Open-label Study of Belimumab Plus Standard Therapy in Chinese Pediatric Participants With Active Systemic Lupus Erythematosus (SLE)

NCT04908865 | Completed Phase 4 Results

• 1 other identifier: 213560
• Study Type: interventional
• Target: 67
• Locations: 1 country
• Sites: 9

Brief Summary

This study will be conducted to evaluate the safety, efficacy and pharmacokinetics of belimumab administered in combination with background standard therapy in pediatric participants with active SLE.

Conditions

Systemic Lupus Erythematosus

Keywords

Pediatric; Belimumab; Pharmacokinetics; Active Systemic Lupus Erythematosus; Safety; Efficacy

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Adverse Events of Special Interest (AESIs) Through Week 52

  An adverse event (AE) is defined as any untoward medical occurrence in a clinical study participant, temporally associated with the use of a study intervention, whether or not considered related to the study intervention. AESIs included malignancies, post-infusion systemic or hypersensitivity reactions, infections (including serious infections of special interest), and depression, suicide, or self-injury. Infections of special interest included opportunistic infections (OI), herpes zoster (HZ), tuberculosis (TB), and sepsis. Number of participants with AESIs as identified by custom Medical Dictionary for Regulatory Activities (MedDRA) query has been reported.

  Up to Week 52

• Number of Participants With Greater Than Equal to (>=) 4 Points Reduction From Baseline to Week 52 in Safety of Estrogen in Lupus National Assessment - Systemic Lupus Erythematosus Disease Activity Index (SELENA-SLEDAI) Score

  The SELENA-SLEDAI score is a cumulative and weighted index for assessing SLE disease activity in participants with SLE. It consists of 24 disease descriptors related to signs and symptoms, laboratory tests, and physician's assessment across 9 organ systems. Each descriptor is assigned a weighted score (8 descriptors with a weight of 8 each, 6 descriptors with a weight of 4 each, 7 descriptors with a weight of 2 each, and 3 descriptors with a weight of 1 each) which is added up if the descriptor is observed during a visit or within the preceding 10 days. The total score ranges from 0 (no disease activity) to 105 (all 24 descriptors present simultaneously). A higher score indicates a more significant degree of disease activity. Baseline was defined as the latest pre-dose assessment with a non-missing value, including those from unscheduled visits. Number of participants with a decrease of 4 points or more in the score at Week 52 compared to their Baseline score is presented.

  Baseline (Day 0) and Week 52

Secondary Outcomes (15)

• Number of Participants With AEs and Serious Adverse Events (SAEs) Through Week 52

  Up to Week 52

• Percentage of Participants With >=4 Points Reduction From Baseline in SELENA-SLEDAI Score by Each Visit

  Baseline (Day 0) and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, and 52

• Change From Baseline to Week 52 in Physician Global Assessment (PGA)

  Baseline (Day 0) and Week 52

• Change From Baseline to Week 52 in Parent Global Assessment (ParentGA)

  Baseline (Day 0) and Week 52

• Change From Baseline in Average Daily Prednisone Equivalent Dose at Week 52

  Baseline (Day 0) and Week 52

Study Arms (1)

Pediatric participants receiving belimumab

EXPERIMENTAL

Drug: Belimumab; Drug: Standard therapy

Interventions

Belimumab DRUG

Belimumab will be administered.

Pediatric participants receiving belimumab

Standard therapy DRUG

Standard therapy will be continued.

Pediatric participants receiving belimumab

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participants have or have had in series, 4 or more of the American College of Rheumatology (ACR) 11 criteria for the classification of SLE.
• Participant's age is 5 to 17 years at the time of informed consent.
• Have active SLE disease defined as a SELENA SLEDAI score \>= 8 at screening (SELENA SLEDAI scoring).
• Have unequivocally positive autoantibody test results defined as an anti-nuclear antibody (ANA) titer \>=1:80 and/or a positive anti-Double stranded deoxyribonucleic acid (dsDNA) serum antibody test.
• Are on a stable SLE therapy at Baseline. The stable treatment at Baseline consists of corticosteroids, anti-malarials, immunosuppressive/immunomodulatory agents and Non-steroidal anti-inflammatory drugs (NSAIDs), alone or in combination, at a fixed dose for a period of at least 30 days prior to Day 0.
• No gender restriction. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
• The investigator, or a person designated by the investigator, will obtain written informed assent from each study participant or the participant's legally acceptable representative, parent(s), or legal guardian and the participant's assent, when applicable, before any study-specific activity is performed. The investigator will retain the original copy of each participant's signed assent document.

