Amicus Therapeutics

Expanded Access for ATB200/AT2221 for the Treatment of LOPD

Official Title: 
Expanded Access for ATB200/AT2221 for the Treatment of Late Onset Pompe Disease Patients That do Not Qualify for Pivotal Study ATB200-03
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.

EA Type
Individual Patients
Pompe Disease

Biological: ATB200

Drug: AT2221

Eligibility Criteria: 

Inclusion Criteria:

1. Subject must have a diagnosis of LOPD based on documentation of one of the following: 1. deficiency of GAA enzyme 2. GAA genotyping

2. Subject must provide signed informed consent prior to any study-related procedures being performed.

Exclusion Criteria:

1. Subject is eligible for enrollment in an ongoing clinical trial using ATB200/AT2221.

2. Subject, if female, is pregnant or breastfeeding at screening.

3. Subject, whether male or female, is planning to conceive a child during the study.

Eligibility Gender: 
Eligibility Age: 
Minimum: 1 Year

For Site

For Patient

Amicus Therapeutics
EA Number: 
Expanded Access, Compassionate Use
MeSH Terms: 
Glycogen Storage Disease Type II
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