Hematologic and Lymphocytic DisorderPhase 2NCT04572815
Ustekinumab for the Prevention of Acute Graft-versus-Host Disease After Unrelated Donor Hematopoietic Cell Transplant
A Phase 2 study testing Quality-of-Life Assessment, Questionnaire Administration and Ustekinumab in people with Hematologic and Lymphocytic Disorder and Hematopoietic and Lymphoid System Neoplasm, run by Fred Hutchinson Cancer Center at 4 sites in United States.
Trial summary
This phase II trial studies how well ustekinumab works in preventing acute graft-versus-host disease after unrelated donor hematopoietic cell transplant. Sometimes the transplanted cells from a donor can attack the body's normal tissues (called graft-versus-host disease). Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Hematologic and Lymphocytic Disorder, Hematopoietic and Lymphoid System Neoplasm
- What is being tested?
- Quality-of-Life Assessment, Questionnaire Administration and Ustekinumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 116 participants
- Where
- 4 sitesUnited States
- Study dates
- 14 May 2021to 30 Jun 2027
- Phase
- Phase 2
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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18 - 70
- Signed informed consent.
- Hematologic malignancy or disorder requiring allogeneic hematopoietic cell transplantation
- Adequate vital organ function:
- Left ventricular ejection fraction (LVEF) ≥ 50%
- Forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), and diffusion capacity of the lung for carbon monoxide (DLCO) ≥ 50% of predicted values on pulmonary function tests
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- Transaminases (aspartate aminotransferase [AST], aspartate aminotransferase [ALT]) < 3 times upper limit of normal values
- Creatinine clearance ≥ 50 cc/min.
- Performance status: Karnofsky Performance Status Score ≥ 70%.
- HCT donor is at least 8/8 (matched at HLA-A, -B, -C, -DRB1) matched with the recipient
- PBSC (peripheral blood mobilized stem cells) as graft source
- Fully myeloablative, reduced-toxicity ablative, or reduced-intensity conditioning regimens. If melphalan is part of the conditioning regimen, dose must be at least 75mg/m^2
Who cannot join
- Active infection not controlled with appropriate antimicrobial therapy
- Human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection
- Anti-thymocyte globulin (ATG) as part of the conditioning regimen or GVHD prophylaxis
- Pregnant or nursing women
- Subjects of childbearing age unwilling to use an effective birth control method or refrain from sexual intercourse until 15 weeks after last dose of study drug
- Non-myeloablative conditioning regimens or conditioning regimens that use less than 75mg/m^2 of melphalan
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- Prior allogeneic transplant
- Non-malignant blood disorders (e.g. sickle cell disease, aplastic anemia)
- Positive screening test for tuberculosis
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Roswell Park Cancer Institute
Buffalo, New York, 14263
City of Hope Comprehensive Cancer Center,
Duarte, California, 91010
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, 98109
H. Lee Moffitt Cancer Center & Research Institute
Tampa, Florida, 33612
About this study
Official title: Randomized, Placebo-Controlled, Phase II Trial Examining Ustekinumab for Prevention of Graft Vs. Host Disease After Allogeneic Hematopoietic Cell Transplantation
This phase II trial studies how well ustekinumab works in preventing acute graft-versus-host disease after unrelated donor hematopoietic cell transplant. Sometimes the transplanted cells from a donor can attack the body's normal tissues (called graft-versus-host disease). Giving ustekinumab after the transplant may help prevent acute graft-versus-host disease by controlling the body's immune response. Funding Source- FDA OOPD.
Read the detailed description
OUTLINE: Patients are randomized to 1 of 2 arms.
ARM I: Between 4 and 72 hours prior to start of HCT conditioning therapy, patients receive ustekinumab intravenously (IV). Beginning 8 weeks after receiving IV ustekinumab, patients receive ustekinumab subcutaneously (SC) on days 50 (+/- 5 days), 100 (+/- 7 days), and 160 (+/- 7 days) post-HCT in the absence of grade III-IV acute GVHD, disease relapse or unacceptable toxicity. NOTE: HCT infusion takes place on day 0.
ARM II: Between 4 and 72 hours prior to start of HCT conditioning therapy, patients receive a placebo IV. Beginning 8 weeks after IV placebo, patients receive a placebo SC on days 50 (+/- 5 days), 100 (+/- 7 days), and 160 (+/- 7 days) post-HCT in the absence grade III-IV acute GVHD, of disease relapse, or unacceptable toxicity. NOTE: HCT infusion takes place on day 0.
After completion of study, patients are followed up at 6, 9, 12, 18, and 24 months post-HCT.
