Status:
ACTIVE_NOT_RECRUITING
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
Lead Sponsor:
Astellas Gene Therapies
Conditions:
Pompe Disease (Late-onset)
Eligibility:
All Genders
16-69 years
Phase:
NA
Brief Summary
Pompe disease is a genetic condition which causes muscle weakness over time. People with Pompe disease have a faulty gene that makes an enzyme called acid alpha-glucosidase (or GAA). This enzyme break...
Detailed Description
No investigational drug will be administered to participants in this study. Blood and urine will be collected as part of the study. The duration of the study is approximately 2 years, participants may...
Eligibility Criteria
Inclusion
- Participant has a documented clinical diagnosis of LOPD.
- Participant is enzyme replacement therapy ERT-naïve (ERT-N) or if the participant is currently taking an approved ERT treatment or is participating in an ERT-interventional study, the ERT must have been received for at least 6 months or more (ERT-experienced \[ERT-E\]).
- Participant is willing and able to comply with study visits and procedures.
- Participant agrees to not start participating in any other clinical study involving an investigational study treatment, including ERT, while participating in this study.
Exclusion
- Participant previously received an AAV-related product (any serotype).
- Participant is currently participating in a Pompe-related interventional study (other than ERT-interventional studies) or has received gene or cell therapy.
- Participant requires any invasive or noninvasive ventilation support while awake and upright (non-invasive support while sleeping with either continuous positive airway pressure (CPAP) or bilevel positive airway pressure (BiPAP) is acceptable for eligibility).
- Participant is unable to ambulate (assistive devices \[e.g., cane or walker\] are acceptable for eligibility).
- Participants who have received any ERT for less than 6 months as of the Baseline visit are not eligible.
Key Trial Info
Start Date :
February 1 2024
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 30 2027
Estimated Enrollment :
119 Patients enrolled
Trial Details
Trial ID
NCT06150820
Start Date
February 1 2024
End Date
June 30 2027
Last Update
January 7 2026
Active Locations (54)
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1
University of California Irvine
Irvine, California, United States, 92868
2
Emory Clinic
Atlanta, Georgia, United States, 30322
3
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
4
University of Michigan
Ann Arbor, Michigan, United States, 48109