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
About this trial
This is an expanded access trial for Pompe Disease focused on measuring expanded access, compassionate use
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)
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
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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