Phase 2 N=93 Randomized Quadruple-blind Treatment

Pediatric Lupus Trial of Belimumab Plus Background Standard Therapy

Systemic Lupus Erythematosus

Bottom Line

View on ClinicalTrials.gov: NCT01649765 ↗

Enrolled (actual)

Serious AEs

32.0%

Results posted

Aug 2018

Primary outcome: Primary: Percentage of Participants With SLE Responder Index (SRI) Response at Week 52 — 43.6; 52.8 Percentage of participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Belimumab 10 mg/kg (Drug); Placebo (Other)
Age: Pediatric · 5+ yrs
Sex: All
Sponsor: GlaxoSmithKline
Primary completion: Jan 2018

Outcome Measures

Outcome	Result	p-value
PRIMARY Percentage of Participants With SLE Responder Index (SRI) Response at Week 52	43.6; 52.8	—
SECONDARY Percentage of Participants Meeting Pediatric Rheumatology International Trials Organization (PRINTO)/ American College of Rheumatology (ACR) Juvenile SLE Response Evaluation Criteria for Improvement in Juvenile SLE at Week 52 Using Definition 1 and 2	35.0; 60.4; 27.5; 52.8	—
SECONDARY Percent Change From Baseline in ParentGA at Week 52	-23.61; -53.85	—
SECONDARY Percent Change From Baseline in PGA at Week 52	-48.802; -56.525	—
SECONDARY Percent Change From Baseline in SELENA SLEDAI at Week 52	-38.0; -43.3	—
SECONDARY Percent Change From Baseline in PedsQL Physical Functioning Domain Score at Week 52	12.5; 10.5	—
SECONDARY Percent Change From Baseline in Proteinuria at Week 52	7.0920; -2.1277	—
SECONDARY Percentage of Participants With a Sustained SRI Response	41.0; 43.4	—
SECONDARY Percentage of Participants With a Sustained ParentGA Response	33.3; 59.1	—
SECONDARY Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	33; 42; 14; 9	—
SECONDARY Maximum Concentration at Steady State (Cmax, ss) and Minimum Concentration at Steady State (Cmin, ss)	315; 50	—
SECONDARY Area Under Curve of Belimumab at Steady State (AUC, ss)	3012	—

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

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.

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Data sourced from ClinicalTrials.gov (NCT01649765). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.