Belimumab for Prevention of Chronic Graft-versus-Host Disease Following Allogeneic Hematopoietic Cell Transplantation

April 16, 2026 updated by: Washington University School of Medicine

A Pilot Study of Belimumab (Benlysta) for Prevention of Chronic Graft-versus-Host Disease Following Allogeneic Hematopoietic Cell Transplantation

Given the role of B cells in the pathophysiology of chronic graft versus host disease (GvHD), the association between elevated BAFF levels post-transplant in abnormal B-cell homeostasis and chronic GvHD, and the efficacy of belimumab in the inhibition of soluble human B lymphocyte stimulator protein (BAFF) signaling, these proof-of-principle findings support the rational for use of belimumab as prophylaxis of chronic GvHD. The investigators propose a pilot and feasibility study to assess the safety and tolerability, as well as preliminary efficacy, of belimumab as prophylaxis of chronic GvHD following allogeneic hematopoietic cell transplantation (alloHCT). The investigators' central hypothesis is that belimumab will be well tolerated and have a favorable effect on incidence and severity of chronic GvHD.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
10

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Washington University School of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • At least 18 years of age
  • Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
  • Diagnosis of hematologic malignancy (i.e. acute myeloid leukemia, acute lymphoblastic leukemia, chronic lymphocytic leukemia, chronic myelogenous leukemia, Hodgkin's lymphoma, non-Hodgkin's lymphoma, myelodysplastic syndrome, chronic myelomonocytic leukemia)
  • Use of myeloablative or non-myeloablative conditioning regimen
  • Use of mobilized peripheral blood stem cells from fully HLA-matched related or unrelated donor as a graft source
  • Acute GvHD prophylaxis with methotrexate and tacrolimus

  • Documented complete remission with full donor engraftment (by STR identity testing) on Day +30 bone marrow biopsy

    • Complete remission: less than 5% blasts in an aspirate bone marrow sample with a count of at least 200 nucleated cells, no blasts with Auer rods or persistence of extramedullary disease PLUS absolute neutrophil count (ANC) > 1,500/μL, platelet count ≥ 50,000/μL and no leukemic blasts in the peripheral blood.
    • Negative minimal residual disease
    • Full donor engraftment by STR testing (either by bone marrow or peripheral blood testing)

  • Adequate end organ function:

    • Serum bilirubin ≤ 1.5 x upper limit of normal (ULN)
    • Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 x ULN
    • Creatinine clearance ≥ 40 mL/min/1.73 m^2 by the Cockcroft-Gault formula
  • Women of childbearing potential must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence) prior to study entry, for the duration of study participation, and for 16 weeks after the last dose of study drug. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately.
  • Able to understand and willing to sign an Institutional Review Board (IRB)-approved written informed consent.

Exclusion Criteria:

  • Active grade III-IV classic acute GvHD; subjects with prior resolved acute GvHD on stable doses of immunosuppression at time of enrollment will be permitted
  • Evidence of classic chronic GvHD or overlap chronic GvHD at time of enrollment
  • Subjects who participated in a clinical trial of acute GvHD prophylaxis in which chronic GvHD was a secondary end point
  • Donor lymphocyte infusion administered to treat relapse or loss of donor chimerism
  • Treatment with rituximab or other anti-B cell specific antibodies within previous 3 months
  • History of other malignancy ≤ 5 years previous with the exception of basal cell or squamous cell carcinoma of the skin which were treated with local resection only or carcinoma in situ of the cervix
  • Currently receiving any other investigational agents
  • Known allergy or intolerance to any component of belimumab, including human or murine proteins or monoclonal antibodies
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations (including current drug or alcohol abuse or dependence, or history of drug or alcohol abuse or dependence within the last year) that would limit compliance with study requirements
  • Use of parenteral (IV or IM) antibiotics (antibacterials, antivirals, anti-fungals, or anti-parasitic agents) within 14 days prior to planned start of therapy
  • Evidence of serious suicide risk including any history of suicidal behavior in the last 6 months and/or any suicidal ideation in the last 2 months and/or poses a significant suicide risk in the judgment of the investigator
  • History of pre-existing immunodeficiency disorder, autoimmune condition, or chronic infection
  • Known HIV positivity
  • Serologic evidence of current or past hepatitis B infection based on the results of testing for HBsAg and anti-HBc - Patients positive for HBsAg or HBcAb are excluded
  • Positive test for hepatitis C antibody (patients with documented clearance of hepatitis C by PCR following treatment will be permitted)
  • Currently on therapy for active chronic infection (such as tuberculosis, pneumocystis, cytomegalovirus, herpes simplex virus, herpes zoster and atypical mycobacteria). Prophylactic therapy is allowed.
  • Has any other clinically significant abnormal laboratory value in the opinion of the investigator

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Belimumab
  • Subjects meeting eligibility criteria will start treatment between Day +30 and Day +80 after alloHCT
  • Belimumab will be administered intravenously every 2 weeks for 3 doses followed by 4 doses at monthly intervals, for a total of 7 doses (6 months)
Drug: Belimumab
-Given over 1 hour
Other Names:
  • Benlysta

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Safety and Tolerability of Belimumab as Prophylaxis of Chronic GvHD in Subjects Following alloHCT as Measured by Number of Participants Who Experience Each Adverse Event
Time Frame: From start of treatment through 30 days following the completion of treatment (median length of follow-up 205 days, full range 56-229 days)
  • Adverse events were graded according to CTCAE v4.03.

  • All adverse events were collected with the exception of:

    • Adverse events that are less than CTCAE grade 3 unless the AE met the definition of a serious adverse event.
    • Adverse events not of special interest as defined in the protocol (special interest AEs will be recorded regardless of grade, including those thought to be related to chronic GvHD)
From start of treatment through 30 days following the completion of treatment (median length of follow-up 205 days, full range 56-229 days)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Incidence and Severity of Chronic GvHD
Time Frame: Cycle 3 (each cycle is 4 weeks), Cycle 4, Cycle 5, Cycle 6, Cycle 7, 6 months, 9 months, 12 months, 15 months, 18 months, 21 months, and 24 months
-The presence of chronic GvHD will be determined according to the 2014 NIH Criteria. If chronic GvHD is diagnosed, each organ will be scored 0-3 and graded, according to the 2014 NIH criteria for Diagnosing and Staging of Chronic GvHD. These data will allow calculation of the NIH global severity score of mild, moderate or severe.
Cycle 3 (each cycle is 4 weeks), Cycle 4, Cycle 5, Cycle 6, Cycle 7, 6 months, 9 months, 12 months, 15 months, 18 months, 21 months, and 24 months
Incidence and Severity of Acute GvHD
Time Frame: Cycle 3 (each cycle is 4 weeks), Cycle 4, Cycle 5, Cycle 6, Cycle 7, and 6 months

  • Acute GvHD staging has 4 organ systems: skin, lower gastrointestinal (GI), upper GI, and liver.

    • Skin (% body surface area): stage 0 no rash, stage 1 < 25%, stage 2 25%-50%, stage 3 >50%, stage 4 generalized erythroderma with bullae
    • Lower GI (diarrhea, mL/day): stage 0 <500, stage 1 >500, stage 2 >1000, stage 3 >1500, stage 4 severe abdominal pain +/- ileus
    • Upper GI: only stage is stage 1 which is persistent, severe nausea
    • Liver (bilirubin, mg/dL): stage 0 ≥2, stage 1 2.1-3, stage 2 3.1-6, stage 3 6.1-15, stage 4 >15

  • Acute GvHD will be graded according to modified Minnesota grading scale. Organ systems are broken down into skin, liver, lower gastrointestinal (GI), and upper gastrointestinal (GI) and the grades are I, II, III, IV.

    • I: skin stage 1-2, liver/lower GI/upper GI stage 0
    • II: skin stage 3, liver/lower GI/upper GI stage 1
    • III: liver stage 2-4, lower GI stage 2-3
    • IV: skin stage 4, lowr GI stage 4 Grade IV is t
Cycle 3 (each cycle is 4 weeks), Cycle 4, Cycle 5, Cycle 6, Cycle 7, and 6 months
Overall Survival
Time Frame: 6 months, 12 months, and 24 months after alloHCT
-Overall survival will be determined from date of belimumab initiation, with death from any cause as the event of interest, and censoring at last follow up date for those with incomplete observations.
6 months, 12 months, and 24 months after alloHCT
Relapse Rate
Time Frame: 6 months, 8 months, 12 months, and 24 months post-alloHCT
6 months, 8 months, 12 months, and 24 months post-alloHCT
Overall Corticosteroid Requirement for Treatment of Chronic GvHD
Time Frame: 6 months after alloHCT
6 months after alloHCT
Overall Corticosteroid Requirement for Treatment of Chronic GvHD
Time Frame: 12 months after alloHCT
12 months after alloHCT
Overall Corticosteroid Requirement for Treatment of Chronic GvHD
Time Frame: 24 months after alloHCT
24 months after alloHCT
Number of Participants That Required Alternative Treatment Modalities for Chronic GvHD
Time Frame: 6 months after alloHCT
-The use of additional systemic immune suppressive agents will be captured at each study visit.
6 months after alloHCT
Number of Participants That Required Alternative Treatment Modalities for Chronic GvHD
Time Frame: 12 months after alloHCT
-The use of additional systemic immune suppressive agents will be captured at each study visit.
12 months after alloHCT
Number of Participants That Required Alternative Treatment Modalities for Chronic GvHD
Time Frame: 24 months after alloHCT
-The use of additional systemic immune suppressive agents will be captured at each study visit.
24 months after alloHCT

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Washington University School of Medicine

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
GlaxoSmithKline
American Cancer Society, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Iskra Pusic, M.D., Washington University School of Medicine

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 16, 2018

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 4, 2022

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 9, 2024

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 29, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 30, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 5, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 20, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 16, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 201709068

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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