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Ofatumumab as Primary Therapy of Chronic Graft Versus Host Disease

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. Read our disclaimer for details. Identifier: NCT01680965
Recruitment Status : Active, not recruiting
First Posted : September 7, 2012
Last Update Posted : December 13, 2019
Information provided by (Responsible Party):
H. Lee Moffitt Cancer Center and Research Institute

Brief Summary:
To study the safety and side effects of Ofatumumab in the treatment of chronic graft-versus-host disease (GvHD). This study will also evaluate effectiveness of Ofatumumab when added to standard steroid treatment for chronic graft-versus-host disease

Condition or disease Intervention/treatment Phase
Chronic Graft Versus Host Disease Drug: Ofatumumab Phase 1 Phase 2

Detailed Description:
This is a phase I-II trial to examine the safety and efficacy of prednisone and escalating dose of ofatumumab for the primary therapy of chronic GVHD.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 44 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Ofatumumab in Combination With Glucocorticoids for Primary Therapy of Chronic Graft Versus Host Disease
Actual Study Start Date : November 14, 2012
Estimated Primary Completion Date : September 2020
Estimated Study Completion Date : September 2022

Arm Intervention/treatment
Experimental: Ofatumumab

Phase I:

Escalating dose of ofatumumab

Phase II:

Maximum tolerated dose (MTD) of Ofatumumab

Drug: Ofatumumab

Phase I: test an escalating dose of ofatumumab at cohorts of 300 mg, 700 mg, and 1000 mg given on day 0 and 14 of study.

Phase II: Ofatumumab MTD on day 0 and 14; patients will be followed for total of 24 months (months 1, 3, 6, 12 after therapy, then at 18 and 24 months following therapy)

Primary Outcome Measures :
  1. Occurrence of Dose Limiting Toxicities [ Time Frame: within 28 days of initiation ]
    The dose limiting toxicity (DLT) will be Common Toxicity Criteria for Adverse Effects (CTCAE) version 4.0 defined grade 4 adverse events attributable to the study agent ofatumumab

Secondary Outcome Measures :
  1. Participants Response Rates [ Time Frame: 6 months following initiation of Ofatumumab ]
    Overall response rate (ORR) at 6 months following initiation of therapy represents the composite outcome of complete and partial response

  2. Overall Survival (OS) at 24 months [ Time Frame: Up to 24 months ]
    Overall Survival is defined as the time period from start of treatment to death.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Hematopoietic cell transplantation (HCT) recipients newly requiring systemic glucocorticoid therapy (at ≥ 1mg/kg/day prednisone or equivalent) for chronic GVHD
  • Participants can be enrolled and begin study therapy with ofatumumab within 14 days from initiation of 1 mg/kg/day prednisone for therapy of chronic GVHD.

Exclusion Criteria:

