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Leukemia, AcutePhase 1NCT05823571

Itacitinib With High-dose Posttransplantation Cyclophosphamide in Older Patients

A Phase 1 study testing Itacitinib in people with Leukemia, Acute and Myelodysplastic Syndromes, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

This research is being done to learn whether drug called itacitinib, which is a novel inflammation- and immune-lowering drug (immunosuppressant), can be given before and after non-myeloablative peripheral blood stem cell transplantation (PBSCT; also known as a 'mini' transplant) to help prevent certain complications… Read more about this study

Who is it for?
Ages 60 years and older
What is being tested?
Itacitinib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
32 participants
Where
1 siteUnited States
Study dates
6 Jul 2023to Mar 2030
Phase
Phase 1
Are there results?
Not yet: the study is under way

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

Who may be able to join

  • Presence of a suitable related, HLA-haploidentical (partially mismatched) stem cell donor.
  • Eligible diagnoses:
  • Acute leukemias in complete remission with minimal residual disease
  • Myelodysplastic syndrome (MDS) with at least one poor-risk feature
  • Chronic myelomonocytic leukemia with at least one poor-risk feature
  • T-cell PLL in PR or better prior to transplantation.
Show all 15Show fewer
  • Tyrosine kinase-refractory CML in first chronic phase, TKI-intolerant CML in first chronic phase, or CML in second or subsequent chronic phase.
  • Philadelphia chromosome negative myeloproliferative disease (including myelofibrosis)
  • Multiple myeloma or plasma cell leukemia with a PR or better to the last treatment regimen
  • Age ≥ 60 years.
  • Adequate end-organ function as measured by:
  • Left ventricular ejection fraction ≥ 35% or shortening fraction > 25%
  • Bilirubin ≤ 3.0 mg/dL (unless due to Gilbert's syndrome or hemolysis), and ALT and AST ≤ 5 x ULN
  • FEV1 and FVC ≥ 40% of predicted
  • ECOG performance status ≤ 2 or Karnofsky score ≥ 60

Who cannot join

  • No active extramedullary leukemia or known active CNS involvement by malignancy.
  • Any previous autologous HSCT must have occurred at least 3 months prior to start of conditioning.
  • No previous allogeneic HSCT.
  • Not pregnant or breast-feeding
  • No uncontrolled infection.
  • No known HIV infection.
  • No active replicating HBV or HCV infection detected by PCR that requires treatment or at risk for HBV reactivation (positive HBsAg)

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

    Baltimore, Maryland, 21231

Other trials in United States

About this study

Official title: Phase 1a/1b Study of Itacitinib (INCB039110) for Cytokine Release Syndrome Prevention and Minimization of Immunosuppression Following Nonmyeloablative Related Partially HLA-mismatched Peripheral Blood Stem Cell Transplant (PBSCT) With High-dose Posttransplantation Cyclophosphamide in Older Patients (Age 60 Years)

This research is being done to learn whether drug called itacitinib, which is a novel inflammation- and immune-lowering drug (immunosuppressant), can be given before and after non-myeloablative peripheral blood stem cell transplantation (PBSCT; also known as a 'mini' transplant) to help prevent certain complications such as cytokine release syndrome (CRS) for patients with blood cancers, using peripheral blood from a relative. The investigators will also examine if by using itacitinib the investigators can reduce the duration of MMF (other immune suppressive drug administration posttransplant).

Read the detailed description

The NMA PBSC haplo transplant is associated with a higher risk of morbidity and mortality from the cytokine (IL-6 and others)-driven CRS and perhaps higher incidence of acute and chronic GVHD compared to bone marrow (BM) haplo allografting with post-transplant cyclophosphamide (PTCy). Notably, severe CRS (grade 3 and higher) appears to be more common in older patients (≥ 60 years) and is associated with significantly higher non-relapse mortality (NRM) in this patient group. Itacitinib has demonstrated safety, tolerability, ability to inhibit cytokines, including IL-6. Data also suggest that itacitinib can be administered safely in peri- and post-transplant period in the setting of Posttransplant CY immune prophylaxis and haploPBSCT with no evidence of delayed engraftment or delayed count recovery, with significant reduction in CRS compared to historical control, and a low rate of aGVHD, cGVHD, and NRM, and with no increase in relapse risk. Thus, the investigators propose a clinical study in which itacitinib will be used prophylactically in recipients age 60 years and older to prevent development of severe CRS, reduce severe CRS-associated NRM, and the incidence of severe GVHD, thus allowing further reduction in posttransplant immunosuppression therapy after PTCy-based NMA related partially-mismatched PB allografting.

