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
- Conditions
- Glycogen storage disease type IIPompe disease10028396
- Registration Number
- NL-OMON54552
- Lead Sponsor
- Amicus Therapeutics
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 1
1. Subject must provide signed informed consent prior to any study-related
procedures being performed. If the subject is under 20 years of age, the
subject must provide written informed consent.
2. Subject must have completed Study ATB200-03.
Note: Subjects who were forced to withdraw from Study ATB200-03 for a
logistical reason not related to the efficacy or safety of ATB200/AT2221 (eg,
hospitalization for a car accident or emergency surgery) and which resulted in
several consecutive missed doses may be eligible to participate in this study
upon approval by the Amicus medical monitor.
3. Female subjects of childbearing potential and male subjects must agree to
use medically accepted methods of contraception during the study and for 90
days after the last dose of study drug.
1. Subject plans to receive gene therapy or participate in another
interventional study for Pompe disease.
2. Subject has a hypersensitivity to any excipients in ATB200 or ATB2221 or
medical condition or any other extenuating circumstance that may, in the
opinion of the investigator or medical monitor, pose an undue safety risk to
the subject or may compromise his/her ability to comply with or adversely
impact protocol requirements. This includes clinical depression (as diagnosed
by a psychiatrist or other mental health professional) with uncontrolled or
poorly controlled symptoms.
3. Subject, if female, is pregnant or breastfeeding.
4. Subject, whether male or female, is planning to conceive a child during the
study.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The long-term safety profile of ATB200/AT2221 will be characterized using<br /><br>incidence of treatment-emergent adverse events (TEAEs), serious adverse events<br /><br>(SAEs), and AEs leading to discontinuation of study drug, frequency and<br /><br>severity of immediate and late IARs, and any abnormalities noted in other<br /><br>safety assessments (eg, clinical laboratory tests, ECGs, vital signs).<br /><br>Immunogenicity to ATB200 will also be described.</p><br>
- Secondary Outcome Measures
Name Time Method