Why are we doing this research?
Cincinnati Children’s is conducting a research study, sometimes known as a clinical trial or clinical study, to compare the effectiveness of an investigational drug called tofacitinib over placebo for treatment of signs and symptoms of Juvenile Idiopathic Arthritis (JIA). A placebo looks like the study drug but does not contain any active ingredient.
Tofacitinib is currently approved by the United States Food and Drug Administration (FDA) for use in adults for the treatment of rheumatoid arthritis but is not currently approved for use in children. Therefore, the use of tofacitinib in this study is investigational.
What will happen in the study?
If your child qualifies and you decide you want them to participate, your child will be in this study for about 44 weeks and have about 14 study visits. However, your child may be asked to stop participation after the 18 week run-in period if he or she does not respond adequately to tofacitinib or experiences a single episode of disease flare.
These are the tests, procedures and assessments that will be completed during one or more study visits, including the screening:
- Review of informed consent (and assent, when required)
- Review of family medical history
- Review of your child’s past and present medical history (including history of arthritis and vaccinations)
- Review of your child’s past and present medication history
- Referral to an eye doctor to assess if your child has any inflammation in the middle layer of the eye, called uveitis (this may be done up to 3 times during the study)
- Physical examination and vital sign measurements (including weight, height, blood pressure, pulse rate and temperature)
- Collection of blood samples for laboratory tests
- Collection of urine samples for laboratory tests
- Urine or blood pregnancy test (if your child is a female and medically capable of becoming pregnant)
- Tuberculosis (TB) skin test (only if the blood sample result is unclear)
- Chest X-ray (may be required to aid in TB status determination)
- Examination of your child’s joints
- Measurements of your child’s arthritis symptoms
- Questions from staff as to how much pain your child is feeling and his or her ability to function
- Questionnaires relating to your child’s condition
- Assessments to see if your child has had a disease flare
- Tanner stage assessment (assessment of your child’s physical development)
- Discussion of any symptoms (side effects or other problems) between study visits while your child is on the study drug
- Additional assessments of tenderness and ratings or your child’s overall and nighttime back pain (if your child has enthesitis-related arthritis or ERA)
- Additional assessments for body surface area (BSA) affected by psoriasis and an assessment of overall psoriasis (if your child has psoriatic arthritis or PsA)
You and your child will have 14 visits to Cincinnati Children’s about every 2 to 4 weeks during the study.
Your child will first have a Screening Visit to determine whether or not he or she meets all of the requirements to take part in this research study.
If your child is eligible to continue after the Screening Visit, he or she will have the first study visit, which will occur not more than 30 days after the Screening Visit.
During the first study visit, your child will receive the investigational study drug, tofacitinib, in either a liquid or tablet (pill) form, depending on his or her weight. Your child will take tofacitinib twice-a-day about 12 hours apart (in the morning and evening), for 18 weeks. This is called the run-in phase of the study.
After the run-in phase, your child’s disease status will be evaluated by the study doctor. The study doctor will determine if your child can continue on in the next phase of the study. If your child is unable to participate in the next phase, the study doctor will choose other appropriate treatments outside of the study.
If your child is able to continue in the study, he or she will be randomly assigned by chance (like flipping a coin) to receive either tofacitinib or placebo for the next 26 weeks.
Your child will have a 50% (1 in 2) chance of receiving tofacitinib and a 50% (1 in 2) chance of receiving placebo. Neither you nor the doctor can choose the group your child will be in. Also, neither you, your child, the doctor, nor the study team will know which of the study drugs your child is receiving. However, the study team can quickly learn this information in case of an emergency.
During the 26 weeks, your child will take their assigned study drug in either a liquid or tablet (pill) form, depending on his or her weight, twice-a-day about 12 hours apart (in the morning and evening).
Parents/guardians 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.
What are the good things that can happen from this research?
Your child may or may not receive any direct benefit from participating in this research study. However, your child’s participation in this study may benefit people with JIA in the future.