Purpose

This is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.

Condition

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  1. Subject must have completed Study ATB200-03.

Exclusion Criteria

  1. Subject plans to receive gene therapy or participate in another interventional study for Pompe disease.
  2. Subject, if female, is pregnant or breastfeeding.
  3. Subject, whether male or female, is planning to conceive a child during the study.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
ATB200/AT2221
Participants received ATB200 co-administered with AT2221 capsule (Miglustat)
  • Drug: AT2221
    Participants received ATB200 co-administered with AT2221 (Miglustat)
    Other names:
    • Miglustat
  • Biological: ATB200
    Enzyme Replacement Therapy via intravenous infusion

Recruiting Locations

University of Arkansas for Medical Sciences
Little Rock, Arkansas 72205

University of California, Irvine
Irvine, California 92868

University of Florida Clinical Research Center
Gainesville, Florida 32610

Emory Clinic
Atlanta, Georgia 30322

IU Health Neuroscience Center
Indianapolis, Indiana 46202

Billings Clinic
Billings, Montana 59101

Hackensack University Medical Center
Hackensack, New Jersey 07061

Northwell Health
Great Neck, New York 11021

NYU School of Medicine
New York, New York 10017

Duke University Medical Center
Durham, North Carolina 27710

University of Cincinnati Gardner Neuroscience Institute
Cincinnati, Ohio 45219

Cincinnati Children's Hospital
Cincinnati, Ohio 45229

The Ohio State University Wexner Medical Center
Columbus, Ohio 43210

University of Pennsylvania
Philadelphia, Pennsylvania 19104

UPMC Montefiore Clinical and Translational Research Center
Pittsburgh, Pennsylvania 15213

University of Utah
Salt Lake City, Utah 84108

Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax, Virginia 22030

More Details

NCT ID
NCT04138277
Status
Recruiting
Sponsor
Amicus Therapeutics

Study Contact

For Site
609-662-2000
PompeSiteInfo@amicusrx.com

Detailed Description

This is an open label extension study for subjects who completed the ATB200-03 study. The subjects will stay in this study until regulatory approval or marketing authorization and/or commercialization in the participating subject's country.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.