Phase 2 Rare Disease Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• You are 18 years old or older and weigh at least 40 kilograms (about 88 pounds) at the time of signing the consent form.
• You have been diagnosed with late-onset Pompe disease (LOPD), confirmed either by a test showing low levels of a specific enzyme (GAA) or by genetic testing.
• Your lung function (breathing capacity) falls within a certain range — either between 30% and 80% when sitting or standing without a breathing machine, or your breathing capacity drops by at least 10% when lying down compared to sitting/standing, with at least 20% capacity when lying down.
• You are able to complete a 6-minute walk test, can walk at least 75 meters during it, and walk less than 90% of the distance expected for a healthy adult of your age and size.
• You are currently receiving enzyme replacement therapy (ERT) and have been on it for at least 24 months, with no changes to your treatment in the last 6 months.

Who may not be able to join:

• You have a medical condition or other circumstances that could put you at risk or make it difficult to follow the trial requirements (confirm with trial site).
• You have an active infection at the time of screening.
• You have been diagnosed with cancer in the past 5 years, except for certain skin cancers that were fully removed with no sign of spread for at least 3 years.
• You have a current or long-term history of liver disease.
• You have a specific genetic mutation in either the GYG or PYGM gene that causes complete loss of function in both copies of the gene (confirm with trial site).
• You have received another experimental treatment or medication for Pompe disease within approximately the last 30 days or more (depending on the treatment) before the trial starts, or plan to during the study.
• You have previously received gene therapy or a type of RNA-based therapy for Pompe disease.
• You are female and are currently pregnant or breastfeeding.
• You are planning to conceive a child during the study period, regardless of your gender.

Important: Always verify eligibility with the trial site directly before applying.