A Phase 2/3 interventional study of Itacitinib and Placebo in Chronic Graft-versus-host Disease, sponsored by Incyte Corporation. Terminated at 133 sites in 16 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-10-20.
Sponsored by Incyte Corporation · Phase 2/3, Interventional, and Treatment
The purpose of this study is to assess the efficacy and safety of itacitinib in combination with corticosteroids as first-line treatment for moderate or severe chronic graft-versus-host disease (cGVHD).
376 studies on the registry are indexed under Bronchiolitis Obliterans Syndrome; 104 are open to participants now.
This study's enrollment of 155 is above the median of 35 across 296 interventional studies indexed under Bronchiolitis Obliterans Syndrome.
Browse Bronchiolitis Obliterans Syndrome studies →Incyte Corporation is the lead sponsor of 286 studies on the registry; 37 are open to participants now.
Of its 144 completed or terminated interventional studies of FDA-regulated products, 93 (65%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
itacitinib administered in combination with corticosteroids.
Drug: Itacitinib · Drug: Methylprednisolone · Drug: Prednisone
itacitinib administered in combination with corticosteroids or corticosteroids alone.
Drug: Itacitinib · Drug: Placebo · Drug: Methylprednisolone · Drug: Prednisone
itacitinib or placebo administered in combination with corticosteroids
Drug: Itacitinib · Drug: Methylprednisolone · Drug: Prednisone
In Part 1dose determination participants will receive itacitinib administered orally once daily at the protocol-defined dose according to cohort enrollment. In Part 1 expansion, participants will receive either itacitinib administered orally either once daily or twice a day or corticosteroid alone based on the assigned treatment regimen according to cohort enrollment. In Part 2, participants will receive the recommended dose from Part 1 expansion.
Also known as: INCB039110
In Part 2, participants will receive matching placebo.
Administered in Parts 1 and 2 as background reference therapy at a dose level that is commensurate with institutional guidelines based on organ involvement and severity of disease.
Also known as: Medrol, Medrol Dosepak, Solu-Medrol
Administered in Parts 1 and 2 as background reference therapy at a dose level that is commensurate with institutional guidelines based on organ involvement and severity of disease.
Also known as: Deltasone, Prednicot, predniSONE Intensol, Rayos, Sterapred, Sterapred DS
Part 1: Number of Participants With Dose-limiting Toxicities (DLTs)
A DLT was defined as the occurrence of any protocol-defined toxicity with onset up to and including Day 28, except those with a clear alternative explanation. Participants who received at least 21 of 28 doses of study drug at the level assigned or had a DLT were considered evaluable for determining tolerability of the dose. Participants who did not achieve this duration of exposure and did not have a DLT were to be replaced for purposes of toxicity identification.
Time frame: up to Day 28
Part 1 Expansion: Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE could therefore have been any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug.
Time frame: until at least 30 days after the last dose of study treatment (up to 1103 days)
Part 2: Response Rate at Month 6
Response rate was defined as the percentage of participants that had complete response (CR) or partial response (PR), per National Institutes of Health (NIH) Consensus Criteria, as determined by the investigator, within 14 days of the post-Baseline visit date until new anti-GVHD therapy or overall response-progression or relapse/progression of underlying disease. CR was defined as the complete resolution of all signs and symptoms of cGvHD in all evaluable organs. PR was defined as an improvement in at least one organ without progression in other organs.
Time frame: Month 6
Part 1 Expansion: Response Rate at Months 3 and 6
Response rate was defined as the percentage of participants that had CR or PR, per NIH Consensus Criteria, as determined by the investigator, within 14 days of the post-Baseline visit date until new anti-GVHD therapy or overall response-progression or relapse/progression of underlying disease. CR was defined as the complete resolution of all signs and symptoms of cGvHD in all evaluable organs. PR was defined as an improvement in at least one organ without progression in other organs.
Time frame: Months 3 and 6
Parts 1 and 1 Expansion: Cmax of Itacitinib
Cmax was defined as the maximum observed concentration of itacitinib.
Time frame: Days 1, 7, and 28: predose and 1, 2, and 5 hours post-dose
Parts 1 and 1 Expansion: Ctau of Itacitinib
Ctau was defined as the trough concentration of itacitinib over the dose interval. For pharmacokinetic steady state Day 7 and Day 28, for the calculation of NCA exposure estimates (as more than 3 PK data points are necessary for the estimation beyond Cmax) it was assumed, that PK concentration returns to the predose value (at 12 hours post-dose for BID dosing; at 24 hours post-dose for QD dosing). Predose PK samples were transposed to 24 hours post-dose for QD administration and to 12 hours post-dose for BID administration to allow the steady-state NCA PK estimates on Day 7 and Day 28.
Time frame: Day 1: predose, and 1, 2, and 5 hours post-dose. Days 7 and 28: predose, and 1, 2, 5, 12 (for BID dosing), and 24 hours post-dose (for QD dosing)
Parts 1 and 1 Expansion: Tmax of Itacitinib
tmax was defined as the time to the maximum concentration of itacitinib. For pharmacokinetic steady state Day 7 and Day 28, for the calculation of NCA exposure estimates (as more than 3 PK data points are necessary for the estimation beyond Cmax) it was assumed, that PK concentration returns to the predose value (at 12 hours post-dose for BID dosing; at 24 hours post-dose for QD dosing). Predose PK samples were transposed to 24 hours post-dose for QD administration and to 12 hours post-dose for BID administration to allow the steady-state NCA PK estimates on Day 7 and Day 28.
Time frame: Day 1: predose, and 1, 2, and 5 hours post-dose. Days 7 and 28: predose, and 1, 2, 5, 12 (for BID dosing), and 24 hours post-dose (for QD dosing)
Parts 1 and 1 Expansion: Cl/F of Itacitinib
Cl/F was defined as the apparent oral dose clearance of itacitinib. For pharmacokinetic steady state Day 7 and Day 28, for the calculation of NCA exposure estimates (as more than 3 PK data points are necessary for the estimation beyond Cmax) it was assumed, that PK concentration returns to the predose value (at 12 hours post-dose for BID dosing; at 24 hours post-dose for QD dosing). Predose PK samples were transposed to 24 hours post-dose for QD administration and to 12 hours post-dose for BID administration to allow the steady-state NCA PK estimates on Day 7 and Day 28.
Time frame: Day 1: predose, and 1, 2, and 5 hours post-dose. Days 7 and 28: predose, and 1, 2, 5, 12 (for BID dosing), and 24 hours post-dose (for QD dosing)
Part 2: Cmax of Itacitinib
Cmax was defined as the maximum observed concentration of itacitinib.
Time frame: Days 1, 7, and 28: predose and 1, 2, and 5 hours post-dose
Part 2: Cmin of Itacitinib
Cmin was defined as the minimum observed plasma or serum concentration of itacitinib over the dose interval.
Time frame: Days 1, 7, and 28: predose and 1, 2, and 5 hours post-dose
Part 2: Tmax of Itacitinib
tmax was defined as the time to the maximum concentration of itacitinib.
Time frame: Days 1, 7, and 28: predose and 1, 2, and 5 hours post-dose
Part 2: AUC0-t of Itacitinib
AUC0-t was defined as the area under the plasma or serum concentration-time curve from time = 0 to the last measurable concentration at time = t.
Time frame: Days 1, 7, and 28: predose and 1, 2, and 5 hours post-dose
Part 2: Cl/F of Itacitinib
Cl/F was defined as the apparent oral dose clearance of itacitinib.
Time frame: Days 1, 7, and 28: predose and 1, 2, and 5 hours post-dose
Part 1: Response Rate at Months 3, 6, and 12
Response rate was defined as the percentage of participants that had CR or PR, per NIH Consensus Criteria, as determined by the investigator, within 14 days of the post-Baseline visit date until new anti-GVHD therapy or overall response-progression or relapse/progression of underlying disease. CR was defined as the complete resolution of all signs and symptoms of cGvHD in all evaluable organs. PR was defined as an improvement in at least one organ without progression in other organs.
Time frame: Months 3, 6, and 12
Part 1 Expansion: Response Rate at Month 12
Response rate was defined as the percentage of participants that had CR or PR, per NIH Consensus Criteria, as determined by the investigator, within 14 days of the post-Baseline visit date until new anti-GVHD therapy or overall response-progression or relapse/progression of underlying disease. CR was defined as the complete resolution of all signs and symptoms of cGvHD in all evaluable organs. PR was defined as an improvement in at least one organ without progression in other organs.
Time frame: Month 12
Part 1 Expansion: Time to Response
Time to response was defined as the interval between randomization and the first response (CR or PR) before initiation of new therapy. CR was defined as the complete resolution of all signs and symptoms of cGvHD in all evaluable organs. PR was defined as an improvement in at least one organ without progression in other organs.
Time frame: up to Month 12
Part 1 Expansion: Duration of Response
Duration to response was defined as the interval between the first response and cGVHD progression, death, or the initiation of a new systemic cGVHD therapy.
Time frame: up to 24 months
Part 1 Expansion: Overall Survival
Overall survival was defined as the interval between the date of randomization and the date of death due to any cause.
Time frame: up to 36 months
Part 1 Expansion: Nonrelapse Mortality (NRM) Rate
NRM was defined as the percentage of participants who died due to causes other than a relapse of their primary hematologic disease.
Time frame: up to 24 months
Part 1 Expansion: Percentage of Participants With a ≥50% Reduction in Daily Corticosteroid Dose at Day 180 From the Corticosteroid Dose on Day 1
The corticosteroid dose at Day 180 was compared to the corticosteroid dose on Day 1 to assess reduction.
Time frame: Day 1; Day 180
Part 1 Expansion: Percentage of Participants Successfully Tapered Off All Corticosteroids at Day 180
The percentage of participants who were not taking any corticosteroids at Day 180 was assessed.
Time frame: Day 180
Part 1 Expansion: Relapse Rate of Malignant and Nonmalignant Hematologic Diseases
The relapse rate was defined as the percentage of participants whose underlying disease relapsed at any time during the course of the study.
Time frame: up to 1073 days
Part 1 Expansion: Time to Primary Hematologic Disease Relapse
Time to primary hematologic disease relapse was defined as the interval between the date of randomization and the date of relapse.
Time frame: up to 24 months
Part 2: Change From Baseline in Lee cGVHD Symptom Scale (LLS) Scores
The LSS consists of 30 items in 7 subscales (skin, eye, mouth, lung, nutrition, energy, and psychological). It was to be used to assess patient-reported changes in health status, symptoms, and well being.
Time frame: Baseline; End of Treatment in Phase 2
Part 2: Change From Baseline in Quality of Life-Short Form-36 Version 2 (QOL-SF-36 v2) Scores
The QOL-SF-36 v2 is 36-item scale that captures changes in health status during the course of treatment. The SF-36 assesses 8 health concepts related to limitations in physical activities, social activities, bodily pain, general mental and physical health, and vitality. It was to be used to assess patient-reported changes in health status, symptoms, and well being.
Time frame: Baseline; End of Treatment in Phase 2
Part 2: Change From Baseline in EQ-5D-3L Scores
The EQ-5D-3L is a descriptive classification consisting of 5 dimensions of health: mobility, self-care, usual activities, anxiety/depression, and pain/discomfort. It was to be used to assess patient-reported changes in health status, symptoms, and well being.
Time frame: Baseline; End of Treatment in Phase 2
Part 2: Change From Baseline in Patient Global Impression of Change (PGIC) Responses
The PGIC is 1 question that captures the overall change in symptoms over the course of treatment. It was to be used to assess patient-reported changes in health status, symptoms, and well being.
Time frame: Baseline; End of Treatment in Phase 2
Part 2: Change From Baseline in Patient Global Impression of Severity (PGIS) Responses
The PGIS is 1 question that captures the overall change in the severity of symptoms over the previous week. It was to be used to assess patient-reported changes in health status, symptoms, and well being.
Time frame: Baseline; End of Treatment in Phase 2
Part 2: Response Rate at Months 3 and 12
Response rate was defined as the percentage of participants that had CR or PR, per NIH Consensus Criteria, as determined by the investigator, within 14 days of the post-Baseline visit date until new anti-GVHD therapy or overall response-progression or relapse/progression of underlying disease. CR was defined as the complete resolution of all signs and symptoms of cGvHD in all evaluable organs. PR was defined as an improvement in at least one organ without progression in other organs.
Time frame: Months 3 and 12
Part 2: Duration of Response
Duration to response was defined as the interval between the first response and cGVHD progression, death, or the initiation of a new systemic cGVHD therapy.
Time frame: up to 24 months
Part 2: Overall Survival
Overall survival was defined as the interval between the date of randomization and the date of death due to any cause.
Time frame: up to 36 months
Part 2: NRM Rate
NRM was defined as the percentage of participants who died due to causes other than a relapse of their primary hematologic disease.
Time frame: up to 24 months
Part 2: Percentage of Participants With a ≥50% Reduction in Daily Corticosteroid Dose at Day 180 From the Corticosteroid Dose on Day 1
The corticosteroid dose at Day 180 was compared to the corticosteroid dose on Day 1 to assess reduction.
Time frame: Day 1; Day 180
Part 2: Percentage of Participants Successfully Tapered Off All Corticosteroids at Day 180
The percentage of participants who were not taking any corticosteroids at Day 180 was assessed.
Time frame: Day 180
Part 2: Relapse Rate of Malignant and Nonmalignant Hematologic Diseases
The relapse rate was defined as the defined as the percentage of participants whose underlying disease relapsed at any time during the course of the study.
Time frame: up to 24 months
Part 2: Time to Primary Hematologic Disease Relapse
Time to primary hematologic disease relapse was defined as the interval between the date of randomization and the date of relapse.
Time frame: up to 24 months
Part 2: Number of Participants With Any TEAE
An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE could therefore have been any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug.
Time frame: up to 30 days after the last dose in Phase 2
| Milestone | Part 1: Itacitinib 200 mg QD + CS | Part 1: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy |
|---|---|---|---|---|---|---|
| Started | 11 | 10 | 0 | 0 | 0 | 0 |
| Completed | 6 | 5 | 0 | 0 | 0 | 0 |
| Not completed | 5 | 5 | 0 | 0 | 0 | 0 |
| Withdrew: Death | 2 | 3 | 0 | 0 | 0 | 0 |
| Withdrew: Withdrawal by subject | 2 | 0 | 0 | 0 | 0 | 0 |
| Withdrew: Lost to follow-up | 1 | 0 | 0 | 0 | 0 | 0 |
| Withdrew: Study terminated by sponsor | 0 | 2 | 0 | 0 | 0 | 0 |
| Milestone | Part 1: Itacitinib 200 mg QD + CS | Part 1: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy |
|---|---|---|---|---|---|---|
| Started | 0 | 0 | 35 | 39 | 29 | 36 |
| Completed | 0 | 0 | 3 | 2 | 1 | 1 |
| Not completed | 0 | 0 | 32 | 37 | 28 | 35 |
| Withdrew: Death | 0 | 0 | 9 | 8 | 4 | 5 |
| Withdrew: Lost to follow-up | 0 | 0 | 0 | 1 | 0 | 0 |
| Withdrew: Physician decision | 0 | 0 | 4 | 3 | 2 | 3 |
| Withdrew: Study terminated by sponsor | 0 | 0 | 17 | 20 | 20 | 23 |
| Withdrew: Withdrawal by subject | 0 | 0 | 2 | 4 | 2 | 3 |
| Withdrew: Discontinued treatment and terminated cs tapering | 0 | 0 | 0 | 1 | 0 | 0 |
| Withdrew: Disease progression; cgvhd flare | 0 | 0 | 0 | 0 | 0 | 1 |
A DLT was defined as the occurrence of any protocol-defined toxicity with onset up to and including Day 28, except those with a clear alternative explanation. Participants who received at least 21 of 28 doses of study drug at the level assigned or had a DLT were considered evaluable for determining tolerability of the dose. Participants who did not achieve this duration of exposure and did not have a DLT were to be replaced for purposes of toxicity identification.
| Participants | Part 1: Itacitinib 200 mg QD + CS | Part 1: Itacitinib 300 mg QD + CS |
|---|---|---|
| Part 1: Number of Participants With Dose-limiting Toxicities (DLTs) | 0 | 1 |
An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE could therefore have been any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug.
| Participants | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy |
|---|---|---|---|---|
| Part 1 Expansion: Number of Participants With Any Treatment-emergent Adverse Event (TEAE) | 34 | 38 | 28 | 32 |
Response rate was defined as the percentage of participants that had complete response (CR) or partial response (PR), per National Institutes of Health (NIH) Consensus Criteria, as determined by the investigator, within 14 days of the post-Baseline visit date until new anti-GVHD therapy or overall response-progression or relapse/progression of underlying disease. CR was defined as the complete resolution of all signs and symptoms of cGvHD in all evaluable organs. PR was defined as an improvement in at least one organ without progression in other organs.
No measurements were reported for this outcome.
Response rate was defined as the percentage of participants that had CR or PR, per NIH Consensus Criteria, as determined by the investigator, within 14 days of the post-Baseline visit date until new anti-GVHD therapy or overall response-progression or relapse/progression of underlying disease. CR was defined as the complete resolution of all signs and symptoms of cGvHD in all evaluable organs. PR was defined as an improvement in at least one organ without progression in other organs.
| percentage of participants | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy |
|---|---|---|---|---|
| Month 3 | 60.0 (42.1 to 76.1) | 69.2 (52.4 to 83.0) | 58.6 (38.9 to 76.5) | 50.0 (32.9 to 67.1) |
| Month 6 | 42.9 (26.3 to 60.6) | 53.8 (37.2 to 69.9) | 34.5 (17.9 to 54.3) | 36.1 (20.8 to 53.8) |
Cmax was defined as the maximum observed concentration of itacitinib.
| nanomoles per Liter (nmol/L) | Parts 1 and 1 Expansion: 100 mg QD + CS | Parts 1 and 1 Expansion: Itacitinib 200 mg QD + CS | Parts 1 and 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 100 mg BID + CS | Part 1 Expansion: Itacitinib 200 mg BID + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS |
|---|---|---|---|---|---|---|---|
| Day 1 | 201 ± NA | 655 ± 82.9 | 1050 ± 87.3 | 951 ± 68.0 | — | 769 ± 118 | 736 ± 79.1 |
| Day 7 | 191 ± NA | 1070 ± 88.9 | 1580 ± 125 | 1190 ± 103 | — | 1520 ± 73.0 | 1110 ± 66.2 |
| Day 28 | 486 ± 140 | 1460 ± 67.3 | 1290 ± 248 | 1350 ± 72.2 | 1350 ± 29.4 | 2040 ± 62.6 | 1580 ± 51.4 |
Ctau was defined as the trough concentration of itacitinib over the dose interval. For pharmacokinetic steady state Day 7 and Day 28, for the calculation of NCA exposure estimates (as more than 3 PK data points are necessary for the estimation beyond Cmax) it was assumed, that PK concentration returns to the predose value (at 12 hours post-dose for BID dosing; at 24 hours post-dose for QD dosing). Predose PK samples were transposed to 24 hours post-dose for QD administration and to 12 hours post-dose for BID administration to allow the steady-state NCA PK estimates on Day 7 and Day 28.
| nmol/L | Parts 1 and 1 Expansion: 100 mg QD + CS | Parts 1 and 1 Expansion: Itacitinib 200 mg QD + CS | Parts 1 and 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 100 mg BID + CS | Part 1 Expansion: Itacitinib 200 mg BID + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS |
|---|---|---|---|---|---|---|---|
| Day 1 | NA ± NA | NA ± NA | 0.153 ± NA | 135 ± 197 | — | 7.54 ± NA | NA ± NA |
| Day 7 | 0.298 ± NA | 53.6 ± 218 | 52.2 ± 425 | 33.5 ± 118 | — | 483 ± 175 | 127 ± 177 |
| Day 28 | 10.4 ± 4440 | 68.0 ± 314 | 42.2 ± 182 | 29.7 ± 152 | 392 ± 85.6 | 460 ± 123 | 195 ± 116 |
tmax was defined as the time to the maximum concentration of itacitinib. For pharmacokinetic steady state Day 7 and Day 28, for the calculation of NCA exposure estimates (as more than 3 PK data points are necessary for the estimation beyond Cmax) it was assumed, that PK concentration returns to the predose value (at 12 hours post-dose for BID dosing; at 24 hours post-dose for QD dosing). Predose PK samples were transposed to 24 hours post-dose for QD administration and to 12 hours post-dose for BID administration to allow the steady-state NCA PK estimates on Day 7 and Day 28.
| hours | Parts 1 and 1 Expansion: 100 mg QD + CS | Parts 1 and 1 Expansion: Itacitinib 200 mg QD + CS | Parts 1 and 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 100 mg BID + CS | Part 1 Expansion: Itacitinib 200 mg BID + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS |
|---|---|---|---|---|---|---|---|
| Day 1 | 1.0 (1.0 to 1.0) | 2.1 (0.9 to 4.8) | 2.1 (0.8 to 5.0) | 2.0 (0.0 to 5.0) | — | 2.0 (1.0 to 5.0) | 2.4 (1.0 to 5.0) |
| Day 7 | 1.0 (1.0 to 1.0) | 2.1 (0.0 to 5.0) | 2.0 (1.0 to 13.4) | 2.0 (1.0 to 5.2) | — | 1.9 (0.0 to 10.7) | 2.0 (0.9 to 5.0) |
| Day 28 | 4.0 (1.0 to 4.8) | 3.1 (0.8 to 12.0) | 2.0 (0.0 to 16.9) | 2.1 (0.9 to 5.0) | 2.0 (2.0 to 2.2) | 2.3 (2.0 to 5.6) | 2.0 (1.0 to 5.0) |
Cl/F was defined as the apparent oral dose clearance of itacitinib. For pharmacokinetic steady state Day 7 and Day 28, for the calculation of NCA exposure estimates (as more than 3 PK data points are necessary for the estimation beyond Cmax) it was assumed, that PK concentration returns to the predose value (at 12 hours post-dose for BID dosing; at 24 hours post-dose for QD dosing). Predose PK samples were transposed to 24 hours post-dose for QD administration and to 12 hours post-dose for BID administration to allow the steady-state NCA PK estimates on Day 7 and Day 28.
| Liters per hour | Parts 1 and 1 Expansion: 100 mg QD + CS | Parts 1 and 1 Expansion: Itacitinib 200 mg QD + CS | Parts 1 and 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 100 mg BID + CS | Part 1 Expansion: Itacitinib 200 mg BID + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS |
|---|---|---|---|---|---|---|---|
| Day 1 | NA ± NA | NA ± NA | 258 ± NA | 41.2 ± 130 | — | 640 ± NA | NA ± NA |
| Day 7 | 224 ± NA | 43.0 ± 105 | 48.9 ± 159 | 85.9 ± 90.7 | — | 31.8 ± 82.9 | 94.4 ± 61.3 |
| Day 28 | 47.7 ± 211 | 30.0 ± 96.9 | 54.8 ± 189 | 81.0 ± 65.1 | 20.2 ± 54.2 | 26.9 ± 75.5 | 67.5 ± 62.1 |
Cmax was defined as the maximum observed concentration of itacitinib.
No measurements were reported for this outcome.
Cmin was defined as the minimum observed plasma or serum concentration of itacitinib over the dose interval.
No measurements were reported for this outcome.
tmax was defined as the time to the maximum concentration of itacitinib.
No measurements were reported for this outcome.
AUC0-t was defined as the area under the plasma or serum concentration-time curve from time = 0 to the last measurable concentration at time = t.
No measurements were reported for this outcome.
Cl/F was defined as the apparent oral dose clearance of itacitinib.
No measurements were reported for this outcome.
Response rate was defined as the percentage of participants that had CR or PR, per NIH Consensus Criteria, as determined by the investigator, within 14 days of the post-Baseline visit date until new anti-GVHD therapy or overall response-progression or relapse/progression of underlying disease. CR was defined as the complete resolution of all signs and symptoms of cGvHD in all evaluable organs. PR was defined as an improvement in at least one organ without progression in other organs.
| percentage of participants | Part 1: Itacitinib 200 mg QD + CS | Part 1: Itacitinib 300 mg QD + CS |
|---|---|---|
| Month 3 | 63.6 (30.8 to 89.1) | 50.0 (18.7 to 81.3) |
| Month 6 | 36.4 (10.9 to 69.2) | 50.0 (18.7 to 81.3) |
| Month 12 | 18.2 (2.3 to 51.8) | 20.0 (2.5 to 55.6) |
Response rate was defined as the percentage of participants that had CR or PR, per NIH Consensus Criteria, as determined by the investigator, within 14 days of the post-Baseline visit date until new anti-GVHD therapy or overall response-progression or relapse/progression of underlying disease. CR was defined as the complete resolution of all signs and symptoms of cGvHD in all evaluable organs. PR was defined as an improvement in at least one organ without progression in other organs.
| percentage of participants | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy |
|---|---|---|---|---|
| Part 1 Expansion: Response Rate at Month 12 | 22.9 (10.4 to 40.1) | 41.0 (25.6 to 57.9) | 24.1 (10.3 to 43.5) | 19.4 (8.2 to 36.0) |
Time to response was defined as the interval between randomization and the first response (CR or PR) before initiation of new therapy. CR was defined as the complete resolution of all signs and symptoms of cGvHD in all evaluable organs. PR was defined as an improvement in at least one organ without progression in other organs.
| days | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy |
|---|---|---|---|---|
| Part 1 Expansion: Time to Response | 16.0 (12 to 120) | 16.0 (12 to 86) | 16.0 (13 to 145) | 16.0 (13 to 88) |
Duration to response was defined as the interval between the first response and cGVHD progression, death, or the initiation of a new systemic cGVHD therapy.
| days | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy |
|---|---|---|---|---|
| Part 1 Expansion: Duration of Response | 581.0 (147.0 to 807.0) | NA (306.0 to NA) | 512.0 (161.0 to NA) | 197.0 (103.0 to NA) |
Overall survival was defined as the interval between the date of randomization and the date of death due to any cause.
| days | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy |
|---|---|---|---|---|
| Part 1 Expansion: Overall Survival | NA (694.0 to NA) | NA (NA to NA) | NA (NA to NA) | NA (NA to NA) |
NRM was defined as the percentage of participants who died due to causes other than a relapse of their primary hematologic disease.
| percentage of participants | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy |
|---|---|---|---|---|
| Part 1 Expansion: Nonrelapse Mortality (NRM) Rate | 25.7 (12.5 to 43.3) | 15.4 (6.2 to 32.0) | 10.3 (2.3 to 28.2) | 11.1 (3.2 to 26.7) |
The corticosteroid dose at Day 180 was compared to the corticosteroid dose on Day 1 to assess reduction.
| percentage of participants | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy |
|---|---|---|---|---|
| Part 1 Expansion: Percentage of Participants With a ≥50% Reduction in Daily Corticosteroid Dose at Day 180 From the Corticosteroid Dose on Day 1 | 90.5 | 100.0 | 100.0 | 100.0 |
The percentage of participants who were not taking any corticosteroids at Day 180 was assessed.
| percentage of participants | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy |
|---|---|---|---|---|
| Part 1 Expansion: Percentage of Participants Successfully Tapered Off All Corticosteroids at Day 180 | 52.4 | 66.7 | 47.1 | 44.4 |
The relapse rate was defined as the percentage of participants whose underlying disease relapsed at any time during the course of the study.
| percentage of participants | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy |
|---|---|---|---|---|
| Part 1 Expansion: Relapse Rate of Malignant and Nonmalignant Hematologic Diseases | 5.7 (0.7 to 19.2) | 7.7 (1.6 to 20.9) | 13.8 (3.9 to 31.7) | 2.8 (0.1 to 14.5) |
Time to primary hematologic disease relapse was defined as the interval between the date of randomization and the date of relapse.
| days | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy |
|---|---|---|---|---|
| Part 1 Expansion: Time to Primary Hematologic Disease Relapse | NA (NA to NA) | NA (NA to NA) | NA (NA to NA) | NA (NA to NA) |
The LSS consists of 30 items in 7 subscales (skin, eye, mouth, lung, nutrition, energy, and psychological). It was to be used to assess patient-reported changes in health status, symptoms, and well being.
No measurements were reported for this outcome.
The QOL-SF-36 v2 is 36-item scale that captures changes in health status during the course of treatment. The SF-36 assesses 8 health concepts related to limitations in physical activities, social activities, bodily pain, general mental and physical health, and vitality. It was to be used to assess patient-reported changes in health status, symptoms, and well being.
No measurements were reported for this outcome.
The EQ-5D-3L is a descriptive classification consisting of 5 dimensions of health: mobility, self-care, usual activities, anxiety/depression, and pain/discomfort. It was to be used to assess patient-reported changes in health status, symptoms, and well being.
No measurements were reported for this outcome.
The PGIC is 1 question that captures the overall change in symptoms over the course of treatment. It was to be used to assess patient-reported changes in health status, symptoms, and well being.
No measurements were reported for this outcome.
The PGIS is 1 question that captures the overall change in the severity of symptoms over the previous week. It was to be used to assess patient-reported changes in health status, symptoms, and well being.
No measurements were reported for this outcome.
Response rate was defined as the percentage of participants that had CR or PR, per NIH Consensus Criteria, as determined by the investigator, within 14 days of the post-Baseline visit date until new anti-GVHD therapy or overall response-progression or relapse/progression of underlying disease. CR was defined as the complete resolution of all signs and symptoms of cGvHD in all evaluable organs. PR was defined as an improvement in at least one organ without progression in other organs.
No measurements were reported for this outcome.
Duration to response was defined as the interval between the first response and cGVHD progression, death, or the initiation of a new systemic cGVHD therapy.
No measurements were reported for this outcome.
Overall survival was defined as the interval between the date of randomization and the date of death due to any cause.
No measurements were reported for this outcome.
NRM was defined as the percentage of participants who died due to causes other than a relapse of their primary hematologic disease.
No measurements were reported for this outcome.
The corticosteroid dose at Day 180 was compared to the corticosteroid dose on Day 1 to assess reduction.
No measurements were reported for this outcome.
The percentage of participants who were not taking any corticosteroids at Day 180 was assessed.
No measurements were reported for this outcome.
The relapse rate was defined as the defined as the percentage of participants whose underlying disease relapsed at any time during the course of the study.
No measurements were reported for this outcome.
Time to primary hematologic disease relapse was defined as the interval between the date of randomization and the date of relapse.
No measurements were reported for this outcome.
An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE could therefore have been any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE was defined as any AE either reported for the first time or the worsening of a pre-existing event after the first dose of study drug.
No measurements were reported for this outcome.
Collected over until at least 30 days after the last dose of study treatment (up to 1103 days). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Part 1: Itacitinib 200 mg QD + CS | 2/11 (18.2%) | 6/11 (54.5%) | 11/11 (100%) |
| Part 1: Itacitinib 300 mg QD + CS | 3/10 (30%) | 5/10 (50%) | 10/10 (100%) |
| Part 1 Expansion: Itacitinib 300 mg QD + CS | 9/35 (25.7%) | 18/35 (51.4%) | 33/35 (94.3%) |
| Part 1 Expansion: Itacitinib 400 mg QD + CS | 8/39 (20.5%) | 22/39 (56.4%) | 37/39 (94.9%) |
| Part 1 Expansion: Itacitinib 300 mg BID + CS | 4/29 (13.8%) | 12/29 (41.4%) | 27/29 (93.1%) |
| Part 1 Expansion: Total | 21/103 (20.4%) | 52/103 (50.5%) | 97/103 (94.2%) |
