Itacitinib Evidence: Trial Results and Peer-Reviewed Publications

Hipa.ai Research · Source: PubMed & ClinicalTrials.gov / AACT · Last updated:

The clinical evidence base for Itacitinib comprises 37 peer-reviewed publications across 15 journals, 3 pivotal-trial primary-outcome rows reported to ClinicalTrials.gov, spanning indications including Scleroderma, Systemic, Graft vs Host Disease, Carcinoma, Non-Small-Cell Lung, and Cytokine Release Syndrome. Most recent publication: The role of the molecular tumor board: learnings from the ROME trial., NPJ Precis Oncol, 2026.

Top peer-reviewed publications

Curated set of pivotal-trial result papers and recent publications in high-tier journals.

  1. JAK/STAT signaling in hematological malignancies.
    Vainchenker W, Constantinescu SN · Oncogene · 2013
    PubMed: PMID 22869151 · NCT05063110 (HLH-JAK)
  2. Neutralization of IFNgamma defeats haemophagocytosis in LCMV-infected perforin- and Rab27a-deficient mice.
    Pachlopnik Schmid J, Ho CH, Chrétien F, et al. · EMBO Mol Med · 2010
    PubMed: PMID 20049711 · NCT05063110 (HLH-JAK)
  3. Primary hemophagocytic syndromes point to a direct link between lymphocyte cytotoxicity and homeostasis.
    Ménasché G, Feldmann J, Fischer A, et al. · Immunol Rev · 2005
    PubMed: PMID 15661029 · NCT05063110 (HLH-JAK)
  4. Therapeutic effect of JAK1/2 blockade on the manifestations of hemophagocytic lymphohistiocytosis in mice.
    Maschalidi S, Sepulveda FE, Garrigue A, et al. · Blood · 2017
    PubMed: PMID 27222478 · NCT05063110 (HLH-JAK)
  5. Macrophage activation syndrome: characteristic findings on liver biopsy illustrating the key role of activated, IFN-gamma-producing lymphocytes and IL-6- and TNF-alpha-producing macrophages.
    Billiau AD, Roskams T, Van Damme-Lombaerts R, et al. · Blood · 2005
    PubMed: PMID 15466922 · NCT05063110 (HLH-JAK)
  6. Efficacy and safety of itacitinib versus placebo in combination with corticosteroids for initial treatment of acute graft-versus-host disease (GRAVITAS-301): a randomised, multicentre, double-blind, phase 3 trial.
    Zeiser R, Socié G, Schroeder MA, et al. · Lancet Haematol · 2022
    PubMed: PMID 34971577 · NCT03139604 · Graft vs Host Disease
  7. The role of the molecular tumor board: learnings from the ROME trial.
    Marchetti P, Curigliano G, Westphalen CB, et al. · NPJ Precis Oncol · 2026
    PubMed: PMID 41963505 · NCT04591431 (ROME) · Carcinoma, Non-Small-Cell Lung
  8. Genomically matched therapy in advanced solid tumors: the randomized phase 2 ROME trial.
    Marchetti P, Curigliano G, Biffoni M, et al. · Nat Med · 2025
    PubMed: PMID 41023484 · NCT04591431 (ROME) · Carcinoma, Non-Small-Cell Lung

Primary-outcome results across pivotal trials

Per-arm reported values from Phase 2/3 and Phase 3 trials with results posted to ClinicalTrials.gov.

TrialIndicationPrimary endpointArmValue
NCT03139604Graft vs Host DiseaseOverall Response Rate Based on Center for International Blood and Marrow Transplant Research (CIBMTR) Response Index
Day 28
Itacitinib Plus Corticosteroids74.0 Percentage of Participants
Placebo Plus Corticosteroids66.4 Percentage of Participants
NCT03584516Graft vs Host DiseasePart 1 Expansion: Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
until at least 30 days after the last dose of study treatment (up to 1103 days)
Part 1 Expansion: CS Monotherapy32 Participants
Part 1 Expansion: Itacitinib 300 mg BID + CS28 Participants
Part 1 Expansion: Itacitinib 300 mg QD + CS34 Participants
Part 1 Expansion: Itacitinib 400 mg QD + CS38 Participants
NCT03584516Graft vs Host DiseasePart 1: Number of Participants With Dose-limiting Toxicities (DLTs)
up to Day 28
Part 1: Itacitinib 200 mg QD + CS0 Participants
Part 1: Itacitinib 300 mg QD + CS1 Participants

Publications by year

2001–2026: 37 publications.

2001
1
2005
2
2006
1
2009
1
2010
1
2011
1
2012
1
2013
3
2014
1
2016
1
2017
4
2019
5
2020
1
2021
2
2022
4
2023
2
2024
1
2025
3
2026
2

Publications by indication

Scleroderma, Systemic (14)

  • Belimumab for the Treatment of Early Diffuse Systemic Sclerosis: Results of a Randomized, Double-Blind, Placebo-Controlled, Pilot Trial.
    Arthritis Rheumatol · 2019 · PMID 29073351 · NCT04789850
  • Review: Frontiers of Antifibrotic Therapy in Systemic Sclerosis.
    Arthritis Rheumatol · 2019 · PMID 27636741 · NCT04789850
  • The use of JAK inhibitors in the treatment of progressive systemic sclerosis.
    J Eur Acad Dermatol Venereol · 2019 · PMID 29444362 · NCT04789850
  • JAK1-dependent transphosphorylation of JAK2 limits the antifibrotic effects of selective JAK2 inhibitors on long-term treatment.
    Ann Rheum Dis · 2017 · PMID 28478401 · NCT04789850
  • Polymorphisms in STAT4 and IRF5 increase the risk of systemic sclerosis: a meta-analysis.
    Int J Dermatol · 2017 · PMID 26712637 · NCT04789850

Graft vs Host Disease (4)

  • Effective treatment of low-risk acute GVHD with itacitinib monotherapy.
    Blood · 2023 · PMID 36095841 · NCT03846479
  • Efficacy and safety of itacitinib versus placebo in combination with corticosteroids for initial treatment of acute graft-versus-host disease (GRAVITAS-301): a randomised, multicentre, double-blind, phase 3 trial.
    Lancet Haematol · 2022 · PMID 34971577 · NCT03139604
  • A biomarker signature to predict complete response to itacitinib and corticosteroids in acute graft-versus-host disease.
    Br J Haematol · 2022 · PMID 35689489 · NCT03139604
  • A phase 1 trial of itacitinib, a selective JAK1 inhibitor, in patients with acute graft-versus-host disease.
    Blood Adv · 2021 · PMID 32324888 · NCT02614612

Carcinoma, Non-Small-Cell Lung (2)

  • The role of the molecular tumor board: learnings from the ROME trial.
    NPJ Precis Oncol · 2026 · PMID 41963505 · NCT04591431
  • Genomically matched therapy in advanced solid tumors: the randomized phase 2 ROME trial.
    Nat Med · 2025 · PMID 41023484 · NCT04591431

Cytokine Release Syndrome (2)

  • Itacitinib for the prevention of IEC therapy-associated CRS: results from the 2-part phase 2 INCB 39110-211 study.
    Blood · 2025 · PMID 40090005 · NCT04071366
  • Itacitinib (INCB039110), a JAK1 Inhibitor, Reduces Cytokines Associated with Cytokine Release Syndrome Induced by CAR T-cell Therapy.
    Clin Cancer Res · 2021 · PMID 32998963 · NCT04071366

Breast Neoplasms (1)

  • Combination of Itacitinib or Parsaclisib with Pembrolizumab in Patients with Advanced Solid Tumors: A Phase I Study.
    Cancer Res Commun · 2023 · PMID 38115208 · NCT02646748

Myeloproliferative Disorders (1)

  • Primary analysis of a phase II open-label trial of INCB039110, a selective JAK1 inhibitor, in patients with myelofibrosis.
    Haematologica · 2017 · PMID 27789678 · NCT01633372

Publications by journal

Trial-results highlights

In a study evaluating Graft vs Host Disease, NCT03139604, the overall response rate based on the Center for International Blood and Marrow Transplant Research (CIBMTR) Response Index at Day 28 was assessed. In the Itacitinib Plus Corticosteroids arm, 74.0 percentage of participants achieved this response. In the Placebo Plus Corticosteroids arm of NCT03139604, 66.4 percentage of participants achieved the overall response rate.

Another study, NCT03584516, investigated the number of participants with any treatment-emergent adverse event (TEAE) in its Part 1 Expansion phase. This was measured until at least 30 days after the last dose of study treatment, up to 1103 days. The reported numbers of participants with any TEAE were:

  • In the Part 1 Expansion: CS Monotherapy arm of NCT03584516, 32 participants.
  • In the Part 1 Expansion: Itacitinib 300 mg BID + CS arm of NCT03584516, 28 participants.
  • In the Part 1 Expansion: Itacitinib 300 mg QD + CS arm of NCT03584516, 34 participants.
  • In the Part 1 Expansion: Itacitinib 400 mg QD + CS arm of NCT03584516, 38 participants.

