Upfront Ruxolitinib for Chronic Graft-vs-host Disease

Upfront Ruxolitinib Treatment for Chronic Graft-vs-host Disease in Children and Young Adults: A Corticosteroid-sparing Pilot Study

While hematopoietic stem cell transplant (HSCT) is an effective therapy, graft versus host disease (GVHD) is the most significant complication after HSCT. Corticosteroids (or steroids) have been the mainstay of treatment for chronic GVHD for many decades now. Increasingly, newer immunosuppressive and immunomodulating agents are being studied in adults and children affected by cGVHD. Ruxolitinib is one of these promising newer agents, which has been shown to be effective in the treatment of cGVHD in both children and adults. Currently, ruxolitinib is generally added to a patient's treatment regimen after (or with) a course of high dose steroids.

The purpose of this study is to examine the effectiveness of upfront single agent ruxolitinib for cGVHD.

Study Overview

• Status: Terminated
• Conditions: Chronic Graft Versus Host Disease
• Intervention / Treatment: Drug: Ruxolitinib

• Study Type: Interventional
• Enrollment (Actual): 1
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Newly diagnosed moderate to severe chronic graft versus host disease (as defined by NIH cGVHD consensus criteria), requiring systemic treatment
• Patient aged ≥12 year-old and ≤30 year-old
• Patient able to take oral or enteral medication
• No active, clinically significant uncontrolled infections
• ALT ≤ 5x upper limit of normal (ULN) and total bilirubin ≤ 5xULN (unless presumed liver GVHD)
• Platelet ≥ 20k and ANC ≥ 500. The use of transfusions or growth factors is permitted to maintain counts at these thresholds
• No prior systemic treatment for chronic GVHD. Patients previously treated for acute GVHD are eligible, including those who received ruxolitinib for treatment of their aGVHD
• Patients must be on ≤ physiologic dosing (i.e. hydrocortisone 8-12mg/m2/day) at enrollment
• Patients with prior acute GVHD on < 1 mg/kg steroids with new onset moderate-severe chronic GVHD may be considered for enrollment if they can taper steroids to reach physiological hydrocortisone in 1 month. If unable to do so these patients will come off study and be replaced

Exclusion Criteria:

• Mild cGVHD (as defined by NIH cGVHD consensus criteria11), that does not require systemic therapy
• Acute or late acute GVHD without any evidence of chronic GVHD features
• Patients who have received corticosteroids for ≥ 24 hours at 1 mg/kg/day of methylprednisolone or prednisone with the intent to treat cGVHD at time of enrollment
• Corticosteroid dosing above physiologic dose hydrocortisone (i.e. > 8-12mg/m2/day) at time of enrollment
• Clinical evidence suggesting active malignancy (including PTLD and primary/secondary malignancy)
• Clinical evidence of clinically significant active infection
• Ongoing cytopenias which cannot be supported with routine supportive care (keeping hemoglobin over 7 g/dL and platelets >20,000 and absolute neutrophil count over 500/uL)
• Active lower gastrointestinal bleeding
• Thrombosis within 6 months (including myocardial infarction, stroke, deep venous thrombosis, pulmonary embolism). IV infiltration will not be included as an example of thrombosis
• Pregnant or lactating females; patients of childbearing age who are not able to comply with contraceptive recommendations
• Other condition that PI feels would preclude the patient from complying with study activities
• ESRD [CLcr < 15 mL/min] not on dialysis
• Overlap syndrome

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ruxolitinib Treatment	Drug: Ruxolitinib; Participants will receive ruxolitinib for 6 months.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Overall Response Rate	6 months

Collaborators and Investigators

• Sponsor: Children's Hospital Medical Center, Cincinnati
• Investigators: Principal Investigator: Pooja Khandelwal, MD, Children's Hospital Medical Center, Cincinnati

Study record dates

Study Major Dates

• Study Start (Actual): February 19, 2025
• Primary Completion (Actual): August 20, 2025
• Study Completion (Actual): September 18, 2025

Study Registration Dates

• First Submitted: November 16, 2024
• First Submitted That Met QC Criteria: November 16, 2024
• First Posted (Actual): November 19, 2024

Study Record Updates

• Last Update Posted (Actual): February 25, 2026
• Last Update Submitted That Met QC Criteria: February 23, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Organizing Pneumonia; Immune System Diseases; Respiratory Tract Diseases; Lung Diseases; Bronchial Diseases; Lung Diseases, Obstructive; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Graft vs Host Disease; Bronchiolitis Obliterans Syndrome; ruxolitinib
• Other Study ID Numbers: 2023-0529

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No