Active Juvenile Idiopathic Arthritis (JIA) Compassionate Use
Condition: Juvenile Idiopathic Arthritis
MeSH terms: Arthritis; Arthritis, Juvenile
Intervention: Adalimumab
Intervention description: Adalimumab solution for injection for subcutaneous use.
Intervention MeSH terms: Adalimumab
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 32.0
Sex: All
Minimum age: 2 Years
Maximum age: 4 Years
Healthy volunteers: No
Keywords: juvenile idiopathic arthritis; compassionate use; open label

Summary:

      The main objective of this study is to evaluate the safety of adalimumab in patients 2 to < 4
      years of age or ≥ 4 years of age weighing < 15 kg, with moderately to severely active
      polyarticular juvenile idiopathic arthritis (JIA) or polyarticular course JIA.
    

Eligibility criteria:

        Inclusion Criteria:

          1. A parent or guardian has voluntarily signed and dated an informed consent form,
             approved by an Institutional Review Board (IRB)/Independent Ethics Committee (IEC),
             after the nature of the study has been explained and the subject's parent or legal
             guardian has had the opportunity to ask questions. The informed consent must be signed
             before any study-specific procedures are performed or before any medication is
             discontinued for the purpose of this study. The parent must also be willing to comply
             with all the requirements of this study protocol.

          2. Subject has a disease diagnosis of moderately to severely active polyarticular or
             polyarticular course juvenile idiopathic arthritis (JIA; defined as arthritis
             affecting > = 5 joints at the time of treatment initiation). This corresponds to the
             International League of Associations for Rheumatology categories of polyarticular
             rheumatoid factor positive (RF+), polyarticular Rheumatoid factor negative (RF-)
             disease, and extended oligoarthritis disease.

          3. Subject must be aged 2 to < 4 years old with moderately to severely active
             polyarticular JIA or polyarticular course JIA or age 4 or greater weighing < 15 kg
             with moderately to severely active polyarticular JIA or polyarticular course JIA, per
             International League of Associations for Rheumatology (ILAR) criteria.

          4. Subject is judged to be in generally good health as determined by the Investigator
             based upon the results of medical history, laboratory profile, and physical
             examination performed at Screening and confirmed at Baseline. This includes, but is
             not limited to, a normal cardiopulmonary and normal neurological exam result.

          5. Parent or legal guardian must be able and willing to administer subcutaneous (SC)
             injections or have a qualified person available to administer SC injections.

          6. Parent or legal guardian must be willing to actively supervise storage and
             administration of study drug and to ensure that the time of each dose is accurately
             recorded in the subject's diary.

          7. Subject must have negative purified protein derivative (PPD) test (or equivalent) at
             Screening. If subject has a positive (greater than or equal to 5mm induration) PPD
             test result, a chest x-ray (posterior-anterior [PA] and lateral view) must be
             performed. If subject has a positive test (or equivalent), has had a past ulcerative
             reaction to PPD placement, and/or a chest x ray consistent with tuberculosis [TB]
             exposure, subject may not be enrolled into the study.

          8. For subjects in the European Union [EU], subject must have previously failed, had an
             insufficient response, or have been intolerant to at least one Disease-Modifying Anti
             Rheumatic Drug (DMARD).

        Exclusion Criteria:

          1. Subject has had prior exposure to Tysabri® (natalizumab) or Raptiva® (efalizumab) or
             any other biologic therapy, such as Orencia® (abatacept), Kineret® (anakinra),
             Actemra® (tocilizumab), Rituxan® (rituximab). Any previous use of anti-tumor necrosis
             factor [TNF] agents, including Enbrel® (etanercept), Remicade® (infliximab), Cimzia®
             (certolizumab pegol), Simponi® (golimumab), and adalimumab is also prohibited.

          2. Infection(s) requiring treatment with intravenous (IV) anti-infectives within 30 days
             prior to Baseline Visit or oral anti-infectives within 14 days prior to the Baseline
             Visit.

          3. Subject has undergone joint surgery within the preceding two months of the Screening
             Visit (at joints to be assessed within the study).

          4. Subject has a previous diagnosis of a condition that could cause arthritis other than
             polyarticular JIA.

          5. Subject has a history of an allergic reaction or significant sensitivity to
             constituents of the study drug, adalimumab.

          6. Subject has been treated with any investigational drug of chemical or biologic nature
             within a minimum of 30 days or 5 half lives (whichever is longer) of the drug prior to
             Baseline visit. Should these biologics become approved, they would continue to be
             excluded.

          7. Subject has a poorly controlled medical condition, such as uncontrolled diabetes,
             unstable heart disease, recent cerebrovascular accidents, seizure disorder, and any
             other condition which, in the opinion of the Investigator, would put the subject at
             risk by participation in the study.

          8. Subject has a history of clinically significant hematologic (e.g., severe anemia,
             leukopenia, thrombocytopenia, a clotting disorder), renal, liver disease (e.g.,
             fibrosis, cirrhosis, hepatitis), or active gastroenteric ulcer.

          9. Subject is considered by the Investigator, for any reason, to be an unsuitable
             candidate for the study.

         10. Subject has evidence of active TB infection.

         11. Subject has history of moderate to severe congestive heart failure (New York Heart
             Association [NYHA] class III or IV), recent cerebrovascular accident or thrombotic
             event and any other condition which, in the opinion of the investigator, would put the
             subject at risk by participation in the protocol.

         12. Evidence of dysplasia or history of malignancy (including lymphoma and leukemia).

         13. History of demyelinating disease (including myelitis) or neurologic symptoms
             suggestive of central nervous system [CNS] demyelinating disease.

         14. History of invasive fungal infection (e.g., listeriosis, histoplasmosis), active viral
             disorders, human immunodeficiency virus (HIV) infection.

         15. Positive Hepatitis B test result.

         16. Chronic recurring infections or active TB.

         17. Screening laboratory and other analyses show any of the following abnormal results:

               -  electrocardiogram [ECG] - with clinically significant abnormalities;

               -  Aspartate transaminases (AST) or alanine transaminase (ALT) > 1.75 the upper
                  limit of the reference range;

               -  Total bilirubin >=3 mg/dL;

               -  Serum creatinine > 1.6 mg/dL (convert to mmol/L).

         18. Evidence of dysplasia or history of malignancy (including lymphoma and leukemia) other
             than a successfully treated non-metastatic cutaneous squamous cell or basal cell
             carcinoma.