A Study of Ruxolitinib in Patients With Graft-Versus Host Disease

Post-marketing Database Study in Patients With Graft-Versus Host Disease for JAKAVI Tablets

This study aims to evaluate the safety and effectiveness of Jakavi® (ruxolitinib) in patients with acute graft-versus host disease (GVHD) and chronic GVHD in clinical practice.

Study Overview

• Status: Not yet recruiting
• Conditions: Graft-Versus-Host Disease

• Study Type: Observational
• Enrollment (Estimated): 395

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: +41613241111; Email: novartis.email@novartis.com
• Study Contact Backup: Name: Novartis Pharmaceuticals

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients treated with ruxolitinib for acute and chronic GVHD after its approval.

Description

Inclusion Criteria:

• Patients with acute and chronic GVHD
• Patients treated with ruxolitinib

Exclusion Criteria:

None

Other protocol-defined inclusion/exclusion criteria may apply.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Ruxolitinib Cohort; Patients with acute and chronic GVHD treated with ruxolitinib.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number and Percentage of Patients With Common Terminology Criteria for Adverse Event (CTCAE) Grade ≥ 3 Infections	Grade ≥ 3 infections according to the CTCAE v5.0.	Up to 1 year
Number and Percentage of Deaths Due to Infection		Up to 1 year
Time to First Event	Time to first event is defined as the time from the start date of treatment with ruxolitinib to the first onset date of infection. Grade ≥ 4 infections according to the CTCAE v5.0.	Up to 1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number and Percentage of CTCAE Grade ≥ 3 Infections	Grade ≥ 3 infections according to the CTCAE v5.0.	Up to 1 year
Odds Ratio of CTCAE Grade ≥ 3 Infections	Odds ratios of infections and their 95% confidence intervals summarized by risk factors for infection.	Up to 1 year
Number and Percentage of Patients With Adverse Events Other Than Infections		Up to 1 year
Number and Percentage of Patients who Died From Reasons Other Than Infection		Up to 1 year
Time to First Onset of Bone-related Events	The time to the first onset is defined as the time from the start date of treatment with ruxolitinib to the first onset date of CTCAE Grade ≥ 4 fracture or bone-related events other than fracture.	Up to 1 year
Overall Response Rate (ORR) Among Patients With Acute GVHD	ORR is defined as the proportion of patients with complete response (CR) or partial response (PR).	Day 28
Number and Percentage of Stages of Disorder for Each Organ Among Patients With Acute GVHD		Baseline, Day 28, up to 1 year
ORR Among Patients With Chronic GVHD	ORR is defined as the proportion of patients with CR or PR.	Month 6
Number and Percentage of Chronic GVHD Patients by Response	Response (CR, PR, mixed response, no change, progression) as entered into the database by the physician in accordance with the Japanese Society for Transplantation and Cellular Therapy guidelines for hematopoietic cell transplantation.	Baseline, Month 6, up to 1 year
Best Overall Response (BOR)	BOR is defined as the proportion of patients with CR or PR at any time point up to Month 12 of treatment.	Baseline, up to 1 year
Overall Survival	Overall survival is defined as the time from the start date of treatment with ruxolitinib to the date of death due to any cause.	Up to 1 year
Duration of Response (DOR) Among Chronic GVHD Patients	DOR is defined for patients who achieved a response (CR or PR) by Month 12 of treatment as the time from the date of first documented response by Month 12 to the date of a DOR event (progression of chronic GVHD, death, or initiation of new systemic therapy for chronic GVHD, whichever occurs first).	Up to 1 year
Time to Response (TTR) Among Chronic GVHD Patients	TTR is defined as the time from the start date of treatment with ruxolitinib to the date of first confirmed response (CR or PR).	Up to 1 year

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Estimated): May 31, 2026
• Primary Completion (Estimated): March 15, 2030
• Study Completion (Estimated): March 15, 2030

Study Registration Dates

• First Submitted: May 11, 2026
• First Submitted That Met QC Criteria: May 11, 2026
• First Posted (Actual): May 19, 2026

Study Record Updates

• Last Update Posted (Actual): May 19, 2026
• Last Update Submitted That Met QC Criteria: May 11, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Ruxolitinib; GVHD
• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease
• Other Study ID Numbers: CINC424C1401

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO