New drug combo aims to tame deadly transplant complication
NCT ID NCT04339101
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase II trial tested whether adding the drug itacitinib to standard medications (tacrolimus and sirolimus) can better prevent graft-versus-host disease (GVHD) in 59 patients with blood cancers receiving donor stem cell transplants. GVHD occurs when donor immune cells attack the recipient's body. The study looked at safety and how well the combination worked to keep patients free from GVHD and relapse one year after transplant.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- itacitinib
- What this could lead to
- If it works, this could reduce the risk of graft-versus-host disease and improve survival after donor stem cell transplants for blood cancers.
- What could go wrong
- This is a small, early-phase trial (59 participants) focused on safety and feasibility. The added drug may cause more side effects or not improve outcomes.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
59 people
The number who actually took part.
- Started
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Nov 2020
- Finished
-
Oct 2025
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 to 75 years
- Sex
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Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Documented informed consent of the participant and/or legally authorized representative * Assent, when appropriate, will be obtained per institutional guidelines * Agreement to allow the use of archival tissue from diagnostic tumor biopsies * If unavailable, exceptions may be granted with study principal investigator (PI) approval * Performance status: Karnofsky \>= 70% * Patients with neoplastic hematologic disorders with indication of allogeneic transplant according to the standard guidelines as follows: * Acute leukemia (AL) in first complete response (CR1) or subsequent complete response (CR) or active disease with bone marrow (BM) blast of \< 5% * Myelodysplastic syndrome (MDS) with intermediate-2 or high risk per International Prognostic Scoring System (IPSS) or * Myelofibrosis; primary or secondary if intermediate-2 or high risk per Dynamic International Prognostic Scoring System (DIPPS) * All candidates for this study must have a matched related donor (MRD) who is willing to donate BM or peripheral blood stem cells or an 8/8 allele matched unrelated donor (MUD) * Total bilirubin =\< 1.5 X upper limit of normal (ULN) (unless has Gilbert's disease) (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated. In case of active disease, evaluation should be done within 15 days) * Aspartate aminotransferase (AST) =\< 2.5 x ULN (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated. In case of active disease, evaluation should be done within 15 days) * Alanine aminotransferase (ALT) =\< 2.5 x ULN (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated. In case of active disease, evaluation should be done within 15 days) * Creatinine clearance of \>= 60 mL/min per 24 hour urine test or the Cockcroft-Gault formula (performed within 28 days prior to day 1 of protocol therapy unless otherwise stated. In case of active disease, evaluation should be done within 15 days) * Left ventricular ejection fraction (LVEF) \>= 50% * Note: To be performed within 28 days prior to day 1 of protocol therapy * If able to perform pulmonary function tests: forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC) and diffusion capacity of the lung for carbon monoxide (DLCO) (diffusion capacity) \>= 50% of predicted (corrected for hemoglobin). If unable to perform pulmonary function tests: oxygen (O2) saturation \> 92% on room air. * Note To be performed within 28 days prior to day 1 of protocol therapy * Seronegative for human immunodeficiency virus (HIV) antigen (Ag)/antibody (Ab) combination (combo), hepatitis C virus (HCV), active hepatitis B virus (HBV) (surface antigen negative), and syphilis (rapid plasma reagin measurement \[RPR\]) * If HCV positive, hepatitis C ribonucleic acid (RNA) quantitation must be performed * Meets other institutional and federal requirements for infectious disease titer requirements * Note Infectious disease testing to be performed within 28 days prior to day 1 of protocol therapy * Women of childbearing potential (WOCBP): negative urine or serum pregnancy test. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required * Agreement by females and males of childbearing potential to use an effective method of birth control or abstain from heterosexual activity for the course of the study through at least 6 months after the last dose of protocol therapy * Childbearing potential defined as not being surgically sterilized (men and women) or have not been free from menses for \> 1 year (women only) Exclusion Criteria: * Chemotherapy, radiation therapy, biological therapy, and/or immunotherapy within 21 days prior to day 1 of protocol therapy * Note: Conditioning regimen within 21 days prior to day 1 of protocol therapy is not considered as an exclusion criterion * History of allergic reactions attributed to compounds of similar chemical or biologic composition to study agent * Psychological issues, no appropriate caregivers identified, or non-compliant to medication * Uncontrolled medical or psychiatric disorders which may preclude patients to undergo clinical studies (Discretion of the attending physician) * Active diarrhea due to inflammatory bowel disease or malabsorption syndrome * Clinically significant uncontrolled illness * Active, uncontrolled systemic infection (bacterial, viral, or fungal) requiring antibiotics * Known history of immunodeficiency virus (HIV) or hepatitis B or hepatitis C infection * Diagnosis of Gilbert's disease * Other active malignancy * Females only: Pregnant or breastfeeding * Any other condition that would, in the investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns with clinical study procedures * Prospective participants who, in the opinion of the investigator, may not be able to comply with all study procedures (including compliance issues related to feasibility/logistics)
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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City of Hope Medical Center
Duarte, California, 91010, United States
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Other studies related to the condition(s) this trial covers.
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