Why are we doing this research?
Cincinnati Children’s is conducting a research study, sometimes known as a clinical trial or clinical study, to learn more about the effects (good and bad) of an investigational study drug when used in children with granulomatosis with polyangiitis (GPA) (formerly called Wegener’s granulomatosis) or microscopic polyangiitis (MPA).
The investigational study drug has been approved in some countries for the treatment of adults with GPA or MPA. However, the drug has not yet been approved by the U.S. Food and Drug Administration (FDA) for the use in children and teens, and is therefore considered investigational for this study.
What will happen in the study?
If you (if you are a participant who is 18) or your child qualifies, and you decide you want to take part in this study, you or your child will come to Cincinnati Children’s at least 16 times over the next 18 months. Then, you or your child will return for follow-up visits every 3 months until the study has ended. This follow-up period could last up to 3 ½ years or more.
Before you or your child can start the study, we will ask you to come in for a screening visit. During this visit, we will conduct some tests, exams and/or procedures to see if you or your child will be able to participate in the study. However, if you or your child has had some of these procedures recently, they may not need to be repeated.
If you or your child is eligible to take part in the study, some of the tests, exams and/or procedures will be repeated at every clinic visit, while others will take place only during certain visits.
These are the tests, exams and procedures that may happen during the screening and/or study visit(s):
- Review of you or your child’s current and previous GPA or MPA (the doctor will assess the occurrence of disease flares)
- You or your child will be asked questions about your daily activities (parents/guardians will be asked these questions of the behalf of younger children)
- Review of you or your child’s medical history
- Review of current medications and past medications
- Review of any problems you or your child have had
- Vital signs (height, weight, Blood pressure, heart rate and temperature)
- ECG (a test that monitors heart activity)
- Chest X-ray and maybe a CT scan (this will be done if you or your child decides to withdraw from the study)*
- Pregnancy test (if you or your child is a female, sexually active and at an age where it is possible to become pregnant)
- The Child Health Questionnaire or CHQ (this is designed to capture the physical, emotional, and social components of health status of children older than 5 years of age)
- The Childhood Health Assessment Questionnaire or CHAQ (this evaluates the difficulty in performing daily functions, use of special aids and assistance from other people)
Before starting the study, you or your child will receive at least 3 intravenous infusion doses of a steroid. This means that the medicine is put directly into the bloodstream through a needle into a vein in the arm.
On day 1, before receiving the study drug, you or you child will be given pre-infusion medicines that can be taken orally (by mouth) or be given as an infusion. You or your child will also have routine exams and blood tests. Then, you or you child will be given the study drug by infusion. This normally takes 4 to 5 hours.
You or your child will be given the study drug (and pre-infusion medications) at Cincinnati Children’s once a week (every 7 days) for the next 4 weeks.
Routine exams and blood tests will be conducted at months 1, 2, 4, 6, 9, 12, 15 and 18. You or your child will also continue taking daily oral prednisone.
Parents interested in having their child participate will be given a consent form that thoroughly explains all of the details of the study. A member of the study staff will review the consent form with you and will be sure that all of your questions are answered.