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Sorafenib Maintenance Therapy for Patients With AML After Allogeneic Stem Cell Transplant

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: NCT01398501
Recruitment Status : Completed
First Posted : July 20, 2011
Last Update Posted : March 22, 2017
Dana-Farber Cancer Institute
Information provided by (Responsible Party):
Yi-Bin A. Chen, MD, Massachusetts General Hospital

Brief Summary:
Sorfenib works by slowing the spread of cancer cells. It has been used in other studies for patients with AML with the FLT3-ITD mutation and information from these studies suggests that sorafenib may help to control leukemia. The purpose of this study is to find the highest dose of sorafenib for maintenance therapy that can be safely used in participants with AML who have undergone allogeneic stem cell transplant.

Condition or disease Intervention/treatment Phase
Acute Myeloid Leukemia Drug: Sorafenib Phase 1

Detailed Description:

Subjects will taken sorafenib orally either once or twice daily. Subjects will come to the Bone Marrow Transplant Clinic 3 times (on Day 8, 15, and 30) during the first month of treatment. After the first month, they will be seen every month for 3 months and then at 9 at 6 and 9 months. Subjects will have a physical exam and be asked questions regarding general health and specific questions about any problems they might be having and any medications they are taking.

Subjects will have standard blood tests every month for 12 months to check liver and kidney function and complete blood count.

Subjects will have research blood tests on Days 8, 15 and 30 during the first month of treatment.

Subjects will have a bone marrow biopsy after 3 months and 12 months of treatment.

Subjects will receive treatment for up to 12 months and be followed for 1 year after completing the study.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 22 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Trial of Sorafenib Maintenance Therapy for Patients With FLT3-ITD AML After Allogeneic Stem Cell Transplantation
Study Start Date : August 2011
Actual Primary Completion Date : September 2015
Actual Study Completion Date : August 2016

Arm Intervention/treatment
Experimental: Post-SCT Sorafenib
Sorafenib will be given as maintenance therapy after allo HCT to patients with FLT3-ITD AML.
Drug: Sorafenib
Oral, 200 to 400 mg QD or BID
Other Name: BAY 43-9006

Primary Outcome Measures :
  1. Maximum Tolerated Dose [ Time Frame: 3 years ]
    To define the maximum tolerated dose (MTD) of maintenance sorafenib after allogeneic HSCT

Secondary Outcome Measures :
  1. Median number of days sorafenib tolerated [ Time Frame: 3 years ]
    Define the median number of days of sorafenib tolerated prior to dose-limiting toxicity or disease relapse

  2. Rate of serious infections [ Time Frame: 3 years ]
    Rate of serious infections (bacterial, viral, fungal, or other) after starting sorafenib

  3. Rate of acute GVHD [ Time Frame: 3 years ]
    Rate of grades II-IV acute graft-vs-host disease (GVHD) after starting sorafenib

  4. Rate of chronic GVHD [ Time Frame: 3 years ]
    Rates of significant chronic GVHD after starting sorafenib

  5. Survival [ Time Frame: 3 years ]
    1-year and 2-year progression-free and overall survival after HSCT

  6. Impact of sorafenib on bone marrow and serum levels of FLT3-ITD quantitative PCR [ Time Frame: 3 years ]
    To assess the impact of sorafenib on quantitative bone marrow and serum levels of FLT3-ITD DNA in patients (as measured by PCR) with FLT3-ITD AML after allogeneic SCT

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

Inclusion Criteria:

  • Subjects with AML with the FLT3-ITD mutation who have undergone allogeneic HSCT
  • Peripheral blood chimerism studies showing >/= 70% of all cells are of donor origin
  • Adequate hematologic and hepatic function
  • ECOG performance status 0-2
  • Able to swallow whole pills

Exclusion Criteria:

  • Evidence of relapsed/recurrent/residual disease as assessed by bone marrow aspirate and biopsy performed between days 30-60 after HSCT
  • Active acute graft vs host disease requiring an equivalent dose of > 0.5 mg/kg/day of prednisone or equivalent or those patients which necessitated the addition of another agent for the treatment of GVHD beyond corticosteroids
  • Ongoing uncontrolled infection
  • Cardiac disease: congestive heart failure > class II NYHA, unstable angina or new onset angina (began within the last 3 months) or myocardial infarction within the past 6 months
  • Cardiac ventricular arrhythmias requiring anti-arrhythmic therapy
  • Uncontrolled hypertension
  • Known HIV infection or chronic hepatitis B or C
  • Thrombotic or embolic events such as cerebrovascular accident including transient ischemic attacks within the past 6 months
  • Pulmonary hemorrhage/bleeding event > CTCAE v 4.0 Grade 2 within 4 weeks of starting study drug
  • Any other hemorrhage/bleeding event > CTCAE v. 4.0 Grade 3 within 4 weeks of starting study drug
  • Serious non-healing wound, non-healing ulcer, or bone fracture
  • Evidence or history of bleeding diathesis or coagulopathy
  • Major surgery or significant traumatic injury within 4 weeks of starting study drug
  • Use of St. John's Wort or rifampin (rifampicin)
  • Known or suspected allergy to sorafenib
  • Pregnant or breast-feeding
  • Receiving any other investigational agents

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): NCT01398501

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United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02214
Sponsors and Collaborators
Massachusetts General Hospital
Dana-Farber Cancer Institute
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Principal Investigator: Yi-Bin Chen, MD Massachusetts General Hospital
Publications of Results:
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Responsible Party: Yi-Bin A. Chen, MD, Physician, Massachusetts General Hospital Identifier: NCT01398501    
Other Study ID Numbers: 11-114
First Posted: July 20, 2011    Key Record Dates
Last Update Posted: March 22, 2017
Last Verified: March 2017
Keywords provided by Yi-Bin A. Chen, MD, Massachusetts General Hospital:
Stem cell transplant
Additional relevant MeSH terms:
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Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action