General
What does investigational mean?
An “investigational” drug is a drug that is still being tested and has not been approved for the treatment of your child’s disease or age group.
The study drug being given in the AspireToo Study is already approved in 45 countries, including the European Union, the UK, Switzerland, Israel, Australia, Canada, South Korea, Japan, and the United States (US) for the treatment of adults and in some countries for children and adolescents aged 12 years and older with Fabry disease. However, as it has not been studied or approved for use in Fabry disease patients ages 2 to < 12, it is considered an investigational drug for these patients.
If my child is turning 12 years soon, are they still eligible?
Children who are aged 11 years must have birthdays more than 30 days after time of enrollment.
Who is sponsoring the study?
The pharmaceutical company Amicus Therapeutics is sponsoring the study. They provide the funds for the study doctor and study clinic to run this study. Doctors at research centers around the world have partnered with Amicus to conduct this study.
Learn more about Amicus’ commitment to finding treatment for devastating rare diseases at
https:/amicusrx.com/
Being in the Study
How many times would my child have to go to the study clinic?
There will be at least 8 scheduled visits to the study clinic if your child is enrolled in the study. There will be additional visits if screening takes longer than 1 visit or if they join the open-label extension.
Will it cost anything to be in the study?
The study drug, study-related tests, procedures, and visits required by the study are provided at no cost to you/your child. The costs of standard medical care that are not part of the study will be billed to you and/or your insurance company in the usual way.
Will my child be paid for being in the study?
Reimbursement for time and expenses related to participation may or may not be available at your study site. Examples of related expenses are modest overnight accommodations, mileage reimbursement, cost of transport (e.g., flights, train, taxi fares) and/or meals required during long travel periods or overnight stays. Please ask the study staff if reimbursement will be available to you.
What if I decide to join the study and then change my mind?
If you/your child decide to take part in the study and later change your mind, you may withdraw your consent and discontinue your child’s participation at any time. Your decision will not affect your child’s ability to receive medical care for their disease and they will not lose any benefits to which they would otherwise be entitled.
Getting Started
What is the first step I should take?
Contact a study staff member to learn more about the AspireToo Study at xxx-xxx-xxxx
Ready to get started?
Contact a study staff member to learn more about the AspireToo Study at: