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Graft Vs Host DiseasePhase 1NCT03207958

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

A Phase 1 study testing Belimumab in people with Graft Vs Host Disease and Graft-versus-host-disease, run by Washington University School of Medicine at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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… Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Belimumab
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteUnited States
Study dates
16 May 2018to 9 Feb 2024
Phase
Phase 1
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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.

Who cannot join

  • 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
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  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Washington University School of Medicine

    St Louis, Missouri, 63110

Other trials in United States

About this study

Official title: 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.

Being tested
Belimumab
Medical subject headings
Graft vs Host Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BelimumabDrug

    Also called Benlysta

    -Given over 1 hour

Study groups in NCT03207958
Study groupRoleWhat they receive
BelimumabExperimental* 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)
Receives: Drug: Belimumab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

    • 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)

    Measured: 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 outcomes (10)

  1. Incidence and Severity of Chronic GvHD

    -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.

    Measured: 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

  2. Incidence and Severity of Acute GvHD

    • 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

    Measured: Cycle 3 (each cycle is 4 weeks), Cycle 4, Cycle 5, Cycle 6, Cycle 7, and 6 months

  3. Overall Survival

    -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.

    Measured: 6 months, 12 months, and 24 months after alloHCT

  4. Relapse Rate

    Measured: 6 months, 8 months, 12 months, and 24 months post-alloHCT

  5. Overall Corticosteroid Requirement for Treatment of Chronic GvHD

    Measured: 6 months after alloHCT

  6. Overall Corticosteroid Requirement for Treatment of Chronic GvHD

    Measured: 12 months after alloHCT

  7. Overall Corticosteroid Requirement for Treatment of Chronic GvHD

    Measured: 24 months after alloHCT

  8. Number of Participants That Required Alternative Treatment Modalities for Chronic GvHD

    -The use of additional systemic immune suppressive agents will be captured at each study visit.

    Measured: 6 months after alloHCT

  9. Number of Participants That Required Alternative Treatment Modalities for Chronic GvHD

    -The use of additional systemic immune suppressive agents will be captured at each study visit.

    Measured: 12 months after alloHCT

  10. Number of Participants That Required Alternative Treatment Modalities for Chronic GvHD

    -The use of additional systemic immune suppressive agents will be captured at each study visit.

    Measured: 24 months after alloHCT

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Jul 2017
  2. Study start16 May 2018
  3. Primary completion4 Jun 2022
  4. Study completion9 Feb 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03207958
Sponsor's protocol ID
201709068
Status last verified
Apr 2026
First submitted
29 Jun 2017
Last updated
20 Apr 2026
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Belimumab in people with Graft Vs Host Disease and Graft-versus-host-disease, run by Washington University School of Medicine at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 16 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 20 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 20 Apr 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03207958.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Apr 2026.

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