NCT03616184

Study of Ruxolitinib in Sclerotic Chronic Graft-Versus-Host Disease After Failure of Systemic Glucocorticoids

Completed Phase 2 Results posted Last updated 5 October 2023

What this trial tests

Phase 2 trial testing Ruxolitinib in Graft-versus-host-disease (GVHD) in 49 participants. Completed in 12 June 2023.

Timeline

5 September 2018

Primary endpoint
22 January 2022

12 June 2023

Quick facts

Lead sponsor	University of Nebraska
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	49
Start date	5 September 2018
Primary completion	22 January 2022
Estimated completion	12 June 2023
Sites	5 locations across United States

Drugs / interventions tested

• Ruxolitinib (RUXOLITINIB) — full drug profile →

Conditions studied

• Graft-versus-host-disease (GVHD) — all drugs for Graft-versus-host-disease (GVHD) →

Sponsor

University of Nebraska

Who can join

18 and older, any sex, with Graft-versus-host-disease (GVHD). 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.

Percentage of Participants With Complete and Partial Responses in Skin and/or Joint Primary · 6 months

The primary endpoint of the study was complete or partial response in skin and/or joint defined according to the 2014 NIH cGVHD Consensus Criteria. Partial response for skin would be a decrease in NIH Skin Score by 1 or more points. Partial response in joint would be a decrease in NIH Joint and Fascia Score by 1 or more points or increase in P-ROM score by 1 point for any site.

Group	Value	95% CI
Ruxolitinib	49

Percentage of Participants With Complete or Partial Responses Overall Secondary · 6 months

As determined by 2014 NIH Criteria which is a clinician overall severity score of 0 for complete response and clinician overall severity score decreased by 2 or more points on a 0-10 scale for partial response.

Group	Value	95% CI
Ruxolitinib	47

Adverse events — posted to ClinicalTrials.gov

Time frame: 2 years. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Ruxolitinib

Serious: 13/47 (28%)

Deaths: 4/47

Serious adverse events (21 terms)

Reaction	System	Ruxolitinib
Investigations	Investigations	—
Lung Infection	Infections and infestations	—
Abdominal Pain	Gastrointestinal disorders	—
Adult respiratory distress syndrome	Respiratory, thoracic and mediastinal disorders	—
Blood and lymphatic system disorders	Blood and lymphatic system disorders	—
Catheter related infection	Infections and infestations	—
Chest pain-cardiac	Cardiac disorders	—
Cholecystitis	Hepatobiliary disorders	—
Dyspnea	Respiratory, thoracic and mediastinal disorders	—
Enterocolitis	Gastrointestinal disorders	—
Fever	General disorders	—
Fracture	Injury, poisoning and procedural complications	—
Hallucinations	Psychiatric disorders	—
Hypoxia	Respiratory, thoracic and mediastinal disorders	—
Infections and infestations	Infections and infestations	—
Nausea	Gastrointestinal disorders	—
Respiratory Failure	Respiratory, thoracic and mediastinal disorders	—
Sepsis	Infections and infestations	—
Sudden death NOS	General disorders	—
Syncope	Nervous system disorders	—
Thromboembolic event	Vascular disorders	—

Other adverse events (13 terms — click to expand)

Reaction	System	Ruxolitinib
Nausea	Gastrointestinal disorders	—
Upper respiratory infection	Infections and infestations	—
Fatigue	General disorders	—
Vomiting	Gastrointestinal disorders	—
Diarrhea	Gastrointestinal disorders	—
weight gain	Investigations	—
Hypertension	Vascular disorders	—
Anemia	Blood and lymphatic system disorders	—
Aspartate aminotransferase increased	Investigations	—
Dizziness	Nervous system disorders	—
Dry Eye	Eye disorders	—
Insomnia	Psychiatric disorders	—
Skin and subcutaneous tissue disorders - Other, specify	Skin and subcutaneous tissue disorders	—

Most-reported serious reactions: Investigations, Lung Infection, Abdominal Pain, Adult respiratory distress syndrome, Blood and lymphatic system disorders, Catheter related infection, Chest pain-cardiac, Cholecystitis.

Data from ClinicalTrials.gov NCT03616184 adverse events section.

Sponsor's own description

The primary objective of the study is to examine the efficacy of ruxolitinib in patients with sclerotic chronic graft-versus-host disease (GVHD).

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. Emerging Topical and Systemic JAK Inhibitors in Dermatology.
   Solimani F, Meier K, Ghoreschi K. · · 2019 · cited 199× · PMID 31849996 · DOI 10.3389/fimmu.2019.02847
2. JAK-STAT signaling in human disease: From genetic syndromes to clinical inhibition.
   Luo Y, Alexander M, Gadina M, O'Shea JJ, et al · · 2021 · cited 92× · PMID 34625141 · DOI 10.1016/j.jaci.2021.08.004
3. New Approaches for the Treatment of Chronic Graft-Versus-Host Disease: Current Status and Future Directions.
   Saidu NEB, Bonini C, Dickinson A, Grce M, et al · · 2020 · cited 64× · PMID 33162993 · DOI 10.3389/fimmu.2020.578314
4. SYK inhibitor entospletinib prevents ocular and skin GVHD in mice.
   Poe JC, Jia W, Di Paolo JA, Reyes NJ, et al · · 2018 · cited 40× · PMID 30282825 · DOI 10.1172/jci.insight.122430
5. A Multicenter Phase II Trial of Ruxolitinib for Treatment of Corticosteroid Refractory Sclerotic Chronic Graft-Versus-Host Disease.
   Bhatt VR, Shostrom VK, Choe HK, Hamilton BK, et al · · 2024 · cited 12× · PMID 39151112 · DOI 10.1200/jco.24.00205
6. Chronic Graft-versus-host Disease: Immune Insights, Therapeutic Advances, and Parallels for Solid Organ Transplantation.
   Boiko JR, Hill GR. · · 2025 · cited 4× · PMID 39682018 · DOI 10.1097/tp.0000000000005298
7. Current therapeutics for sclerotic and fibrotic manifestations of chronic graft-versus-host disease.
   Choe H, DeFilipp Z. · · 2026 · PMID 41238758 · DOI 10.1038/s41409-025-02743-7