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The Use of Etanercept Enbrel as Sole Treatment for Grade I Acute 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: NCT00726375
Recruitment Status : Completed
First Posted : July 31, 2008
Results First Posted : August 4, 2014
Last Update Posted : January 5, 2016
Information provided by (Responsible Party):
University of Michigan Rogel Cancer Center

Brief Summary:

This is a clinical trial to see if treatment with etanercept for early skin graft-versus-host disease (GVHD) can effectively treat and prevent progression of the disease without using high dose steroids.

GVHD is a common complication following a bone marrow transplant from another donor. GVHD occurs after transplant, when the donor's blood cells (called lymphocytes) recognize parts of your body, such as the skin, as foreign. A certain chemical, called Tumor Necrosis Factor, or TNF, also causes damage to the skin. The main effect on the skin is a red rash, when the skin GVHD is mild, but in more severe forms the skin can blister.

We have been studying GVHD at the University of Michigan for the past decade. We know that high levels of TNF makes GVHD worse. Our research has shown that adding an anti-TNF drug (called etanercept or Enbrel®) to the standard GVHD treatment of high dose steroids leads to improvement in the GVHD in twice as many patients compared to when steroids alone are used. It is now standard practice at the University of Michigan and many other centers to treat GVHD with both steroids and etanercept.

The management of early skin GVHD for most patients involves treatment with steroids, given both as a cream and by either the mouth (in pills) or IV. Early skin GVHD is also called grade I GVHD, which means the skin rash covers less than half of the body. Steroid treatment can be effective; however, it also causes many complications such as an increased risk of infection, weight gain, stomach ulcers, muscle weakness and bone damage, among many others. We have developed this study to test whether starting treatment with etanercept and steroid creams alone can treat the GVHD without requiring the use of high dose steroids. The goal is to avoid the complications that come with high dose oral or IV steroid treatment. The high dose steroid treatment would only begin if your GVHD got worse.

Condition or disease Intervention/treatment Phase
Acute Graft Versus Host Disease Drug: Etanercept (Enbrel) Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 34 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: The Use of Etanercept (Enbrel) as Sole Treatment for Grade I Acute Graft Versus Host Disease
Study Start Date : May 2008
Actual Primary Completion Date : July 2013
Actual Study Completion Date : September 2014

Arm Intervention/treatment
Experimental: Etanercept
a maximum of 8 SQ doses of 'Etanercept (Enbrel) at 0.4mg/kg per dose up to a maximum of 25 mg per dose
Drug: Etanercept (Enbrel)
Etanercept will begin within 72 hours of the diagnosis of Grade I acute GVHD and after consent for this study. Subjects receive eight doses of etanercept over four weeks. All doses will be administered by SQ injection. All subsequent doses will be given as subcutaneous injections into the skin. Injections will be given twice weekly with at least one day in between injections. The injections can be given in clinic, in the hospital, or self administered injections.

Primary Outcome Measures :
  1. The Percentage of Patients Who Progress Within 28 Days of Initiation of Etanercept Treatment [ Time Frame: 28 days ]
    We hypothesized that treatment of grade 1 acute GVHD (Graft Versus Host Disease) with etanercept would reduce the proportion of patients who progressed to grade 2 to 4 acute GVHD within 4 weeks of diagnosis from 58%, historically observed at our institution, to 38%.

Secondary Outcome Measures :
  1. The Number of Patients in Complete Remission (CR) at Four Weeks. [ Time Frame: 28 days ]

    Estimate the proportion of patients in complete remission (CR) at four weeks who remain alive and never require additional therapy four weeks after the last dose of etanercept.

    Complete remission is defined as the resolution of all manifestations of GVHD (Graft Versus Host Disease) within the first four weeks of treatment. All organs must have a Grade 0.

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:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Patient must have undergone HCT (donor cells from any source) with either a myeloablative or nonmyeloablative preparative regimen.
  2. Patient may be any age.
  3. Patient must have biopsy-proven Grade I acute GVHD (Appendix A). Biopsy report does not have to be back from Pathology prior to enrollment. Patients whose biopsy for GVHD identifies pathology inconsistent with GVHD will be removed from the study and replaced. However, because GVHD is a clinical diagnosis, biopsies which are non-diagnostic or do not show a clear non-GVHD etiology will not be cause to remove the patient from the study.

Exclusion Criteria:

  1. Patients who are pregnant (positive urine or serum test) or nursing.
  2. Active infections which are unresponsive to antibiotics (> 2 consecutive [at least 24 hours apart], positive blood cultures after initiation of treatment).
  3. Allergic or otherwise undesirable reaction to etanercept.
  4. Use of any oral or intravenous steroids at any previous time for GVHD treatment. Prior use of steroid therapy (i.e. hydrocortisone) as pre-medication for transfusions is permissible. Prior use of topical steroids is allowed.
  5. Use of etanercept for any other purpose.
  6. Noncompliance with medications.
  7. Grade II-IV GVHD (history of or at time of study entry).

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): NCT00726375

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United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
Sponsors and Collaborators
University of Michigan Rogel Cancer Center
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Principal Investigator: Sung Choi, MD The University of Michigan Comprehensive Cancer Center

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: University of Michigan Rogel Cancer Center Identifier: NCT00726375    
Other Study ID Numbers: UMCC 2007.139
HUM 17897 ( Other Identifier: University of MI Medical School IRB )
First Posted: July 31, 2008    Key Record Dates
Results First Posted: August 4, 2014
Last Update Posted: January 5, 2016
Last Verified: December 2015
Keywords provided by University of Michigan Rogel Cancer Center:
Additional relevant MeSH terms:
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Graft vs Host Disease
Immune System Diseases
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Inflammatory Agents
Antirheumatic Agents
Gastrointestinal Agents
Immunosuppressive Agents
Immunologic Factors