An Expanded Access Program of Ruxolitinib for the Treatment of Graft-Versus-Host Disease Following Allogeneic Hematopoietic Stem Cell Transplant

An Open-Label, Expanded Access Program of Ruxolitinib for the Treatment of Graft-Versus-Host Disease Following Allogeneic Hematopoietic Stem Cell Transplant

To provide ruxolitinib through an expanded access program for the treatment of graft-versus-host disease (GVHD) in United States to patients who are ineligible or unable to participate in any actively enrolling Incyte-sponsored clinical studies for ruxolitinib in the treatment of GVHD.

Study Overview

• Status: Approved for marketing
• Conditions: Graft-versus-host Disease (GVHD)
• Intervention / Treatment: Drug: Ruxolitinib

• Study Type: Expanded Access
• Expanded Access Type: Intermediate-size Population

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85054
      • Mayo Clinic Arizona
    • Scottsdale, Arizona, United States, 85258
      • Cancer Transplant Institute at HonorHealth
  • California
    • Duarte, California, United States, 91010
      • City of Hope Medical Center
    • La Jolla, California, United States, 92037
      • Scripps Clinic
  • Colorado
    • Denver, Colorado, United States, 80218
      • Colorado Blood Cancer Institute
  • Florida
    • Miami, Florida, United States, 33155
      • Nicklaus Children's Hospital
    • Orlando, Florida, United States, 32804
      • Blood & Marrow Transplant Center
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern Memorial Hospital
    • Chicago, Illinois, United States, 60612
      • University of Illinois Chicago
  • Indiana
    • Indianapolis, Indiana, United States, 46237
      • Indiana Blood and Marrow Transplantation
  • Kansas
    • Wichita, Kansas, United States, 67214
      • Cancer Center of Kansas
  • Massachusetts
    • Boston, Massachusetts, United States, 02214
      • Massachusetts General Hospital
  • Michigan
    • Ann Arbor, Michigan, United States, 48109-5271
      • University of Michigan Comprehensive Cancer Center
    • Grand Rapids, Michigan, United States, 49503
      • Spectrum Health
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic Minnesota
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Washington University School of Medicine
  • New Jersey
    • Hackensack, New Jersey, United States, 07601
      • Hackensack University Medical Center
    • New Brunswick, New Jersey, United States, 08903
      • Rutgers Cancer Institute
  • New Mexico
    • Albuquerque, New Mexico, United States, 87109
      • New Mexico Cancer Center
  • New York
    • New York, New York, United States, 10065
      • Memorial Sloan Kettering Cancer Center
    • New York, New York, United States, 10021
      • Weill Cornell Medical College
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic
    • Columbus, Ohio, United States, 43210
      • Ohio State University
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health & Science University
  • Pennsylvania
    • Gettysburg, Pennsylvania, United States, 17325
      • Gettysburg Cancer Center
    • Pittsburgh, Pennsylvania, United States, 15224
      • Allegheny Health Network Cancer Institute
  • South Carolina
    • Greenville, South Carolina, United States, 29615
      • Greenville Health System Cancer Institute
  • South Dakota
    • Sioux Falls, South Dakota, United States, 57105
      • Avera Research Institute
  • Texas
    • Dallas, Texas, United States, 75246
      • Texas Oncology
    • Houston, Texas, United States, 77030
      • Houston Methodist
    • San Antonio, Texas, United States, 78229
      • Methodist Hospital
  • Washington
    • Seattle, Washington, United States, 98109-1024
      • Fred Hutchinson Cancer Research Center
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Medical College of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: N/A

Description

Key Inclusion Criteria:

• Male or female, 12 years of age or older.
• Have undergone an allo-HSCT from any donor source using bone marrow, peripheral blood stem cells, or cord blood for hematologic malignancies. Recipients of nonmyeloablative and myeloablative conditioning regimens are eligible.
• Clinically suspected all grades chronic GVHD according to NIH Consensus Criteria that is refractory or intolerant to corticosteroids, occurring after allo-HSCT with any conditioning regimen and any anti-GVHD prophylactic regimen. Clinical suspicion of steroid-refractory chronic GVHD by the treating physician is also sufficient.
• Evidence of myeloid engraftment (eg, absolute neutrophil count ≥ 1.0 × 10^9/L for 3 consecutive days if ablative therapy was previously used). Use of growth factor supplementation is allowed.
• Evidence of platelet engraftment (ie, platelets ≥ 20 × 10^9/L).
• Eastern Cooperative Oncology Group (ECOG) performance status 0 to 3.

