A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

Launched by ASTELLAS GENE THERAPIES · Nov 21, 2023

Keywords

ClinConnect Summary

This clinical trial is studying people with late-onset Pompe disease, a genetic condition that causes muscle weakness over time. The researchers want to learn about the levels of antibodies against a particular type of virus used in gene therapy, as well as antibodies against the enzyme used in standard treatment for Pompe disease. They are also interested in other substances in the blood called biomarkers, which can give more information about the disease. The trial will involve older teenagers and adults who either have never received enzyme replacement therapy or who have been treated for six months or more.

Participants can expect to visit the study clinic several times over a two-year period, with some visits possibly taking place at home. They will undergo medical examinations and have their vital signs checked, such as blood pressure and heart rate. Blood and urine samples will be collected to measure the levels of antibodies and biomarkers. To qualify for the study, participants must have a confirmed diagnosis of late-onset Pompe disease and must not have previously received certain types of gene therapy or invasive ventilation support. This study will not provide any treatment but aims to gather important information that could help improve future therapies for Pompe disease.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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 Criteria:
• • 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.