Clinical Trial Alert: Amicus Therapeutics Seeks Participants for a Phase 3 Late-Onset Pompe Disease Study

Researchers at Amicus Therapeutics are looking for participants (age 0-18) 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 receive the study drugs ATB200 (cipaglucosidase alfa) and AT2221 (miglustat) for 52 weeks during the treatment period. At the end of the study, participants will be given the option of enrolling into the long-term extension of the study.

The study drugs are administered to the participants every other week. The AT2221 is administered orally followed by an intravenous (IV) infusion of the ATB200. 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, blood tests, 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 0 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

·        Participants 12 to 18 years old must weigh between 25kg and 115 kg

·        Participants 12 to 18 years old must have a sitting forced vital capacity (FVC) ≥ 30% of the predicted value for healthy adolescents

·        Participants 12 to 18 years old must be able to walk > 75 meters on the 6-minute-walk test. 

·        Participants aged  5 to < 12 years old must be able to walk > 40 meters on the 6-minute walk test.

·        Must meet additional study criteria

Travel support is available.

Home infusion is available after 6 months of participation in the study.

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 or https://antidote.me/match/study-page/nct/NCT03911505

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