Tofacitinib - Systemic.
Summary
Tofacitinib is a drug that affects the immune system. The immune system normally fights infection, but in diseases like arthritis, the immune system may be overactive or act incorrectly, leading to inflammation that can result in swelling and pain of the joints, fever, rash, or other symptoms affecting the whole body. This drug is being developed as a disease-modifying antirheumatic drug (DMARD – pronounced DEE-mard) for the treatment of various forms of arthritis. The purpose of this type of treatment is to:
• Control disease activity;
• Improve symptoms;
• Keep up normal, every-day functions and activities;
• Slow down joint damage.
Tofacitinib is currently approved in the United States and more than 45 other countries around the world 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.
1st Phase of Study: Open-label
If your child participates in the study, he/she will receive tofacitinib for the first 12 to 40 weeks. During this period the study doctor will check every 4 weeks (or more often in the first month) if your child’s disease improves. There are 2 dose levels of tofacitinib that will be tested in the Open-label phase: 5 mg and 10 mg tofacitinib. Each dose must be taken two times per day. At the beginning of the study, the 5 mg dose level will be tested first in about 14 to 21 participants. If there are enough participants who have disease improvement, and the 5mg dose is safe, then more patients will be allowed to receive 5 mg tofacitinib twice daily. At the same time, the 10 mg dose level will be tested in about 14 to 21 participants. If enough of these first patients have disease improvement and the 10 mg dose is safe, then more patients will be allowed to receive 10 mg tofacitinib twice daily.
2nd Phase of Study: Double-Blind
At the end of the open-label period, the study doctor will check if your child’s disease has improved enough for him/ her to continue to the next phase of the study. If your child’s disease has not improved enough he/ she will discontinue from the study. If your child’s disease has improved sufficiently, he/ she will be assigned by chance (like the flip of a coin) to receive either tofacitinib or placebo. They will have a 50% (1 in 2) chance of receiving tofacitinib and a 50% (1 in 2) chance of receiving placebo. If your child is assigned to receive tofacitinib, he/she will get the same dose that they received in the open-label phase.
Eligibility
Eligible gender: Male, Female
Eligible ages: 2 to 17
Inclusion criteria:
1. Male or female aged 2 to <18 years.
2. Diagnosed with systemic Juvenile Arthritis(sJIA) and in the opinion of the investigator, have active disease for at least 6 weeks prior to screening. Subjects must have active disease at the time of enrollment, defined as:
a. Documented intermittently spiking temperature >38°C/100.4°F for at least 1 day
due to sJIA in the screening period and within 1 week before the first dose, and
the presence of at least 2 joints with active arthritis at screening and baseline, and
an ESR ˃30 mm/hr at screening.
OR
b. Only after cohort review is completed and enrollment is opened without restrictions at a particular dose level: The presence of at least 5 joints with active arthritis at screening and baseline, and an ESR ˃30 mm/hr at screening.
Exclusion criteria:
1. Previous JIA treatment with tofacitinib.
2. Current symptoms or findings of myocarditis, endocarditis or more than minimal
pericardial effusion associated with sJIA.
3. Current symptoms or findings of more than minimal pleuritis with sJIA.
4. Subjects who have previously failed treatment with more than two biologics..
Participate
Additional information
Contact information
Please contact any Pediatric Rheumatologist or Study Coordinator Dwaraka Veeramreddy @ 403-955-3194
Principal investigator:
Heinrike Schmeling
Clinical trial:
Yes
REB-ID:
REB17-1654