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

Running, not enrolling · Phase 3

Conditions studied: Pompe Disease (Late-onset)

In brief

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

Key facts

Study ID: NCT03911505
Run by: Amicus Therapeutics
People needed: 21
Starts: 2020-02-13
Expected to finish: 2026-06-01
Last updated by the study team: 2025-10-27

Who can join

Age: any, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

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)

Where it is running

University of Florida Clinical Research Center — Gainesville, Florida, United States
Wolfson Children's Hospital — Jacksonville, Florida, United States
Woodruff Memorial Research Building — Atlanta, Georgia, United States
St. Louis Children's Hospital — St Louis, Missouri, United States
Duke University Medical Center — Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
University of Utah, Clinical and Translational Sciences Institute — Salt Lake City, Utah, United States
Lysosomal and Rare Disorders Research and Treatment Center, Inc. — Fairfax, Virginia, United States
Women's and Children's Hospital — North Adelaide, South Australia, Australia
University of Calgary — Calgary, Alberta, Canada
SphinCS GmbH Clinical Science for LSD — Hochheim am Main, Hesse, Germany
San Gerardo Hospital — Monza, Italy
Izumi City General Hospital — Osaka, Izumi-Shi, Japan
Gunma University Hospital — Gunma, Japan
Tohoku University Hospital — Miyagi, Japan
Tokyo Women's Medical University — Tokyo, Japan

Full record on ClinicalTrials.gov

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