Researchers at Amicus Therapeutics are looking for adolescents (age 12-18) 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 14 months, consisting of infusions every other week. PK assessments at day one, week 26, and week 52 will measure how the drug is absorbed, distributed, and removed from the body. Every three months, a 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 be age 12 to 18
- 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 adolescents
- Must have two 6-minute-walk tests (6MWTs) that meet the following criteria:
- both screening values of 6MWT are ≥ 75 meters
- both screening values of 6MWT are ≤ 90% of the predicted value for healthy adolescents
- the lower value of 6MWT is within 20% of the higher value of 6MWT
- Must meet additional study criteria
Travel support is available.
To find out more information about the study, including a list of active trial sites, you may also visit https://clinicaltrials.gov/ct2/show/NCT03911505.