In the Part 1 phase of NCT03584516, the number of participants with dose-limiting toxicities (DLTs) up to Day 28 was also reported. In the Part 1: Itacitinib 200 mg QD + CS arm, 0 participants experienced DLTs. In the Part 1: Itacitinib 300 mg QD + CS arm of NCT03584516, 1 participant experienced DLTs.

All values presented are sourced from primary registry reporting; individual papers should be consulted for clinical decisions.

All Itacitinib publications (37)

2026 (2 papers)

  1. The role of the molecular tumor board: learnings from the ROME trial.
    Marchetti P, Curigliano G, Westphalen CB, et al. · NPJ Precis Oncol · 2026 · Derived
    PubMed: PMID 41963505 · NCT04591431 (ROME) · Carcinoma, Non-Small-Cell Lung
  2. Phase 1B pilot study of itacitinib with alemtuzumab in patients with T-cell prolymphocytic leukemia.
    Kadia TM, Jain A, Rausch CR, et al. · Blood Neoplasia · 2026 · Derived
    PubMed: PMID 41536780 · NCT03989466 · Leukemia, Prolymphocytic, T-Cell

2025 (3 papers)

  1. Genomically matched therapy in advanced solid tumors: the randomized phase 2 ROME trial.
    Marchetti P, Curigliano G, Biffoni M, et al. · Nat Med · 2025 · Derived
    PubMed: PMID 41023484 · NCT04591431 (ROME) · Carcinoma, Non-Small-Cell Lung
  2. Itacitinib for the prevention of IEC therapy-associated CRS: results from the 2-part phase 2 INCB 39110-211 study.
    Frigault MJ, Maziarz RT, Park JH, et al. · Blood · 2025 · Derived
    PubMed: PMID 40090005 · NCT04071366 · Cytokine Release Syndrome
  3. Itacitinib for prevention of graft-versus-host disease and cytokine release syndrome in haploidentical transplantation.
    Abboud R, Schroeder MA, Rettig MP, et al. · Blood · 2025 · Derived
    PubMed: PMID 39576962 · NCT03755414 · Myelodysplastic Syndromes

2024 (1 paper)

  1. Safety and efficacy of itacitinib, a selective JAK1 inhibitor, in advanced hepatocellular cancer: Phase 1b trial (JAKAL).
    Troiani A, Martinez M, Ward C, et al. · Future Oncol · 2024 · Background
    PubMed: PMID 39283290 · NCT04358185 (JAKaL)

2023 (2 papers)

  1. Combination of Itacitinib or Parsaclisib with Pembrolizumab in Patients with Advanced Solid Tumors: A Phase I Study.
    Munster P, Iannotti N, Cho DC, et al. · Cancer Res Commun · 2023 · Derived
    PubMed: PMID 38115208 · NCT02646748 · Breast Neoplasms
  2. Effective treatment of low-risk acute GVHD with itacitinib monotherapy.
    Etra A, Capellini A, Alousi A, et al. · Blood · 2023 · Derived
    PubMed: PMID 36095841 · NCT03846479 · Graft vs Host Disease

2022 (4 papers)

  1. Efficacy and safety of itacitinib versus placebo in combination with corticosteroids for initial treatment of acute graft-versus-host disease (GRAVITAS-301): a randomised, multicentre, double-blind, phase 3 trial.
    Zeiser R, Socié G, Schroeder MA, et al. · Lancet Haematol · 2022 · Derived
    PubMed: PMID 34971577 · NCT03139604 · Graft vs Host Disease
  2. A biomarker signature to predict complete response to itacitinib and corticosteroids in acute graft-versus-host disease.
    Pratta M, Paczesny S, Socie G, et al. · Br J Haematol · 2022 · Derived
    PubMed: PMID 35689489 · NCT03139604 · Graft vs Host Disease
  3. Exploring the safety, effect on the tumor microenvironment, and efficacy of itacitinib in combination with epacadostat or parsaclisib in advanced solid tumors: a phase I study.
    Naing A, Powderly JD, Nemunaitis JJ, et al. · J Immunother Cancer · 2022 · Derived
    PubMed: PMID 35288468 · NCT02559492
  4. The ABNL-MARRO 001 study: a phase 1-2 study of randomly allocated active myeloid target compound combinations in MDS/MPN overlap syndromes.
    Moyo TK, Mendler JH, Itzykson R, et al. · BMC Cancer · 2022 · Background
    PubMed: PMID 36153475 · NCT04061421 (ABNL-MARRO)

