Clinical trial NCT03147742: An Expanded Access Program of Ruxolitinib for the Treatment of Graft-Versus-Host Disease Following Allogeneic Hematopoietic Stem Cell Transplant
Official title: An Open-Label, Expanded Access Program of Ruxolitinib for the Treatment of Graft-Versus-Host Disease Following Allogeneic Hematopoietic Stem Cell Transplant
Study type: EXPANDED_ACCESS
Overall status: APPROVED_FOR_MARKETING
Conditions: Graft-versus-host Disease (GVHD)
Keywords: ruxolitinib
acute or chronic steroid-refractory GVHD
hematopoietic stem cell transplantation (allo-HSCT)
Lead sponsor: Incyte Corporation
Sponsor class: INDUSTRY
First posted: 2017-05-10
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Mayo Clinic Arizona
state: Arizona
city: Scottsdale
country: United States
facility: Cancer Transplant Institute at HonorHealth
state: Arizona
city: Duarte
country: United States
facility: City of Hope Medical Center
state: California
city: La Jolla
country: United States
facility: Scripps Clinic
state: California
city: Denver
country: United States
facility: Colorado Blood Cancer Institute
state: Colorado
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
city: Orlando
country: United States
facility: Blood & Marrow Transplant Center
state: Florida
city: Chicago
country: United States
facility: Northwestern Memorial Hospital
state: Illinois
city: Chicago
country: United States
facility: University of Illinois Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Indiana Blood and Marrow Transplantation
state: Indiana
city: Wichita
country: United States
facility: Cancer Center of Kansas
state: Kansas
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Comprehensive Cancer Center
state: Michigan
city: Grand Rapids
country: United States
facility: Spectrum Health
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic Minnesota
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: New Brunswick
country: United States
facility: Rutgers Cancer Institute
state: New Jersey
city: Albuquerque
country: United States
facility: New Mexico Cancer Center
state: New Mexico
city: New York
country: United States
facility: Weill Cornell Medical College
state: New York
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Columbus
country: United States
facility: Ohio State University
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Gettysburg
country: United States
facility: Gettysburg Cancer Center
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Allegheny Health Network Cancer Institute
state: Pennsylvania
city: Greenville
country: United States
facility: Greenville Health System Cancer Institute
state: South Carolina
city: Sioux Falls
country: United States
facility: Avera Research Institute
state: South Dakota
city: Dallas
country: United States
facility: Texas Oncology
state: Texas
city: Houston
country: United States
facility: Houston Methodist
state: Texas
city: San Antonio
country: United States
facility: Methodist Hospital
state: Texas
city: Seattle
country: United States
facility: Fred Hutchinson Cancer Research Center
state: Washington
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
Study officials:
affiliation: Incyte Corporation
name: John Galvin, MD
role: STUDY_DIRECTOR
References: