Reducing treatment frequency for late-onset Pompe disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests whether adults with late-onset Pompe disease can safely receive their enzyme replacement therapy less often, every 4 weeks instead of every 2 weeks, while maintaining stability. It aims to see if a lower treatment frequency is just as effective and improves quality of life.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 50 years or older.
- You have been on the standard enzyme replacement therapy (alglucosidase alfa) for at least 4 years.
- Your condition has been relatively stable over the past year.
- You can walk at least 150 meters in 6 minutes.
- Your lung function is above a certain level (more than 55% of expected in sitting, and more than 45% in lying down).
- You do not need a wheelchair or breathing support (except for sleep apnea).
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study tests a one-time gene therapy (AB-1009) for adults with late-onset Pompe disease. It aims to see if the treatment is safe and improves muscle and breathing function.
This trial tests a new gene therapy for late-onset Pompe disease. It aims to see if the treatment is safe and effective, and might provide a long-term alternative to enzyme replacement therapy.
This trial tests a new gene therapy for late-onset Pompe disease. The therapy aims to help your body produce the enzyme needed to break down glycogen, which may improve muscle and breathing function.
This trial tests a new enzyme treatment for Pompe disease, a condition that causes muscle weakness. It aims to improve symptoms and quality of life.
This study tests the safety and effects of the enzyme replacement therapy avalglucosidase alfa (Nexviazyme) in children with infantile-onset Pompe disease. It is for children who have had Pompe symptoms since infancy and heart problems at diagnosis.
This trial is a registry, meaning it collects health information from people with confirmed Pompe disease. It may help researchers better understand the disease and improve future treatments.
Hear when a new Pompe Disease (Late-onset) trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.