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Itacitinib for Low Risk GVHD

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03846479
Recruitment Status : Recruiting
First Posted : February 19, 2019
Last Update Posted : October 14, 2019
Information provided by (Responsible Party):
John Levine, Icahn School of Medicine at Mount Sinai

Brief Summary:
Graft-versus-host disease (GVHD) is treated with high doses of systemic steroids which can lead to serious complications. A new blood test can identify patients whose GVHD is most likely to respond to well to treatment (low risk GVHD). This study will test whether patients with low risk GVHD can be successfully treated without steroids. Patients who participate with this study will be treated with itacitinib instead of steroids. Itacitinib is an experimental drug with an excellent safety record and appears to have activity as a GVHD treatment.

Condition or disease Intervention/treatment Phase
Low Risk Acute Graft-versus-host Disease Graft-versus-host-disease GVHD Drug: Itacitinib Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Open label, single arm, non-inferiority study
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Itacitinib Monotherapy for Low Risk Graft-vs-Host Disease
Actual Study Start Date : March 25, 2019
Estimated Primary Completion Date : December 31, 2019
Estimated Study Completion Date : December 31, 2020

Arm Intervention/treatment
Experimental: Itacitinib
Itacitinib 200 mg administered orally daily
Drug: Itacitinib
for up to 56 days

Primary Outcome Measures :
  1. Minnesota standard risk clinical criteria [ Time Frame: Day 28 ]
    The Minnesota standard risk clinical criteria is defined as CR= complete remission, PR = partial remission, and TRM - treatment related mortality

  2. Ann Arbor Score 1 (AA1) [ Time Frame: Day 28 ]
    Ann Arbor Score - a scoring system that uses an algorithm of three biomarkers (TNFR1, ST2 and REG3a). A low Ann Arbor Score is Ann Arbor 1 (AA1) =<10%

Secondary Outcome Measures :
  1. Number of adverse events [ Time Frame: Day 28 ]
    Number of adverse events at Day 28 to assess safety

  2. Incidence of serious infectious complications [ Time Frame: Day 90 ]
    The incidence of serious infectious complications in patients with GVHD treated with itacitinib. Serious infectious complications is defined as any viral and bacterial infections requiring treatment and proven fungal infections.

  3. Overall Response Rate [ Time Frame: Day 56 ]
    Number of overall response rate (ORR), defined as complete and partial responses.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   12 Years to 75 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Newly diagnosed GVHD that meets criteria for Minnesota standard risk
  • Ann Arbor 1 GVHD by biomarkers
  • GVHD not previously treated systemically (topical therapies and non-absorbed steroids are allowed)
  • Any donor type, HLA-match, conditioning regimen is acceptable
  • Age 12 - 75 years (children <18 years must also weigh 50 kg or more)
  • Patients must be engrafted post-transplant (ANC >500/μL and platelet count >20,000). Use of growth factor supplementation to maintain neutrophil count is allowed.
  • Direct bilirubin must be <2 mg/dL unless the elevation is known to be due to Gilbert syndrome within 3 days prior to enrollment.
  • ALT/SGPT and AST/SGOT must be <5x the upper limit of the normal range within 3 days prior to enrollment.
  • Signed and dated written informed consent obtained from patient or legal representative.

Exclusion Criteria:

  • Patients currently being treated with any JAK inhibitor including ruxolitinib
  • Relapsed, progressing, or persistent malignancy requiring withdrawal of systemic immune suppression
  • Patients with uncontrolled infection (i.e., progressive symptoms related to infection despite treatment or persistently positive microbiological cultures despite treatment or any other evidence of severe sepsis)
  • Severe organ dysfunction including requirement for dialysis, mechanical ventilation or oxygen supplementation exceeding 40% FiO2 within 7 days of enrollment.
  • Creatinine clearance or estimated glomerular filtration rate <30 ml/min as calculated by institutional practice (e.g., Cockcroft-Gault equation, CKD-EPI equation, etc)
  • A clinical presentation resembling de novo chronic GVHD or overlap syndrome developing before or present at the time of enrollment
  • Patients receiving corticosteroids >10 mg/day prednisone (or other steroid equivalent) for any indication within 7 days before the onset of acute GVHD except for adrenal insufficiency or premedication for transfusions/IV meds
  • Patients who are pregnant
  • Patients receiving investigational agents within 30 days of enrollment. However, the Principal Investigator (PI) may approve prior use of an investigational agent if the agent is not expected to interfere with the safety or the efficacy of itacitinib
  • History of allergic reaction to itacitinib or any JAK inhibitor

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03846479

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Contact: Rachel Young 212-659-5605

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United States, California
City of Hope Comprehensive Cancer Center Not yet recruiting
Duarte, California, United States, 91010
Contact: Ryotara Nakamura, MD    626-256-4673   
Principal Investigator: Ryotara Nakamura, MD         
Children's Hospital of Los Angeles Not yet recruiting
Los Angeles, California, United States, 90027
Contact: Michael Pulsipher, MD    323-361-2546   
Principal Investigator: Michael Pulsipher, MD         
United States, Georgia
Emory University Not yet recruiting
Atlanta, Georgia, United States, 30003
Contact: Edmund Waller, MD    404-727-4995   
Principal Investigator: Edmund Waller, MD         
Children's Healthcare of Atlanta Not yet recruiting
Atlanta, Georgia, United States, 30322
Contact: Muna Qayed, MD    404-785-6177   
Principal Investigator: Muna Qayed, MD         
United States, Kansas
University of Kansas Cancer Center Not yet recruiting
Fairway, Kansas, United States, 66205
Contact: Sunil Abhyankar, MD    913-588-6030   
Principal Investigator: Sunil Abhyankar, MD         
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Yi-Bin Chen, MD    617-724-1124   
Principal Investigator: Yi-Bin Chen, MD         
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: William Hogan, MD    507-319-6580   
Principal Investigator: William Hogan, MD         
United States, New York
The Mount Sinai Hospital Recruiting
New York, New York, United States, 10029
Contact: Rachel Young    212-659-5605   
Principal Investigator: John Levine, MD, MS         
United States, Ohio
Ohio State University Recruiting
Columbus, Ohio, United States, 43210
Contact: Hannah Choe, MD    614-293-3271   
Principal Investigator: Hannah Choe, MD         
United States, Pennsylvania
Children's Hospital of Philadelphia Not yet recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Stephen Grupp, MD    215-590-5475   
Principal Investigator: Stephen Grupp, MD         
University of Pennsylvania, Abramson Cancer Center Not yet recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: David Porter, MD    215-662-2862   
Principal Investigator: David Porter, MD         
United States, Tennessee
Vanderbilt University Recruiting
Nashville, Tennessee, United States, 37232
Contact: Carrie Kitko, MD    615-936-2088   
Principal Investigator: Carrie Kitko, MD         
United States, Texas
MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Contact: Amin Alousi, MD    713-745-8613   
Principal Investigator: Amin Alousi, MD         
Sponsors and Collaborators
John Levine
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Principal Investigator: John Levine, MD, MS Icahn School of Medicine at Mount Sinai

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Responsible Party: John Levine, Professor, Icahn School of Medicine at Mount Sinai Identifier: NCT03846479     History of Changes
Other Study ID Numbers: GCO 18-1684
First Posted: February 19, 2019    Key Record Dates
Last Update Posted: October 14, 2019
Last Verified: October 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by John Levine, Icahn School of Medicine at Mount Sinai:
Graft-versus-host disease
Additional relevant MeSH terms:
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Graft vs Host Disease
Immune System Diseases