| Part 1 Expansion: CS Monotherapy | 5/36 (13.9%) | 9/36 (25%) | 31/36 (86.1%) |
| Event | Part 1: Itacitinib 200 mg QD + CS | Part 1: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: Total | Part 1 Expansion: CS Monotherapy |
|---|---|---|---|---|---|---|---|
| PneumoniaInfections and infestations | 2/11 | 1/10 | 2/35 | 5/39 | 2/29 | 9/103 | 0/36 |
| PyrexiaGeneral disorders | 2/11 | 0/10 | 0/35 | 4/39 | 0/29 | 4/103 | 1/36 |
| Anal haemorrhageGastrointestinal disorders | 0/11 | 1/10 | 0/35 | 0/39 | 0/29 | 0/103 | 0/36 |
| AspirationRespiratory, thoracic and mediastinal disorders | 0/11 | 1/10 | 0/35 | 0/39 | 0/29 | 0/103 | 0/36 |
| Bronchopulmonary aspergillosisInfections and infestations | 0/11 | 1/10 | 2/35 | 0/39 | 0/29 | 2/103 | 0/36 |
| Cardiac failureCardiac disorders | 0/11 | 1/10 | 1/35 | 0/39 | 0/29 | 1/103 | 0/36 |
| Hepatic fibrosisHepatobiliary disorders | 0/11 | 1/10 | 1/35 | 0/39 | 0/29 | 1/103 | 0/36 |
| Liver injuryHepatobiliary disorders | 0/11 | 1/10 | 1/35 | 0/39 | 0/29 | 1/103 | 0/36 |
| Myocardial infarctionCardiac disorders | 0/11 | 1/10 | 0/35 | 0/39 | 0/29 | 0/103 | 0/36 |
| OedemaGeneral disorders | 0/11 | 1/10 | 1/35 | 0/39 | 0/29 | 1/103 | 0/36 |
| Event | Part 1: Itacitinib 200 mg QD + CS | Part 1: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: Total | Part 1 Expansion: CS Monotherapy |
|---|---|---|---|---|---|---|---|
| Platelet count decreasedInvestigations | 5/11 | 3/10 | 6/35 | 7/39 | 9/29 | 22/103 | 2/36 |
| AnaemiaBlood and lymphatic system disorders | 3/11 | 2/10 | 14/35 | 9/39 | 10/29 | 33/103 | 1/36 |
| DiarrhoeaGastrointestinal disorders | 1/11 | 4/10 | 6/35 | 4/39 | 6/29 | 16/103 | 5/36 |
| Muscular weaknessMusculoskeletal and connective tissue disorders | 3/11 | 4/10 | 5/35 | 4/39 | 0/29 | 9/103 | 1/36 |
| FatigueGeneral disorders | 4/11 | 2/10 | 6/35 | 7/39 | 4/29 | 17/103 | 5/36 |
| HypertensionVascular disorders | 2/11 | 2/10 | 6/35 | 7/39 | 9/29 | 22/103 | 7/36 |
| Dry skinSkin and subcutaneous tissue disorders | 0/11 | 3/10 | 3/35 | 2/39 | 0/29 | 5/103 | 0/36 |
| Blood cholesterol increasedInvestigations | 3/11 | 2/10 | 3/35 | 4/39 | 4/29 | 11/103 | 1/36 |
| Blood creatinine increasedInvestigations | 3/11 | 2/10 | 3/35 | 4/39 | 3/29 | 10/103 | 1/36 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 3/11 | 1/10 | 5/35 | 2/39 | 1/29 | 8/103 | 5/36 |
| Age, Continuous(years) | Part 1: Itacitinib 200 mg QD + CS | Part 1: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy | Total |
|---|---|---|---|---|---|---|---|
| Mean | 57.2 ± 7.07 | 58.4 ± 15.08 | 53.6 ± 14.01 | 52.6 ± 13.79 | 52.6 ± 12.86 | 55.7 ± 13.32 | 54.2 ± 13.23 |
| Sex: Female, Male(Participants) | Part 1: Itacitinib 200 mg QD + CS | Part 1: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy | Total |
|---|---|---|---|---|---|---|---|
| Female | 3 | 3 | 17 | 16 | 14 | 19 | 72 |
| Male | 8 | 7 | 18 | 23 | 15 | 17 | 88 |
| Race/Ethnicity, Customized(Participants) | Part 1: Itacitinib 200 mg QD + CS | Part 1: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy | Total |
|---|---|---|---|---|---|---|---|
| White/Caucasian | 11 | 8 | 31 | 36 | 27 | 34 | 147 |
| Black/African-American | 0 | 1 | 2 | 0 | 0 | 0 | 3 |
| Asian | 0 | 1 | 1 | 3 | 2 | 1 | 8 |
| Captured as "Other" in Database | 0 | 0 | 1 | 0 | 0 | 1 | 2 |
| Race/Ethnicity, Customized(Participants) | Part 1: Itacitinib 200 mg QD + CS | Part 1: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg QD + CS | Part 1 Expansion: Itacitinib 400 mg QD + CS | Part 1 Expansion: Itacitinib 300 mg BID + CS | Part 1 Expansion: CS Monotherapy | Total |
|---|---|---|---|---|---|---|---|
| Hispanic or Latino | 4 | 2 | 6 | 8 | 1 | 5 | 26 |
| Not Hispanic or Latino | 6 | 8 | 25 | 26 | 19 | 29 | 113 |
| Not Reported | 1 | 0 | 2 | 2 | 4 | 2 | 11 |
| Unknown | 0 | 0 | 2 | 1 | 1 | 0 | 4 |
| Captured as "Other" in Database | 0 | 0 | 0 | 2 | 4 | 0 | 6 |
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Plan to share: Yes — Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency
Supporting information: Study protocol, Sap
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