Researchers at Amicus Therapeutics are looking for adults with late-onset Pompe disease (LOPD) to participate in a Phase 3 study. The goal of the study is to evaluate the safety and efficacy of ATB200/AT2221, which researchers hope may improve muscle function and respiratory function compared to the standard of care.
Participants will be randomized to receive either ATB200 and AT2221 or standard of care and placebo. At the end of the study, subjects will be given the option of enrolling into a long-term extension study.
The total study duration for each patient will be about one year, with 26 clinic visits (infusion every other week), and eight study evaluation visits. Each study evaluation visit will consist of a physical exam and blood tests along with muscle strength, motor function, and pulmonary function tests. Additionally, participants will complete questionnaires to assess changes in quality of life.
In order to be eligible to participate, candidates must meet the criteria below, along with additional study criteria:
- Must be an adult age 18 or older
- Must have a diagnosis of LOPD based on documentation of one of the following:
- deficiency of GAA enzyme
- GAA genotyping
- Must either be currently receiving treatment with enzyme replacement therapy (ERT) or have never received ERT
- Must have a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy adults
- Must have two 6-minute-walk tests (6MWTs) that meet the following criteria:
- both screening values of 6MWD are ≥ 75 meters
- both screening values of 6MWD are ≤ 90% of the predicted value for healthy adults
- the lower value of 6MWD is within 20% of the higher value of 6MWD
Travel support for the participant and one travel companion for each visit is available.
To learn more or to inquire about participation, contact firstname.lastname@example.org.
To read about the results of the Phase 1/2 trial, click here.