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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

More Details

NCT ID
NCT04138277
Status
Active, not recruiting
Sponsor
Amicus Therapeutics

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.