Pediatric Lupus Trial of Belimumab Plus Background Standard Therapy

NCT ID: NCT01649765

Last Updated: 2022-11-16

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

93 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-09-07

Study Completion Date

2028-04-05

Brief Summary

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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

Detailed Description

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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.

Conditions

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Systemic Lupus Erythematosus

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Arm 1

belimumab 10mg/kg IV monthly

Group Type EXPERIMENTAL

belimumab 10mg/kg

Intervention Type DRUG

belimumab 10mg/kg IV monthly

Arm 2

Normal Saline IV monthly

Group Type PLACEBO_COMPARATOR

placebo

Intervention Type OTHER

Normal Saline 250 ml

Interventions

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belimumab 10mg/kg

belimumab 10mg/kg IV monthly

Intervention Type DRUG

placebo

Normal Saline 250 ml

Intervention Type OTHER

Other Intervention Names

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Normal Saline

Eligibility Criteria

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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.
Minimum Eligible Age

5 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Human Genome Sciences Inc., a GSK Company

INDUSTRY

Sponsor Role collaborator

GlaxoSmithKline

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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GSK Clinical Trials

Role: STUDY_DIRECTOR

GlaxoSmithKline

Locations

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GSK Investigational Site

Phoenix, Arizona, United States

Site Status

GSK Investigational Site

Washington D.C., District of Columbia, United States

Site Status

GSK Investigational Site

Augusta, Georgia, United States

Site Status

GSK Investigational Site

St Louis, Missouri, United States

Site Status

GSK Investigational Site

New York, New York, United States

Site Status

GSK Investigational Site

The Bronx, New York, United States

Site Status

GSK Investigational Site

Cincinnati, Ohio, United States

Site Status

GSK Investigational Site

Rosario, Santa Fe Province, Argentina

Site Status

GSK Investigational Site

Capital Federal, , Argentina

Site Status

GSK Investigational Site

Santa Fe, , Argentina

Site Status

GSK Investigational Site

Calgary, Alberta, Canada

Site Status

GSK Investigational Site

Toronto, Ontario, Canada

Site Status

GSK Investigational Site

Aichi, , Japan

Site Status

GSK Investigational Site

Kagoshima, , Japan

Site Status

GSK Investigational Site

Miyagi, , Japan

Site Status

GSK Investigational Site

Tokyo, , Japan

Site Status

GSK Investigational Site

San Luis Potosí City, , Mexico

Site Status

GSK Investigational Site

Lima, , Peru

Site Status

GSK Investigational Site

Lima, , Peru

Site Status

GSK Investigational Site

Lima, , Peru

Site Status

GSK Investigational Site

Lodz, , Poland

Site Status

GSK Investigational Site

Warsaw, , Poland

Site Status

GSK Investigational Site

Moscow, , Russia

Site Status

GSK Investigational Site

Saint Petersburg, , Russia

Site Status

GSK Investigational Site

Tolyatti, , Russia

Site Status

GSK Investigational Site

Esplugues de Llobregat. Barcelona, , Spain

Site Status

GSK Investigational Site

Madrid, , Spain

Site Status

GSK Investigational Site

Valencia, , Spain

Site Status

GSK Investigational Site

Bristol, , United Kingdom

Site Status

GSK Investigational Site

Liverpool, , United Kingdom

Site Status

GSK Investigational Site

London, , United Kingdom

Site Status

GSK Investigational Site

London, , United Kingdom

Site Status

Countries

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United States Argentina Canada Japan Mexico Peru Poland Russia Spain United Kingdom

References

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Brunner HI, Abud-Mendoza C, Viola DO, Calvo Penades I, Levy D, Anton J, Calderon JE, Chasnyk VG, Ferrandiz MA, Keltsev V, Paz Gastanaga ME, Shishov M, Boteanu AL, Henrickson M, Bass D, Clark K, Hammer A, Ji BN, Nino A, Roth DA, Struemper H, Wang ML, Martini A, Lovell D, Ruperto N; Paediatric Rheumatology International Trials Organisation (PRINTO) and the Pediatric Rheumatology Collaborative Study Group (PRCSG). Safety and efficacy of intravenous belimumab in children with systemic lupus erythematosus: results from a randomised, placebo-controlled trial. Ann Rheum Dis. 2020 Oct;79(10):1340-1348. doi: 10.1136/annrheumdis-2020-217101. Epub 2020 Jul 22.

Reference Type BACKGROUND
PMID: 32699034 (View on PubMed)

Brunner HI, Abud-Mendoza C, Mori M, Pilkington CA, Syed R, Takei S, Viola DO, Furie RA, Navarra S, Zhang F, Bass DL, Eriksson G, Hammer AE, Ji BN, Okily M, Roth DA, Quasny H, Ruperto N. Efficacy and safety of belimumab in paediatric and adult patients with systemic lupus erythematosus: an across-study comparison. RMD Open. 2021 Sep;7(3):e001747. doi: 10.1136/rmdopen-2021-001747.

Reference Type BACKGROUND
PMID: 34531304 (View on PubMed)

Zhou X, Lee TI, Zhu M, Ma P. Prediction of Belimumab Pharmacokinetics in Chinese Pediatric Patients with Systemic Lupus Erythematosus. Drugs R D. 2021 Dec;21(4):407-417. doi: 10.1007/s40268-021-00363-2. Epub 2021 Oct 9.

Reference Type BACKGROUND
PMID: 34628605 (View on PubMed)

Arends EJ, Zlei M, Tipton CM, Cotic J, Osmani Z, de Bie FJ, Kamerling SWA, van Maurik A, Dimelow R, Gregan YI, Fox NL, Rabelink TJ, Roth DA, Sanz I, van Dongen JJM, van Kooten C, Teng YKO. Disruption of memory B-cell trafficking by belimumab in patients with systemic lupus erythematosus. Rheumatology (Oxford). 2024 Sep 1;63(9):2387-2398. doi: 10.1093/rheumatology/keae286.

Reference Type DERIVED
PMID: 38775637 (View on PubMed)

Hannon CW, McCourt C, Lima HC, Chen S, Bennett C. Interventions for cutaneous disease in systemic lupus erythematosus. Cochrane Database Syst Rev. 2021 Mar 9;3(3):CD007478. doi: 10.1002/14651858.CD007478.pub2.

Reference Type DERIVED
PMID: 33687069 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2011-000368-88

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

114055

Identifier Type: -

Identifier Source: org_study_id

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