  • Relapse of primary hematologic malignancy that served as indication for HCT.
  • Previous systemic glucocorticoid therapy (at ≥ 1mg/kg/day prednisone or equivalent) for chronic GVHD
  • Prior systemic glucocorticoid therapy for acute GVHD is permitted
  • Prior or ongoing systemic immune suppressive agents (including, but not limited to common examples such as calcineurin inhibitors, sirolimus, mycophenolate mofetil) provided for either prevention or treatment of acute GVHD are permitted and part of routine standard of care
  • Current active hepatic or biliary disease (with exception of liver disease secondary to chronic GVHD, or patients with Gilbert's syndrome, asymptomatic gallstones, or stable chronic liver disease per investigator assessment).
  • Patients with abnormal liver function tests due to chronic GVHD are specifically not excluded from the study. This is a common manifestation of chronic GVHD, and thus a major target for the study therapy.
  • Treatment with experimental non-FDA approved therapy within 5 terminal half lives or 4 weeks prior to enrollment, whichever is longer
  • Other past or current solid tumor malignancy
  • Have been free of malignancy for at least 5 years, or have a history of completely resected non-melanoma skin cancer, or successfully treated in situ carcinoma are eligible.
  • Prior treatment with anti-CD20 monoclonal antibody or alemtuzumab within 3 months prior to start of therapy.
  • Uncontrolled infectious complications not responsive to appropriate antimicrobial therapy.
  • History of significant cerebrovascular disease (i.e. stroke or TIA) in the past 6 months or ongoing event with active symptoms or sequelae
  • HIV positivity
  • Uncontrolled, current significant cardiac disease including unstable angina, acute myocardial infarction within six months prior to randomization, congestive heart failure (NYHA III-IV), and arrhythmia unless controlled by therapy, with the exception of extra systoles or minor conduction abnormalities.
  • A history of cardiac disease, such as coronary disease, arrhythmia or congestive heart failure that are on appropriate medical therapy and without evidence of current decompensation are eligible.
  • Significant concurrent, uncontrolled medical condition including, but not limited to, renal, hepatic, gastrointestinal, endocrine, pulmonary, neurological, cerebral or psychiatric disease which in the opinion of the investigator may represent a risk for the patient.
  • Those patients with medical conditions that are controlled with medical therapy are eligible.
  • Clinically active Hepatitis B defined as positive HBsAg; or positive HBcAb with detectable hepatitis B virus (HBV) DNA viral load. Patients who are HBcAb with undetectable HBV DNA viremia are eligible.
  • Positive serology for hepatitis C (HC) defined as a positive test for HCAb and confirmed by HC RIBA or hepatitis C virus (HCV) RNA viral load
  • Screening laboratory value exclusion criteria: platelets < 50 x 10^9/L (patients with platelet counts > 50 x 10^9/L supported by platelet transfusion are eligible); neutrophils < 1.0 x 10^9/L (patients with an absolute neutrophil count > 1.0 x 10^9/L supported by growth factors are eligible); creatinine > 2.0 times upper normal limit; total bilirubin >1.5 times upper normal limit (unless due to chronic GVHD); alanine transaminase (ALT) > 2.0 times upper normal limit (unless due to chronic GVHD); alkaline phosphatase > 2.5 times upper normal limit (unless due to chronic GVHD).
  • Women who are pregnant or lactating. Women of childbearing potential must have a negative pregnancy test at screening.
  • Women of child bearing potential must undergo pregnancy testing within 7 days of the first dose of study therapy. Women must also undergo pregnancy test at 6 months after the last dose.
  • Women of childbearing potential, including women whose last menstrual period was less than one year prior to screening, unable or unwilling to use adequate contraception from study start to one year after the last dose of protocol therapy. Adequate contraception is defined as hormonal birth control, intrauterine device, double barrier method or total abstinence.
  • Males unable or unwilling to use adequate contraception methods from study start to one year after the last dose of protocol therapy.

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): NCT01680965

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United States, Arizona
Mayo Clinic Cancer Center
Phoenix, Arizona, United States, 85054
United States, Florida
H.Lee Moffitt Cancer Center & Research Institute
Tampa, Florida, United States, 33612
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
United States, Washington
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States, 98109
Sponsors and Collaborators
H. Lee Moffitt Cancer Center and Research Institute
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Principal Investigator: Joseph Pidala, MD, MS H. Lee Moffitt Cancer Center and Research Institute
Additional Information:
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Responsible Party: H. Lee Moffitt Cancer Center and Research Institute Identifier: NCT01680965    
Other Study ID Numbers: MCC-17071
First Posted: September 7, 2012    Key Record Dates
Last Update Posted: December 13, 2019
Last Verified: December 2019

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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 H. Lee Moffitt Cancer Center and Research Institute:
Graft vs. Host Disease
chronic graft versus host disease (cGVHD)
Allogeneic Transplant
Additional relevant MeSH terms:
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Graft vs Host Disease
Immune System Diseases
Antineoplastic Agents