Pediatric Lupus Trial of Belimumab Plus Background Standard Therapy

A Multi-center, Randomized, Placebo-Controlled Trial to Evaluate the Safety, Efficacy, and Pharmacokinetics of Belimumab, a Human Monoclonal Anti-BLyS Antibody, Plus Standard Therapy in Pediatric Patients With Systemic Lupus Erythematosus

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

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.