18 and older, any sex, with Chronic Graft-versus-host-disease. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
cGVHD Overall Response Rate (ORR) at 6 Months on TreatmentPrimary· Response assessed Day 1 of every cycle up until the end of treatment, up to 24 months. This outcome measure is the ORR for all patients once they have been on treatment for 6 months.
Defined as the rate of participants with a cGVHD response of complete response (CR) or partial response (PR) after 6 months of treatment with Itacitinib as defined by 2014 National Institutes of Health Consensus Development Project on Clinical Trials in cGVHD. CR is defined as resolution of all manifestations of cGVHD in each organ or site. PR is defined as the improvement in at least one organ or site without progression in any other organ or site.
Group
Value
95% CI
Itacitinib
93
Number of Participants That Can Withdraw or Decrease SteroidsSecondary· From first dose of itacitinib until end of study treatment, up to 24 months
Ability to withdraw or decrease steroids to ˂0.5 mg/kg of methylprednisolone or equivalent
Group
Value
95% CI
Itacitinib
9
Overall SurvivalSecondary· Every 3 months for 1 year after last dose of study treatment, up to 29 months.
Overall Survival (OS) is defined as the time from the first day of study drug administration (Day 1) to death on study. Patients who are alive will be censored at the date of last known date alive.
Group
Value
95% CI
Itacitinib
NA
15.5 – NA
Number of Participants With Treatment-emergent Adverse Events as a Measure of Safety and Tolerability of ItacitinibSecondary· Safety will be assessed from the time that informed consent is signed until 30 days after the last dose of study treatment, up to 25 months.
Adverse events will be graded according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE Version 5.0)
Group
Value
95% CI
Itacitinib
15
Number of Participants With Improvement in Quality of Life Per the Lee Symptom ScaleSecondary· Quality of life will be assessed prior during screening and end of treatment, up to 24 months of treatment. Participants showing improvement in LSS score from Screening to End of Treatment are shown here.
Changes in symptom burden will be measured using the Lee Symptom Scale. Subscale scores and the summary score range from 0 to 100, with a higher score indicating worse symptoms.
Group
Value
95% CI
Itacitinib
11
Number of Participants With Recurrence or Progression of cGVHDSecondary· cGVHD status will be assessed at cycle 1 day 1, day 1 of every cycle and at the end of treatment, up to 24 months
Participants will be assessed for severity of cGVHD using the Clinician-Reported Global cGVHD Activity Assessment Form and Patient-reported cGVHD Activity Assessment Form. Progression in at least one organ or site without a response in any other organ or site per NIH 2014 Consensus Development Project on Clinical Trials in cGVHD.
Group
Value
95% CI
Itacitinib
5
Relapse Rate of Underlying MalignancySecondary· Relapse of underlying malignancy will be assessed on Day 1 of each cycle, at end of treatment and survival follow-up. Follow-up visits every 3 months for 1 year after last dose of study treatment, up to 29 months
Participants will be closely monitored for any evidence of underlying disease relapse or recurrence. Formal re-staging will be done at physician discretion.
Group
Value
95% CI
Itacitinib
2
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse events were assessed from the time that informed consent is signed until 30 days after the last dose of study treatment, up to 25 months. All-Cause Mortality was assessed up to 29 months..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Itacitinib
Serious: 6/15 (40%)
Deaths: 2/15
Serious adverse events (13 terms)
Reaction
System
Itacitinib
Brain contusion
Injury, poisoning and procedural complications
—
Cellulitis
Infections and infestations
—
Device related infection
Infections and infestations
—
Embolism
Vascular disorders
—
Eye contusion
Injury, poisoning and procedural complications
—
Pneumatosis
General disorders
—
Pneumonia
Infections and infestations
—
Pneumoperitoneum
Gastrointestinal disorders
—
Pulmonary embolism
Respiratory, thoracic and mediastinal disorders
—
Respiratory tract infection bacterial
Infections and infestations
—
Small intestinal obstruction
Gastrointestinal disorders
—
Subarachnoid haemorrhage
Nervous system disorders
—
Subdural haematoma
Injury, poisoning and procedural complications
—
Other adverse events (139 terms — click to expand)
This study is being done in patients who have been receiving corticosteroids or other immunosuppressive therapies for the treatment of cGVHD for at least 6 months. The purpose of this study is to find out if itacitinib in combination with corticosteroids or other immunosuppressive therapies is safe and effective in people with cGVHD.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT05660421 — Itacitinib for the Treatment Steroid Refractory Immune Related Adverse Events Arising From Immune Checkpoint Inhibitors
· Phase 2
· withdrawn
NCT05364762 — Adding Itacitinib to Cyclophosphamide and Tacrolimus for the Prevention of Graft Versus Host Disease in Patients Undergo
· Phase 2
· active not recruiting
NCT05063110 — Treatment of Non Severe Hemophagocytosis Lymphohistiocytosis With ITACITINIB
· Phase 2
· completed
NCT04859946 — Itacitinib for the Prevention of Graft Versus Host Disease
· Phase 2
· active not recruiting
NCT04239989 — Itacitinib for the Treatment of Bronchiolitis Obliterans Syndrome After Donor Hematopoietic Cell Transplant
· Phase 1
· terminated
Other recruiting trials for Chronic Graft-versus-host-disease
Currently open trials in the same condition.
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· recruiting
NCT06843408 — A Ph1b/2 Study of Axatilimab Monotherapy in Chinese Participants With Recurrent or Refractory cGVHD
· Phase 1, PHASE2
· recruiting
NCT06821542 — A Study to Evaluate Axatilimab Versus Best Available Therapy in Participants With Chronic Graft Versus Host Disease Afte
· Phase 3
· active not recruiting
NCT06585774 — A Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease
· Phase 3
· recruiting
NCT06388564 — A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Dia
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by SCRI Development Innovations, LLC
Last refreshed: 6 November 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04200365.