General
How can I find out about eligibility?
Talk to the study doctor and staff. They will be able to discuss a child’s eligibility. Note that in order for any Screening tests and procedures to take place, consent forms must be signed by the child’s parent/legal guardian and the child (age 7 years and above).
Is there a cost to participate?
No. Study-related treatment, medicine, tests, and doctor visits are provided at no cost. The costs of standard medical care that are not part of the study will be paid for in the usual way (billed to the child’s insurance company or paid out of pocket).
Will study participants be paid?
Yes. A payment for each visit may be provided as reimbursement for the time required to be part of the study.
What kind of financial help is available for study participants?
Expenses related to being in the study, such as modest overnight accommodations, transportation (for example mileage on your car, flights, train, and taxi fares), and/or meals required during long travel periods or overnight stays may be reimbursed.
Ask the study staff to explain what financial help is available at your study location.
Who is sponsoring this study?
The study is sponsored by Amicus Therapeutics. Amicus is a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering high-quality medicines for people living with rare metabolic diseases.
Treatment
What study medicines will participants get?
There are 2 study medicines. One study medicine is given by infusion. The other is given by mouth or into a feeding tube. Both are given on the same day once per week.
These study medicines can only be given to children with Pompe disease as part of a clinical study. The combination of the study medicines has been approved for adults with late-onset Pompe disease in the United States, Europe, and the United Kingdom.
Have the study medicines been given to people before?
Yes. As of March 19, 2024, a total of 151 adult patients with Pompe disease and 16 children with Pompe disease have received the study medicines in clinical studies.
Are the study medicines safe?
All medicines have side effects. Learning about side effects in children is one goal of this study. The study doctor will explain what is known about the safety of the study medicines. Ask any questions you have.
Will study participants be able to continue taking the study medicines after the study ends?
There is no guarantee that the study medicines will be available by prescription for children with Pompe disease after the study ends. The child’s doctor will determine what therapy is most suitable at that time.