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

Last updated: September 16, 2024
Sponsor: Amicus Therapeutics
Overall Status: Active - Recruiting

Phase

3

Condition

Pompe Disease

Treatment

Miglustat

Cipaglucosidase Alfa

Clinical Study ID

NCT03911505
ATB200-04
  • Ages < 17
  • All Genders

Study 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

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Male or female subjects (ERT-naïve [have never received a dose of rhGAA] orERT-experienced [have received rhGAA every 2 weeks for at least 6 months immediatelybefore enrollment, and if ERT dosage has been modified, must have been on themodified dosage for at least 3 months before enrollment]) diagnosed with LOPD whoare aged 12 to <18 years at screening (Cohort 1 only) or aged 0 months to < 12 yearsat 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 studyprotocol

  4. If of reproductive potential and if sexually active, female and male subjects agreeto use a highly effective method of contraception throughout the duration of thestudy 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 forhealthy 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, orsubject (aged ≥ 5 to < 12 years; Cohort 2) performs one 6MWT (≥ 40 meters) atscreening that is valid, as determined by the clinical evaluator

Exclusion

Exclusion Criteria:

  1. Subject has received any investigational/experimental drug, oral anabolic steroid orderivative, biologic, or device within 30 days or 5 half-lives of the therapy ortreatment, whichever is longer, before screening

  2. Subject has received treatment with prohibited medications within 30 days ofscreening

  3. Subject has received any gene therapy at any time

  4. Subject has any intercurrent illness or condition at screening or baseline that maypreclude the subject from fulfilling the protocol requirements or suggests to theinvestigator and/or the medical monitor that the potential subject may have anunacceptable 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 withclassic IOPD

  9. In the opinion of the investigator, the parent or legally authorized representativeis unlikely or unable to comply with the study requirements

  10. Subject has any prior history of illness or condition known to affect motorfunction, such as, but not limited to, Guillain-Barre syndrome, cerebral palsy, etc

  11. Subject who is diagnosed with Pompe disease via newborn screening and isasymptomatic (ie, showing no signs and symptoms of Pompe disease (Cohort 2 Only)

Study Design

Total Participants: 22
Treatment Group(s): 2
Primary Treatment: Miglustat
Phase: 3
Study Start date:
February 13, 2020
Estimated Completion Date:
June 30, 2026

Connect with a study center

  • Women's and Children's Hospital

    North Adelaide, South Australia 5006
    Australia

    Active - Recruiting

  • University of Calgary

    Calgary, Alberta T3B 6A8
    Canada

    Completed

  • SphinCS GmbH Clinical Science for LSD

    Hochheim, Hessen 65239
    Germany

    Active - Recruiting

  • San Gerardo Hospital

    Monza, 20900
    Italy

    Active - Recruiting

  • Izumi City General Hospital

    Osaka, Izumi-Shi 594-0073
    Japan

    Active - Recruiting

  • Gunma University Hospital

    Gunma, 371-8511
    Japan

    Active - Recruiting

  • Tohoku University Hospital

    Miyagi, 980-8574
    Japan

    Active - Recruiting

  • Tokyo Women's Medical University

    Tokyo, 162-8666
    Japan

    Active - Recruiting

  • National Taiwan University Hospital

    Taipei, 100
    Taiwan

    Site Not Available

  • Neuromuscular Research Center

    Phoenix, Arizona 85028
    United States

    Site Not Available

  • Arkansas Children's Hospital

    Little Rock, Arkansas 72202
    United States

    Site Not Available

  • UCSF Benioff Children's Hospital

    Oakland, California 94609
    United States

    Site Not Available

  • University of Florida Clinical Research Center

    Gainesville, Florida 32610
    United States

    Active - Recruiting

  • Wolfson Children's Hospital

    Jacksonville, Florida 32207
    United States

    Active - Recruiting

  • Woodruff Memorial Research Building

    Atlanta, Georgia 30322
    United States

    Site Not Available

  • Infusion Associates

    Grand Rapids, Michigan 49525
    United States

    Site Not Available

  • Mayo Clinic

    Rochester, Minnesota 55905
    United States

    Site Not Available

  • St. Louis Children's Hospital

    Saint Louis, Missouri 63110
    United States

    Active - Recruiting

  • Duke University Medical Center

    Durham, North Carolina 27710
    United States

    Active - Recruiting

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
    United States

    Active - Recruiting

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania 15224
    United States

    Active - Recruiting

  • University of Utah, Clinical and Translational Sciences Institute

    Salt Lake City, Utah 84108
    United States

    Active - Recruiting

  • Lysosomal and Rare Disorders Research and Treatment Center, Inc.

    Fairfax, Virginia 22030
    United States

    Active - Recruiting

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