• Be willing to avoid pregnancy or fathering children based on 1 of the following criteria:

  • Women of non-childbearing potential (ie, surgically sterile with a hysterectomy and/or bilateral oophorectomy OR ≥ 12 months of amenorrhea).
  • Woman of childbearing potential who has a negative serum pregnancy test at screening and who agrees to take appropriate precautions to avoid pregnancy (with at least 99% certainty) from screening through safety follow-up. Permitted methods that are at least 99% effective in preventing pregnancy should be communicated to the patient and their understanding confirmed.
  • Man who agrees to take appropriate precautions to avoid fathering children (with at least 99% certainty) from screening through safety follow-up. Permitted methods that are at least 99% effective in preventing pregnancy should be communicated to the patient and their understanding confirmed.
• Able to provide written informed consent and/or assent from the patient, parent, or guardian.

Key Exclusion Criteria:

• Eligible for an existing and actively enrolling Incyte sponsored clinical trial for ruxolitinib for the treatment of GVHD.
• Clinically suspected all grades of acute GVHD as per Minnesota-Center for International Blood and Marrow Transplant Research (MN-CIBMTR) criteria or clinical suspicion of acute GVHD by the treating physician.
• Patients or legal guardians unable to review and sign informed consent form.
• Females who are pregnant or breastfeeding, and males and females who cannot comply with requirements to avoid fathering a child or becoming pregnant.
• Patients with inadequate liver function (alanine aminotransferase above 4 × upper limit of normal (ULN) or direct bilirubin 4 × ULN and the laboratory abnormalities are considered to be due to underlying liver dysfunction) unless attributed to GVHD.
• Patients with end stage renal function (creatinine clearance (CrCl) < 15 mL/min or glomerular filtration rate < 15 mL/min), regardless of whether hemodialysis is required.
• Any underlying or current medical or psychiatric condition that, in the opinion of the treating physician, would place the patient at an unacceptable risk if he or she were to participate in the program.
• Previous allergic reactions to Janus kinase (JAK) inhibitors or excipients.
• Patients who are currently taking any anticancer therapy (eg, chemotherapy, radiation therapy, surgery, immunotherapy, biologic therapy, hormonal therapy, or tumor embolization).
• Concomitant use of any JAK inhibitor.
• Initiating therapy with any investigational medication.
• Presence of an active uncontrolled infection. An active uncontrolled infection is defined as hemodynamic instability attributable to sepsis or new symptoms, worsening physical signs, or radiographic findings attributable to infection. Persisting fever without signs or symptoms will not be interpreted as an active uncontrolled infection.
• Known HIV infection.
• Active hepatitis B virus (HBV) or hepatitis C virus infection that requires treatment or at risk for HBV reactivation. At risk for HBV reactivation is defined as hepatitis B surface antigen positive or anti-hepatitis B core antibody positive. Previous test results obtained as part of standard of care before allo-HSCT that confirm a patient is immune and not at risk for reactivation (ie, hepatitis B surface antigen negative, surface antibody positive) may be used for purposes of eligibility.

Study Plan

How is the study designed?

Collaborators and Investigators

• Sponsor: Incyte Corporation
• Investigators: Study Director: John Galvin, MD, Incyte Corporation

Study record dates

Study Registration Dates

• First Submitted: May 8, 2017
• First Submitted That Met QC Criteria: May 8, 2017
• First Posted (Actual): May 10, 2017

Study Record Updates

• Last Update Posted (Actual): June 7, 2022
• Last Update Submitted That Met QC Criteria: June 6, 2022
• Last Verified: June 1, 2022

More Information

Terms related to this study

• Keywords: ruxolitinib; acute or chronic steroid-refractory GVHD; hematopoietic stem cell transplantation (allo-HSCT)
• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease
• Other Study ID Numbers: INCB 18424-MA-GD-301