		52 weeks
Secondary	Assessment of pharmacokinetic parameters	ATB200 and AT2221 concentrations in plasma	52 weeks