Single Patient Use of Tocilizumab in Systemic Onset Juvenile Idiopathic Arthritis
Information source: Tufts Medical Center
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Arthritis, Juvenile Rheumatoid; Still's Disease, Juvenile Onset
Intervention: tocilizumab (Biological)
Phase: N/A
Status: Terminated
Sponsored by: Tufts Medical Center Official(s) and/or principal investigator(s): Marc D Natter, MD, Principal Investigator, Affiliation: Tufts Medical Center
Summary
The purpose of this study is to see if tocilizumab is safe and effective for treating
systemic onset Juvenile Idiopathic Arthritis (soJIA). Another purpose is to see if
tocilizumab helps reduce the amount of steroids (prednisone) needed to control symptoms of
soJIA.
Clinical Details
Official title: Single Patient Use of Tocilizumab for Treatment of Steroid Dependent, Active Systemic Onset Juvenile Idiopathic Arthritis
Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Efficacy of tocilizumab as defined by presence of an equal to or greater than 30% improvement in JIA core set (i.e. ACR JIA30 response)Efficacy of tocilizumab as defined by reduction of oral prednisone dose by at least 20%, or to less than 0.5mg/kg/day, whichever is of lesser daily dose, while maintaining an ACR JIA30 response To evaluate the safety of tocilizumab administration in this subject
Secondary outcome: To assess normalization of laboratory parameters of active disease, specifically C-reactive protein, hemoglobin, platelets, white blood cell count, ferritin, immunoglobulins.To assess sustained clinical response to tocilizumab, including active joint count, joints with limited range of motion, and absence of fever or rash.
Detailed description:
Systemic onset Juvenile Idiopathic Arthritis (soJIA) is a type of arthritis (inflammation of
the joints) that occurs with other symptoms including fever, swollen lymph nodes (glands),
rash, and body aches. Because soJIA can be difficult to treat, children with soJIA can have
severe problems from long-term use of steroids (prednisone). These problems include low bone
density (weak bones), fractures, failure to grow properly, and large weight gain. The
arthritis that occurs in soJIA often causes damage to many joints. This can make it hard to
move around or do basic tasks like dressing. Also, a life-threatening illness called
Macrophage Activation Syndrome (MAS) can occur when starting, stopping, or changing drugs
that are used to treat soJIA.
SoJIA can be hard to treat and many children with soJIA do not respond to drugs that work
for other kinds of arthritis. Research doctors have studied a chemical signal called IL-6
that the body uses to manage inflammation. This signal has been found to be very high in
patients with active soJIA. A drug called tocilizumab (TCZ) has been designed to block IL-6.
For about 6 years, TCZ has been tested in Japan for treating soJIA. It is now being tested
in studies in the United States. These studies can have very strict rules for enrolling
patients. This trial is a single-patient research study for a subject who otherwise does not
meet the rules for enrollment in ongoing trials.
Eligibility
Minimum age: 2 Years.
Maximum age: 16 Years.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- Systemic Juvenile Idiopathic Arthritis according to ILAR criteria (2001)
- Duration of disease ≥ 6 months since onset
- Presence of active disease as determined by the presence of at least 5 active joints,
OR at least 2 active joints if receiving prednisone at a dose > 0. 2 mg/kg/day or > 10
mg/day (whichever is less)
- Incomplete prior response to methotrexate treatment for at least 3 months at a
minimum dose of 15 mg/M2/week, or intolerance to methotrexate
- Discontinued treatment with other biologics prior to first tocilizumab infusion, for
approximately two pharmacokinetic half-lives as per specific biologic (e. g. 48 hours
for anakinra, 7 days for etanercept)
- Not receiving corticosteroids, OR taking oral corticosteroids and the dose has
remained stable for 1 week prior to the first tocilizumab infusion at ≤ 2 mg/kg/day
prednisone or prednisolone and no more than 80 mg/day
Exclusion Criteria:
- Concomitant administration of biologic therapies
- Serum creatinine >1. 5 ULN (upper limits normal)
- AST or ALT > 1. 5 ULN
- Total bilirubin > 1. 3 mg/dL
- Platelet count < LLN (lower limits normal)
- Hemoglobin < 6. 0 g/dL
- WBC count < 5,000/mm3
- Neutrophil count < 2,000/ mm3
- Fibrinogen < LLN
Locations and Contacts
Tufts Medical Center/Floating Hospital for Children, Boston, Massachusetts 02111, United States
Additional Information
A Study of Tocilizumab in Patients With Active Systemic Juvenile Idiopathic Arthritis
Related publications: Yokota S, Imagawa T, Mori M, Miyamae T, Aihara Y, Takei S, Iwata N, Umebayashi H, Murata T, Miyoshi M, Tomiita M, Nishimoto N, Kishimoto T. Efficacy and safety of tocilizumab in patients with systemic-onset juvenile idiopathic arthritis: a randomised, double-blind, placebo-controlled, withdrawal phase III trial. Lancet. 2008 Mar 22;371(9617):998-1006. doi: 10.1016/S0140-6736(08)60454-7.
Starting date: March 2009
Last updated: December 6, 2012
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