Skip to main content
Update Location

My Location

Update your location to show providers, locations, and services closest to you.

Enter a zip code
Or
Select a campus/region
(352) 733-0111

Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

  • Status
    Accepting Candidates
  • Age
    18 Years - N/A
  • Sexes
    All
  • Healthy Volunteers
    No
I'm interested
Share this study
Facebook
Twitter
LinkedIn

Objective

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.

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.

Details

Full study title A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease
Protocol number OCR29422
ClinicalTrials.gov ID NCT04138277
Phase Phase 3

Eligibility

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.

Lead researcher

Participate in a study

Here are some general steps to consider when participating in a research study:

  1. Step
    1

    Contact the research team

    Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.

    Primary contact

    Barry Byrne
  2. Step
    2

    Get screened to confirm eligibility

    You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.

  3. Step
    3

    Provide your consent to participate

    If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.

  4. Step
    4

    Participate

    If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.