- Being tested
- Placebo Administration, Quality-of-Life Assessment, Questionnaire Administration, Ustekinumab
- Medical subject headings
- Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Placebo AdministrationDrug
Given IV and SC
Quality-of-Life AssessmentOther
Also called Quality of Life Assessment
Ancillary studies
Questionnaire AdministrationOther
Ancillary studies
UstekinumabBiological
Also called CNTO 1275, CNTO1275, Immunoglobulin G1, Anti-(Human Interleukin-12 Subunit beta (IL-12B, CLMF p40, NKSF2)) (Human Monoclonal CNTO 1275 gamma1-chain), Disulfide with Human Monoclonal CNTO 1275 kappa-chain, Dimer, Stelara
Given IV and SC
| Study group | Role | What they receive |
|---|---|---|
| Arm I (ustekinumab) | Experimental | Between 4 and 72 hours prior to start of HCT conditioning therapy, patients receive ustekinumab IV. Beginning 8 weeks after receiving IV ustekinumab, patients receive ustekinumab SC on days 50 (+/- 5 days), 100 (+/- 7 days), and 160 (+/- 7 days) post-HCT in the absence of grade III-IV acute GVHD, disease relapse or unacceptable toxicity. NOTE: HCT infusion takes place on day 0. Receives: Other: Quality-of-Life Assessment; Other: Questionnaire Administration; Biological: Ustekinumab |
| Arm II (placebo) | Placebo comparator | Between 4 and 72 hours prior to start of HCT conditioning therapy, patients receive a placebo IV. Beginning 8 weeks after IV placebo, patients receive a placebo SC on days 50 (+/- 5 days), 100 (+/- 7 days), and 160 (+/- 7 days) post-HCT in the absence of grade III-IV acute GVHD, disease relapse, or unacceptable toxicity. NOTE: HCT infusion takes place on day 0. Receives: Drug: Placebo Administration; Other: Quality-of-Life Assessment; Other: Questionnaire Administration |
What is being measured
The primary outcome is the main question the study is designed to answer.
Grade II-IV acute graft versus host disease (GVHD) survival
Will be treated as a binary outcome, and the Cochran-Mantel-Haenszel test will be used to compare the two groups based on the stratification factors.
Measured: At 6 months post-hematopoietic cell transplantation (HCT)
Secondary outcomes (9)
Cumulative incidence of grade II-IV and grade III-IV acute GVHD
Measured: At 100 days post-HCT
Cumulative incidence of grade II-IV and grade III-IV acute GVHD
Measured: At 6 months post-HCT
Acute GVHD organ staging, overall grading, and classification
Minnesota risk criteria will be used to assess organ involvement, individual organ staging, and overall acute GVHD grade. Risk classification will be performed per MacMillan et al.
Measured: From time of HCT, assessed up to day 100 post-HCT
Incidence of overall chronic GVHD
Will be assessed at serial study visits, and scored according to National Institutes of Health Consensus criteria.
Measured: From time of HCT, assessed up to 2 years post-HCT
Incidence of moderate-severe chronic GVHD
Will be assessed at serial study visits, and scored according to National Institutes of Health Consensus criteria.
Measured: From time of HCT, assessed up to 2 years post-HCT
Incidence of post-HCT relapse
Relapse is defined as hematologic relapse or any unplanned intervention (including withdrawal of immune suppression) to prevent progression of disease in patients with evidence (molecular, cytogenetic, flow cytometric, radiographic) of malignant disease. Will be compared using either the log-rank test (if no competing risks) or Gray's test (if competing risks are present). For time-to-event endpoints with competing risks, the log-rank test will also be used for exploratory purposes.
Measured: From time of HCT, assessed up to 2 years post-HCT
Incidence of non-relapse mortality
Non-relapse mortality indicates death with primary malignancy that served as HCT indication in remission. Will be compared using either the log-rank test (if no competing risks) or Gray's test (if competing risks are present). For time-to-event endpoints with competing risks, the log-rank test will also be used for exploratory purposes.
Measured: From time of HCT, assessed up to 2 years post-HCT
Relapse-free survival
Relapse is defined as hematologic relapse or any unplanned intervention (including withdrawal of immune suppression) to prevent progression of disease in patients with evidence (molecular, cytogenetic, flow cytometric, radiographic) of malignant disease. Will be compared using either the log-rank test (if no competing risks) or Gray's test (if competing risks are present). For time-to-event endpoints with competing risks, the log-rank test will also be used for exploratory purposes.
Measured: From time of HCT, assessed up to 2 years post-HCT
Overall survival
Will be compared using either the log-rank test (if no competing risks) or Gray's test (if competing risks are present). For time-to-event endpoints with competing risks, the log-rank test will also be used for exploratory purposes.
Measured: From time of HCT, assessed up to 2 years post-HCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2020
- Study start14 May 2021
- Primary completion27 Oct 2025
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematologic and Lymphocytic DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 116 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fred Hutchinson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Stephanie J. Lee, Principal investigator, Fred Hutch/University of Washington Cancer Consortium
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04572815
- Sponsor's protocol ID
- RG1005588
- Other IDs
- NCI-2020-02617, 10421, R01FD006836
- Status last verified
- Dec 2025
- First submitted
- 28 Sep 2020
- Last updated
- 2 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No