Safety Study of Calcineurin Inhibitor Free GvHD Prophylaxis in Allogeneic Stem Cell Transplantation

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: NCT00856505
Recruitment Status : Unknown
Verified March 2009 by University Hospital Freiburg.
Recruitment status was:  Recruiting
First Posted : March 5, 2009
Last Update Posted : March 5, 2009
Information provided by:
University Hospital Freiburg

Brief Summary:
In stem cell transplantation as treatment for malignant diseases, calcineurin inhibitors like cyclosporine A are commonly used to prevent tissue destruction (GvHD) by activated donor immune cells. The hypothesis for this study is, that replacing calcineurin inhibitors by everolimus and mycophenolate as GvHD prophylaxis not only reduces toxicity of the treatment but also improves tolerance induction of the donor T cells toward the host, eventually increasing the safety of stem cell transplantation.

Condition or disease Intervention/treatment Phase
Hematologic Diseases Drug: Everolimus and mycophenolate sodium Phase 1 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 38 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Everolimus and Mycophenolate Sodium as GvHD Prophylaxis in Allogeneic Stem Cell Transplantation
Study Start Date : March 2008
Estimated Primary Completion Date : March 2010
Estimated Study Completion Date : March 2011

Arm Intervention/treatment
Experimental: Everolimus and mycophenolate sodium
Combination of experimental immunosuppressants for GvHD prophylaxis
Drug: Everolimus and mycophenolate sodium
Everolimus tablets, 1.5mg/day bid, dosage adjusted to plasma levels Mycophenolate sodium, 720mg/day bid Duration: Mycophenolate tapering starts at day 56 after stem cell transplantation Everolimus tapering starts at day 100 after stem cell transplantation if no GvHD evident

Primary Outcome Measures :
  1. Toxicity according to CTCAE v3.0 [ Time Frame: after 100 days and one year after treatment start ]

Secondary Outcome Measures :
  1. Hematopoietic engraftment [ Time Frame: day 30 after stem cell transplantation ]
  2. Incidence of acute and chronic GvHD [ Time Frame: one year after stem cell transplantation ]
  3. Progression free survival [ Time Frame: Day 100 and one year after stem cell transplantation ]
  4. Overall survival [ Time Frame: day 100 and one year after stem cell transplantation ]

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Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of hematologic malignancies, indicated for allogeneic stem cell transplantation:
  • acute myeloid leukemia (AML), in CR1, ≥ CR2, primary refractory, relapse
  • chronic myeloid leukemia (CML), in chronic phase, in acceleration or blast crisis
  • myelodysplastic syndrome (MDS), RA/RARS (transfusion dependent), RAEB, RAEB-t and CMML
  • Lymphoma:

    • plasmocytoma
    • immunocytoma (M. Waldenström)
    • chronic-lymphatic leukemia (CLL)
    • additional low and high grade Non-Hodgkin Lymphoma
  • Hodgkins disease
  • HLA-matched (HLA-A, -B, -DRB1) related or unrelated donor available
  • Signed informed consent

Exclusion Criteria:

  • CNS involvement by underlying disease
  • Pulmonary disease with VC < 55%, DLCO < 40%
  • Cardiac ejection fraction < 30%, uncontrollable arrhythmia
  • Creatinin > 1,5 mg/dl or Creatinin-Clearance < 30 ml/min
  • Bilirubin > 2 mg/dl
  • Active Hepatitis B or C
  • HIV serologic positive
  • Pregnancy and lactation
  • Pre-menstrual women without medical safe contraception
  • Participation on another clinical trial in between 30 days before start or during the study only if the clinical trial interferes with the outcome measures.
  • Known allergy to study medication or ingredients of the formulation
  • Drug- or alcohol abuse
  • Non-compliance

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): NCT00856505

Contact: Reinhard Marks, MD 49-761-270- ext 3278
Contact: Juergen Finke, MD 49-761-270- ext 3408

University Medical Center, Division Hematology/Oncology Recruiting
Freiburg, Baden-Wuerttemberg, Germany, 79104
Contact: Reinhard Marks, MD    49-761-270 ext 3278   
Sub-Investigator: Reinhard Marks, MD         
Principal Investigator: Juergen Finke, MD         
Sponsors and Collaborators
University Hospital Freiburg
Principal Investigator: Juergen Finke, MD University Medical Center Freiburg, Div. Hematology/Oncology
Study Director: Reinhard Marks, MD University Medical Center Freiburg, Div. Hematology/Oncology

Responsible Party: Prof. Jürgen Finke, University Hospital Freiburg Identifier: NCT00856505     History of Changes
Other Study ID Numbers: 00557
First Posted: March 5, 2009    Key Record Dates
Last Update Posted: March 5, 2009
Last Verified: March 2009

Keywords provided by University Hospital Freiburg:
Hematopoietic Stem Cell Transplantation
Drug Therapy, Combination

Additional relevant MeSH terms:
Hematologic Diseases
Mycophenolic Acid
Calcineurin Inhibitors
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antifungal Agents
Antibiotics, Antitubercular
Antitubercular Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action