You may not qualify if:

• Have an estimated glomerular filtration rate (eGFR) as calculated by Schwartz Formula of less than 30 mL/minutes.
• Have acute severe nephritis defined as a significant worsening of renal disease (for example \[e.g.\], the presence of urinary sediments and other lab abnormalities) that, in the opinion of the study investigator, may lead to the participant requiring induction therapy with intravenous (IV) cyclophosphamide, Mycophenolate mofetil (MMF) or high dose corticosteroids during the first 6 months of the study.
• Have a history of a major organ transplant (e.g., heart, lung, kidney, liver) or hematopoietic stem cell/marrow transplant.
• Have clinical evidence of significant, unstable or uncontrolled, acute or chronic diseases not due to SLE (cardiovascular, pulmonary, hematologic, gastrointestinal, hepatic, renal, neurological, malignancy or infectious diseases) which, in the opinion of the investigator, could confound the results of the study or put the participant at undue risk.
• Have a planned surgical procedure or a history of any other medical disease (e.g., cardiopulmonary), laboratory abnormality, or condition (e.g., poor venous access) that, in the opinion of the investigator, makes the participant unsuitable for the study.
• Have a history of malignant neoplasm within the last 5 years.
• Have a history of a primary immunodeficiency.
• Have an Immunoglobulin A (IgA) deficiency (IgA level less than \[\<\]10 mg/deciliters \[milligrams/dL\]).
• Have acute or chronic infections requiring management.
• Have recent infections that, in the opinions of the investigator, makes the participant unsuitable for the study or could put the participant at undue risk.
• Have current drug or alcohol abuse or dependence, or a history of drug or alcohol abuse or dependence within 364 days prior to Day 0.
• Have a Grade 3 or greater laboratory abnormality based on the protocol toxicity scale except for the following that are allowed:
• Stable Grade 3 prothrombin time (PT) secondary to warfarin treatment.
• Stable Grade 3 partial thromboplastin time (PTT) due to lupus anticoagulant and not related to liver disease or anti-coagulant therapy.
• Stable Grade 3 hypoalbuminemia due to lupus nephritis and not related to liver disease or malnutrition.

Sponsors & Collaborators

• GlaxoSmithKline: lead

Study Sites (9)

GSK Investigational Site

Beijing, 100045, China

Location

GSK Investigational Site

Changchun, 130021, China

Location

GSK Investigational Site

Changsha, 410007, China

Location

GSK Investigational Site

Chongqing, 400014, China

Location

GSK Investigational Site

Hangzhou, 310052, China

Location

GSK Investigational Site

Nanjing, 210011, China

Location

GSK Investigational Site

Shanghai, 361006, China

Location

GSK Investigational Site

Suzhou, 215007, China

Location

GSK Investigational Site

Xi'an, 710054, China

Location

MeSH Terms

Conditions

Lupus Erythematosus, Systemic

Interventions

belimumab; Standard of Care

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Limitations and Caveats

Data has not been disclosed for PK parameters derived from population PK analysis, as this analysis, integrating data from Study 213560 and other studies, is still under discussion and review to fully understand the PK characteristics. The data for all pre-specified PK parameters will be disclosed by 30 June 2026.

Results Point of Contact

• Title: GSK Response Center
• Organization: GlaxoSmithKline

GSKClinicalSupportHD@gsk.com

866-435-7343

Study Officials

• GSK Clinical Trials

  GlaxoSmithKline

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 26, 2021
• First Posted: June 1, 2021
• Study Start: October 21, 2021
• Primary Completion: May 23, 2024
• Study Completion: August 13, 2024
• Last Updated: July 23, 2025
• Results First Posted: July 23, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: IPD will be made available within 6 months of publishing the results of the primary endpoints, a key secondary endpoints and safety data of the study.
• Access Criteria: Access is provided after a research proposal is submitted and has received approval from the Independent Review Panel and after a Data Sharing Agreement is in place. Access is provided for an initial period of 12 months but an extension can be granted, when justified, for up to another 12 months.

Locations

CN (9)