Pediatric Lupus Trial of Belimumab Plus Background Standard Therapy

This is a multi-center study to evaluate the safety, pharmacokinetics, and efficacy of
belimumab intravenous (IV) in pediatric patients 5 to 17 years of age with active systemic
lupus erythematosus (SELENA SLEDAI score ≥ 6). The study will consist of three phases: a
52-week randomized, placebo-controlled, double-blind phase; a long term open label
continuation phase; and a long term safety follow up phase. The long term open label
continuation and safety follow up periods will continue for at least 5 years and possibly up
to 10 years from a subject's initial treatment with belimumab. Enrolment will be staggered by
age cohorts to allow safety and PK interim analyses. Subjects will be randomized to belimumab
10mg/kg or placebo IV monthly dosing while continuing to receive background standard therapy
throughout the study. An independent data monitoring committee (IDMC) will monitor the study
as it progresses.

Inclusion Criteria:

- 5 years to 17 years of age at enrollment

- Have a clinical diagnosis of SLE according to the American College of Rheumatology
(ACR) classification criteria.

- Have active SLE disease (SELENA SLEDAI score ≥ 6).

- Have positive anti-nuclear antibody (ANA) test results.

- Are on a stable SLE treatment regimen at a fixed dose for a period of at least 30 days
prior to Day 0.

- Females of childbearing age are willing to use appropriate contraception

- Subject age appropriate assent and parent or legal guardian informed consent to
participate

Exclusion Criteria:

- Pregnant or nursing.

- Have received treatment with belimumab (BENLYSTA®) at any time. (BENLYSTA® is a
registered trademark of the GSK group of companies.)

- Treatment with any B cell targeted therapy (for example, rituximab) or an
investigational biological agent in the past year.

- Have received anti-TNF therapy; Interleukin-1 receptor antagonist; IVIG; or
plasmapheresis within 90 days of Day 0.

- Have received high dose prednisone or equivalent (>1.5mg/kg/day) within 60 days of
baseline.

- Have received intravenous (IV) cyclophosphamide within 60 days of Day 0.

- Have received any new immunosuppressive/immunomodulatory agent, anti-malarial agent
within 60 days of baseline.

- Have severe lupus kidney disease.

- Have active central nervous system (CNS) lupus.

- Have had a major organ transplant.

- Have significant unstable or uncontrolled acute or chronic diseases or conditions not
due to SLE.

- Have a planned surgical procedure.

- History of malignant neoplasm within the last 5 years.

- Have required management of acute or chronic infections in the past 60 days.

- Have current drug or alcohol abuse or dependence.

- Have a historically positive test, or test positive at screening for HIV, Hepatitis B,
or Hepatitis C.

- Have an IgA deficiency.

- Have severe laboratory abnormalities.

- Have had anaphylactic reaction to X-ray contrast agents or biologic agents.

- Suicidal behavior or ideation.

- Children in Care(CiC): a child who has been placed under the control or protection of
an agency, organisation, institution or entity by the courts, the government or a
government body, acting in accordance with powers conferred on them by law or
regulation.