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Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease

Primary Purpose

Pompe Disease

Status
Available
Phase
Locations
Study Type
Expanded Access
Intervention
ATB200
AT2221
Sponsored by
Amicus Therapeutics
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Pompe Disease focused on measuring expanded access, compassionate use

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All Sexes

Inclusion Criteria:

  1. Subject must have a diagnosis of Pompe disease based on documentation of one of the following:

    1. deficiency of GAA enzyme
    2. GAA genotyping
  2. Patient does not currently qualify for an Amicus sponsored on-going clinical trial or is declining on currently approved ERT (e.g. Myozyme)
  3. The patient must be willing to receive treatment with ATB200/AT2221 via this program, which includes signing an authorization form for sharing clinical data with Amicus Therapeutics, and its agent Early Access Care LLC.

Exclusion Criteria:

  1. Patient, whether male or female, is planning to conceive a child during the treatment program.
  2. Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221
  3. Patient has a medical or any other extenuating condition or circumstance that may, in the opinion of the investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with or adversely impact protocol requirements.
  4. Patient has received any gene therapy at any time.
  5. Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca), acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb, or Volibo)

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    March 5, 2019
    Last Updated
    July 25, 2023
    Sponsor
    Amicus Therapeutics
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    1. Study Identification

    Unique Protocol Identification Number
    NCT03865836
    Brief Title
    Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease
    Official Title
    Named Patient or Compassionate Use for Treatment Use of ATB200/AT2221 for Patients With Pompe Disease
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    July 2023
    Overall Recruitment Status
    Available
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Amicus Therapeutics

    4. Oversight

    5. Study Description

    Brief Summary
    This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.
    Detailed Description
    This program is being offered on a patient by patient basis and will require company, IRB/IEC and single patient IND approval.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Pompe Disease
    Keywords
    expanded access, compassionate use

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Biological
    Intervention Name(s)
    ATB200
    Intervention Type
    Drug
    Intervention Name(s)
    AT2221

    10. Eligibility

    Sex
    All
    Eligibility Criteria
    Inclusion Criteria: Subject must have a diagnosis of Pompe disease based on documentation of one of the following: deficiency of GAA enzyme GAA genotyping Patient does not currently qualify for an Amicus sponsored on-going clinical trial or is declining on currently approved ERT (e.g. Myozyme) The patient must be willing to receive treatment with ATB200/AT2221 via this program, which includes signing an authorization form for sharing clinical data with Amicus Therapeutics, and its agent Early Access Care LLC. Exclusion Criteria: Patient, whether male or female, is planning to conceive a child during the treatment program. Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221 Patient has a medical or any other extenuating condition or circumstance that may, in the opinion of the investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with or adversely impact protocol requirements. Patient has received any gene therapy at any time. Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca), acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb, or Volibo)
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    For Site
    Phone
    609-662-2000
    Email
    PompeSiteInfo@amicusrx.com
    First Name & Middle Initial & Last Name or Official Title & Degree
    For Patient
    Phone
    609-662-2000
    Email
    patientadvocacy@amicusrx.com

    12. IPD Sharing Statement

    Links:
    URL
    https://clinicaltrials.gov/ct2/show/NCT03729362?term=ATB200-03&rank=1
    Description
    Related Info

    Learn more about this trial

    Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease

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