Atorvastatin for the Prophylaxis of Acute GVHD in Patients Undergoing Matched Sibling Allogeneic 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: NCT01175148
Recruitment Status : Completed
First Posted : August 4, 2010
Results First Posted : June 7, 2017
Last Update Posted : June 7, 2017
Information provided by (Responsible Party):
West Virginia University

Brief Summary:
Atorvastatin for prevention of acute GVHD

Condition or disease Intervention/treatment Phase
Graft vs Host Disease Drug: Atorvastatin calcium (Lipitor) Phase 2

Detailed Description:
This is a phase II study of atorvastatin for the prophylaxis of acute GVHD in patients undergoing matched-sibling allogeneic HSCT. This study will explore a two-pronged acute GVHD prophylaxis strategy, consisting of pre-treating consenting sibling donors with atorvastatin before stem cell collection, followed by the addition of atorvastatin to methotrexate/tacrolimus-based GVHD prophylaxis.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 60 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: Phase II Study Evaluating the Safety and Efficacy of Atorvastatin for the Prophylaxis of Acute Graft-versus-host Disease in Patients Undergoing Matched Sibling Hematopoietic Stem Cell Transplantation
Study Start Date : July 2010
Actual Primary Completion Date : December 2014
Actual Study Completion Date : December 2014

Arm Intervention/treatment
Experimental: Recipient - Atorvastatin to prevent GVHD
Atorvastatin calcium (Lipitor) will be administered at dose of 40mg orally daily starting on day -14, to permit an approximately 1-week observation period to rule out any acute atorvastatin-induced side effects before the initiation of transplant conditioning. Patients will receive atorvastatin until +180 days or development of grade 2 GVHD. This is the experimental arm for outcome measures.
Drug: Atorvastatin calcium (Lipitor)
40 mg PO daily
Other Name: Lipitor

Donor - Atorvastatin conditioning for donors
Sibling donors will start taking Atorvastatin calcium (Lipitor) orally at 40mg once daily between 14-28 days before the anticipated first day of apheresis or bone marrow harvest.
Drug: Atorvastatin calcium (Lipitor)
40 mg PO daily
Other Name: Lipitor

Primary Outcome Measures :
  1. Cummalative Incidence of Grade 2 to 4 Acute Graft vs Host Diesease (GVHD) at Day 100 Post Transplant in HSCT Recipients [ Time Frame: 100 days post transplant ]
    Cummalative incidence of grade 2 to 4 acute Graft vs Host Diesease (GVHD) at day 100 post transplant will be will be histologically confirmed and graded in Hematopoietic Stem Cell Transplantation Recipients

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  1. Donors must be ≥18 years of age, and willing/able to provide informed consent.
  2. Female donors of child-bearing potential should have a negative pregnancy test, and must be not be breast feeding.
  3. Adequate hepatic function with bilirubin, AST and ALT < 2.5 x upper limit of normal.
  4. Adequate renal function as defined by a serum creatinine clearance of ≥ 40% of normal calculated by Cockcroft-Gault equation.
  5. Adequate cardiac function as per institutional guidelines.
  6. Donors with positive HIV serologies are not eligible.
  7. No clinical evidence of uncontrolled active bacterial, viral or fungal infection at the time of stem cell mobilization.
  8. Donors must have a Karnofsky performance score of ≥60.
  9. Donors with history of intolerance or allergic reactions with atorvastatin will not be eligible. Hypersensitivity to any component of atorvastatin.
  10. Method of stem-cell collection from the sibling donor will be at the discretion of the treating physician. Although it is anticipated that majority of sibling donors will undergo G-CSF induced stem cell mobilization; however donors undergoing bone marrow harvest or stem cell mobilization with experimental agents (e.g. plerixafor) will remain eligible for the study.


  1. Patients with a history of a hematological malignancy or bone marrow failure syndrome suitable for matched sibling allogeneic stem cell transplantation in the opinion of treating transplant physician.
  2. Patients aged 18-75 years of age are eligible. Patients with age > 18 and ≤ 50 years will be eligible for myeloablative conditioning (MAC), while patients > 50 years of age, or those with previous history of autologous transplantation, high hematopoietic cell transplant comorbidity index (HCT-CI) score (>2), and baseline diagnosis of hodgkin's lymphoma, chronic lymphocytic leukemia and follicular lymphoma will be suitable for reduced intensity conditioning (RIC) transplantation (however intensity of conditioning regimen will remain at the discretion of treating physician).
  3. All patients must have at least one suitable HLA-matched sibling donor according to transplant center's guidelines (for selection of appropriate sibling donor).
  4. Patient must provide informed consent.
  5. Left ventricular ejection fraction > 40%. No uncontrolled arrhythmias or uncontrolled New York Heart Association class III-IV heart failure.
  6. Bilirubin <2mg/dl and AST and ALT < 3 x normal; and absence of hepatic cirrhosis.
  7. Adequate renal function as defined by a serum creatinine clearance of ≥ 40% of normal calculated by Cockcroft-Gault equation.
  8. DLCO (diffusion capacity; corrected for hemoglobin) ≥ 50% of predicted.
  9. Karnofsky performance status > 70.
  10. A negative pregnancy test will be required for all women of child bearing potential. Breast feeding is not permitted.
  11. Patients with positive HIV serology are not eligible.
  12. No evidence of active uncontrolled bacterial, viral or fungal infection at the time of transplant conditioning.
  13. Patients with history of intolerance or allergic reactions with atorvastatin will not be eligible.
  14. Patients who have previously been taking atorvastatin or any other statin drug will be eligible as long as there is no contraindication to switch to atorvastatin (40mg/day) in the opinion of the treating physician.
  15. Patients undergoing a T-cell depleted allogeneic transplantation will not be eligible.
  16. Patients receiving conditioning regimens containing antithymocyte globulin, and/or campath will not be eligible.

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): NCT01175148

United States, West Virginia
West Virginia University Hospitals Mary Babb Randolph Cancer Center
Morgantown, West Virginia, United States, 26506
Sponsors and Collaborators
West Virginia University
Principal Investigator: Mehdi Hamadani, MD West Virginia University

Responsible Party: West Virginia University Identifier: NCT01175148     History of Changes
Other Study ID Numbers: WVU11010
First Posted: August 4, 2010    Key Record Dates
Results First Posted: June 7, 2017
Last Update Posted: June 7, 2017
Last Verified: May 2017

Keywords provided by West Virginia University:
HMG-CoA Reductase Inhibitor
Hematopoietic stem cell transplant
allogeneic transplant
Graft vs Host Disease

Additional relevant MeSH terms:
Graft vs Host Disease
Immune System Diseases
Calcium, Dietary
Atorvastatin Calcium
Bone Density Conservation Agents
Physiological Effects of Drugs
Anticholesteremic Agents
Hypolipidemic Agents
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Hydroxymethylglutaryl-CoA Reductase Inhibitors
Enzyme Inhibitors