Being tested
Itacitinib
Medical subject headings
Leukemia, Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic, Leukemia, Prolymphocytic, T-Cell, Myeloproliferative Disorders, Multiple Myeloma, Leukemia, Plasma Cell

What participants receive

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

  • ItacitinibDrug

    A standard 3+3 design will be used to evaluate the safety of itacitinib plus different immunosuppression regimens. This study has four predefined Regimens that will be explored in the optimal Regimen-finding phase and are listed in Table 2 of the protocol. Itacitinib will be given in conjunction with each of four different regimens for immunosuppression. Regimen 1 is the current standard for our BMT patients, with a duration of MMF from day 5-35. Regimen 2 will decrease the duration of MMF from 35 to day 25. Regimen 3 will decrease the duration of MMF from 35 to day 15. Regimen 4 will eliminate MMF altogether. We will start with Regimen 1, which combines itacitinib with the current standard of immunosuppression. Progression through cohorts (Regimens) will be based on a standard 3+3 design to find the optimal regimen.

Study groups in NCT05823571
Study groupRoleWhat they receive
ItacitinibExperimentalItacitinib will be given at 200 mg orally daily from day -3 to day 90. Itacitinib will be given in conjunction with one of four different regimens for immunosuppression. These 4 regimens are listed in Table 2, Section 5.2 of the protocol. Itacitinib may continue beyond day +90 if there is GVHD. NOTE: If patient develops GVHD requiring treatment after all immune suppression, including itacitinib, is stopped on day +90, the itacitinib will not be restarted and the patient will be treated per standard of care.
Receives: Drug: Itacitinib

What is being measured

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

  1. Number of participant deaths

    Number of participant deaths will be used to assess the efficacy of itacitinib in preventing the occurrence of death.

    Measured: 14 days

  2. Number of participants with grade 3 or higher CRS

    Number of participants with grade 3 or higher CRS will be used to assess the efficacy of itacitinib in preventing the development of severe (grade 3 or higher) cytokine release syndrome (CRS).

    Measured: 14 days

  3. Number of participants with grade 1-2 CRS that requires additional CRS-directed treatment

    Number of participants with grade 1-2 CRS that requires additional CRS-directed treatment will be used to assess the efficacy of itacitinib in preventing the development of grade 1-2 CRS that requires additional CRS-directed treatment.

    Measured: 14 days

  4. Number of participants with treatment limiting toxicities by Day 60

    Number of participants with treatment limiting toxicities by Day 60 will be used to identify the safe PTCy-based immunosuppressive regimen that incorporates itacitinib with tacrolimus and a reduced duration of immunosuppression with mycophenolate (MMF).

    Measured: 60 days

Timeline

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

  1. First posted21 Apr 2023
  2. Study start6 Jul 2023
  3. Primary completionMar 2028 (estimated)
  4. Study completionMar 2030 (estimated)

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
32 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05823571
Sponsor's protocol ID
J2283
Other IDs
IRB00298829
Status last verified
Oct 2026
First submitted
10 Apr 2023
Last updated
7 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Itacitinib in people with Leukemia, Acute and Myelodysplastic Syndromes, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins 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 15 inclusion and 7 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: under way, not looking for participants, as of the sponsor's last update on 7 Oct 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?

This study is still under way. Results are not available in the source record yet.

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?

Number of participant deaths (time frame: 14 days). The study lists 4 primary outcomes in all. 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 NCT05823571.

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 7 Oct 2026.

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