pediatric lupus trial of belimumab plus background standard therapy

Conditions: Systemic Lupus Erythematosus

Summary: 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.

Eligibility criteria:
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.

Keywords: sle flare index sri belimumab efficacy safety b lymphocyte blys selena sledai bilag lupus systemic lupus erythematosus (sle) pga pharmacokinetics placebo printo