2021 (2 papers)

  1. A phase 1 trial of itacitinib, a selective JAK1 inhibitor, in patients with acute graft-versus-host disease.
    Schroeder MA, Khoury HJ, Jagasia M, et al. · Blood Adv · 2021 · Derived
    PubMed: PMID 32324888 · NCT02614612 · Graft vs Host Disease
  2. Itacitinib (INCB039110), a JAK1 Inhibitor, Reduces Cytokines Associated with Cytokine Release Syndrome Induced by CAR T-cell Therapy.
    Huarte E, O'Connor RS, Peel MT, et al. · Clin Cancer Res · 2021 · Derived
    PubMed: PMID 32998963 · NCT04071366 · Cytokine Release Syndrome

2020 (1 paper)

  1. A Phase Ib/II Study of the JAK1 Inhibitor, Itacitinib, plus nab-Paclitaxel and Gemcitabine in Advanced Solid Tumors.
    Beatty GL, Shahda S, Beck T, et al. · Oncologist · 2020 · Derived
    PubMed: PMID 30115734 · NCT01858883 · Pancreatic Neoplasms

2019 (5 papers)

  1. Phase 1 study of the PI3Kδ inhibitor INCB040093 ± JAK1 inhibitor itacitinib in relapsed/refractory B-cell lymphoma.
    Phillips TJ, Forero-Torres A, Sher T, et al. · Blood · 2019 · Derived
    PubMed: PMID 29695516 · NCT01905813
  2. Parsaclisib, a potent and highly selective PI3Kδ inhibitor, in patients with relapsed or refractory B-cell malignancies.
    Forero-Torres A, Ramchandren R, Yacoub A, et al. · Blood · 2019 · Derived
    PubMed: PMID 30803990 · NCT02018861
  3. Belimumab for the Treatment of Early Diffuse Systemic Sclerosis: Results of a Randomized, Double-Blind, Placebo-Controlled, Pilot Trial.
    Gordon JK, Martyanov V, Franks JM, et al. · Arthritis Rheumatol · 2019 · Background
    PubMed: PMID 29073351 · NCT04789850 (SCLERITA) · Scleroderma, Systemic
  4. Review: Frontiers of Antifibrotic Therapy in Systemic Sclerosis.
    Distler JH, Feghali-Bostwick C, Soare A, et al. · Arthritis Rheumatol · 2019 · Background
    PubMed: PMID 27636741 · NCT04789850 (SCLERITA) · Scleroderma, Systemic
  5. The use of JAK inhibitors in the treatment of progressive systemic sclerosis.
    Deverapalli SC, Rosmarin D · J Eur Acad Dermatol Venereol · 2019 · Background
    PubMed: PMID 29444362 · NCT04789850 (SCLERITA) · Scleroderma, Systemic

2017 (4 papers)

  1. Therapeutic effect of JAK1/2 blockade on the manifestations of hemophagocytic lymphohistiocytosis in mice.
    Maschalidi S, Sepulveda FE, Garrigue A, et al. · Blood · 2017 · Trial result
    PubMed: PMID 27222478 · NCT05063110 (HLH-JAK)
  2. Primary analysis of a phase II open-label trial of INCB039110, a selective JAK1 inhibitor, in patients with myelofibrosis.
    Mascarenhas JO, Talpaz M, Gupta V, et al. · Haematologica · 2017 · Derived
    PubMed: PMID 27789678 · NCT01633372 · Myeloproliferative Disorders
  3. JAK1-dependent transphosphorylation of JAK2 limits the antifibrotic effects of selective JAK2 inhibitors on long-term treatment.
    Zhang Y, Liang R, Chen CW, et al. · Ann Rheum Dis · 2017 · Background
    PubMed: PMID 28478401 · NCT04789850 (SCLERITA) · Scleroderma, Systemic
  4. Polymorphisms in STAT4 and IRF5 increase the risk of systemic sclerosis: a meta-analysis.
    Xu Y, Wang W, Tian Y, et al. · Int J Dermatol · 2017 · Background
    PubMed: PMID 26712637 · NCT04789850 (SCLERITA) · Scleroderma, Systemic

2016 (1 paper)

  1. Quality of life in systemic sclerosis.
    Almeida C, Almeida I, Vasconcelos C, et al. · Autoimmun Rev · 2016 · Background
    PubMed: PMID 26212726 · NCT04789850 (SCLERITA) · Scleroderma, Systemic

2014 (1 paper)

  1. Inhibition of Janus kinase/signal transducer and activator of transcription (JAK/STAT) signalling pathway in rheumatoid synovial fibroblasts using small molecule compounds.
    Migita K, Izumi Y, Torigoshi T, et al. · Clin Exp Immunol · 2014 · Background
    PubMed: PMID 23968543 · NCT04789850 (SCLERITA) · Scleroderma, Systemic

2013 (3 papers)

  1. JAK/STAT signaling in hematological malignancies.
    Vainchenker W, Constantinescu SN · Oncogene · 2013 · Trial result
    PubMed: PMID 22869151 · NCT05063110 (HLH-JAK)
  2. Towards a functional proteomics approach to the comprehension of idiopathic pulmonary fibrosis, sarcoidosis, systemic sclerosis and pulmonary Langerhans cell histiocytosis.
    Landi C, Bargagli E, Bianchi L, et al. · J Proteomics · 2013 · Background
    PubMed: PMID 23528693 · NCT04789850 (SCLERITA) · Scleroderma, Systemic
  3. Inactivation of the transcription factor STAT-4 prevents inflammation-driven fibrosis in animal models of systemic sclerosis.
    Avouac J, Fürnrohr BG, Tomcik M, et al. · Arthritis Rheum · 2013 · Background
    PubMed: PMID 21360510 · NCT04789850 (SCLERITA) · Scleroderma, Systemic

2012 (1 paper)

  1. SSc in 2011: From mechanisms to medicines.
    Mouthon L · Nat Rev Rheumatol · 2012 · Background
    PubMed: PMID 22231235 · NCT04789850 (SCLERITA) · Scleroderma, Systemic

2011 (1 paper)

  1. Preclinical evaluation of local JAK1 and JAK2 inhibition in cutaneous inflammation.
    Fridman JS, Scherle PA, Collins R, et al. · J Invest Dermatol · 2011 · Background
    PubMed: PMID 21677670 · NCT04789850 (SCLERITA) · Scleroderma, Systemic

2010 (1 paper)

  1. Neutralization of IFNgamma defeats haemophagocytosis in LCMV-infected perforin- and Rab27a-deficient mice.
    Pachlopnik Schmid J, Ho CH, Chrétien F, et al. · EMBO Mol Med · 2010 · Trial result
    PubMed: PMID 20049711 · NCT05063110 (HLH-JAK)

2009 (1 paper)

  1. The safety and efficacy of a JAK inhibitor in patients with active rheumatoid arthritis: Results of a double-blind, placebo-controlled phase IIa trial of three dosage levels of CP-690,550 versus placebo.
    Kremer JM, Bloom BJ, Breedveld FC, et al. · Arthritis Rheum · 2009 · Background
    PubMed: PMID 19565475 · NCT04789850 (SCLERITA) · Scleroderma, Systemic

2006 (1 paper)

  1. Association between enhanced type I collagen expression and epigenetic repression of the FLI1 gene in scleroderma fibroblasts.
    Wang Y, Fan PS, Kahaleh B, et al. · Arthritis Rheum · 2006 · Background
    PubMed: PMID 16802366 · NCT04789850 (SCLERITA) · Scleroderma, Systemic

2005 (2 papers)

  1. Primary hemophagocytic syndromes point to a direct link between lymphocyte cytotoxicity and homeostasis.
    Ménasché G, Feldmann J, Fischer A, et al. · Immunol Rev · 2005 · Trial result
    PubMed: PMID 15661029 · NCT05063110 (HLH-JAK)
  2. Macrophage activation syndrome: characteristic findings on liver biopsy illustrating the key role of activated, IFN-gamma-producing lymphocytes and IL-6- and TNF-alpha-producing macrophages.
    Billiau AD, Roskams T, Van Damme-Lombaerts R, et al. · Blood · 2005 · Trial result
    PubMed: PMID 15466922 · NCT05063110 (HLH-JAK)

2001 (1 paper)

  1. Up-regulated expression of transforming growth factor beta receptors in dermal fibroblasts in skin sections from patients with localized scleroderma.
    Kubo M, Ihn H, Yamane K, et al. · Arthritis Rheum · 2001 · Background
    PubMed: PMID 11263790 · NCT04789850 (SCLERITA) · Scleroderma, Systemic

Sources and methodology

This page summarizes published evidence for general reference and does not constitute medical advice. For clinical decisions, consult the linked primary publications and your healthcare provider. Data sourced from PubMed and the ClinicalTrials.gov / AACT database maintained by the Clinical Trials Transformation Initiative (CTTI).