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Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies

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.
 
ClinicalTrials.gov Identifier: NCT00153985
Recruitment Status : Completed
First Posted : September 12, 2005
Results First Posted : March 12, 2013
Last Update Posted : July 30, 2013
Sponsor:
Collaborators:
Beth Israel Deaconess Medical Center
Massachusetts General Hospital
Brigham and Women's Hospital
Emory University
Feist-Weiller Cancer Center at Louisiana State University Health Sciences
Ohio State University
Information provided by (Responsible Party):
Catherine Wu, MD, Dana-Farber Cancer Institute

Study Description
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Brief Summary:
The purpose of this study is to determine if treatment with reduced-dose busulfex, fludarabine and alemtuzumab (CAMPATH) followed by sten cell infusion will allow for donor stem cells to grow in patients with hemoglobinopathies bone marrow and restore circulating blood counts. In addition the incidence and severity of side effects and of graft vs. host disease (GVHD) will be monitored.

Condition or disease Intervention/treatment Phase
Hemoglobinopathies Sickle Cell Disease Thalassemia Drug: Busulfex Drug: Fludarabine Drug: Alemtuzumab Procedure: Stem Cell Transfusion Phase 2

Detailed Description:
  • In order to undergo transplant procedure, patients will be admitted to the hospital for approximately 10-14 days.
  • To prepare patient's bone marrow to accept donor stem cells, they will receive fludarabine and busulfex. Fludarabine will be given intravenously once daily for 4 days. Busulfex will be given once daily for the same 4 days.
  • One day before patients receive busulfex and fludarabine, they will also be given alemtuzumab intravenously once daily for 5 days.
  • Three days after the end of chemotherapy, patients will receive the infusion of donor stem cells.
  • If patients have thalassemia, they will receive subcutaneous injections of filgrastim starting on day one after the donor stem cell transfusion and will continue receiving filgrastim every day until it appears that the donor stem cells have been accepted. If the patient has sickle cell disease, filgrastim will not be given,
  • Additional drugs will be given to help prevent infection (i.e. antibiotics).
  • After stem cell infusion patients will be examined and have blood tests weekly for 1 month. Bone marrow biopsies, and blood work will also be performed 1 month, 3 months, 6 months and 1 year after stem cell infusion.
  • Patients will be on the study for about 12 months. After study is completed progress will be monitored on an annual basis.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Multi-Center Study Using Allogeneic Stem Cell Transplantation Following Reduced Intensity Chemotherapy in Patients With Hemoglobinopathies
Study Start Date : March 2004
Actual Primary Completion Date : March 2008
Actual Study Completion Date : July 2009

Resource links provided by the National Library of Medicine

Drug Information available for: Alemtuzumab

Arms and Interventions
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Intervention Details:
  • Drug: Busulfex
    Given once daily for 4 days
  • Drug: Fludarabine
    Given intravenously once daily for 4 days
  • Drug: Alemtuzumab
    One day before fludarabine and busulfex are started, alemtuzumab will be given once daily for 5 days.
    Other Name: CAMPATH
  • Procedure: Stem Cell Transfusion
    Performed three days after the end of chemotherapy

Outcome Measures
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Primary Outcome Measures :
  1. Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy. [ Time Frame: 3 years ]
    Outcome was measured by ANC >500 for three consecutive days prior to day 30 after PBSC infusion, >25% of hematopoietic cells are donor derived as determined by molecular chimerism assays or cytogenetic methods prior to day 45 after PBSC infusion and >25% of hematopoietic cells are donor derived as determined by molecular chimerism assays or cytogenetic methods after day 180 after PBSC infusion.


Secondary Outcome Measures :
  1. Solid Organ Toxicity Related to the Conditioning Regimen. [ Time Frame: 3 years ]
    Outcome was measured by the assessment of organ toxicity related to Busulfex, fludarabine and alemtuzumab.

  2. The Incidence of Grade II-IV Acute Graft vs. Host Disease. [ Time Frame: 3 years ]
    Outcome was measured by incidence and severity of acute and chronic GVHD following donor stem cell infusion.


Eligibility Criteria
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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
Criteria

Inclusion Criteria:

  • Patients with sickle cell disease should have one or more of the following: acute chest syndrome requiring hospitalization; nonhemorrhagic stroke or central nervous system event lasting longer than 24 hours; recurrent caso-occlusive pain or recurrent priapism; sickle neuropathy; bilateral proliferative retinopathy and major visual impairment of at least one eye; osteonecrosis of multiple joints; transfusion dependence; vaso-occlusive.
  • Patients with thalassemia should have one or more of the following: transfusion dependence; iron overload; presence of 2 or more alloantibodies against red cell antigens.

Exclusion Criteria:

  • Pregnancy
  • Acute hepatitis
  • Cardiac ejection fraction < 30%
  • Severe renal impairment
  • Severe residual functional neurologic impairment
  • Evidence of HIV infection
Contacts and Locations
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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 ClinicalTrials.gov identifier (NCT number): NCT00153985


Locations
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United States, Georgia
Winship Cancer Institute-Emory University
Atlanta, Georgia, United States, 30322
United States, Louisiana
Feist-Weiller Cancer Center-LSU
Shreveport, Louisiana, United States, 71130
United States, Massachusetts
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States, 02115
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Massachusetts General Hospital
Boston, Massachusetts, United States, 02115
United States, Ohio
Ohio State University College of Medicine
Columbus, Ohio, United States, 43210
Sponsors and Collaborators
Dana-Farber Cancer Institute
Beth Israel Deaconess Medical Center
Massachusetts General Hospital
Brigham and Women's Hospital
Emory University
Feist-Weiller Cancer Center at Louisiana State University Health Sciences
Ohio State University
Investigators
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Principal Investigator: Catherine J. Wu, MD Dana-Farber Cancer Institute
More Information
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Publications of Results:

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Responsible Party: Catherine Wu, MD, Principal Investigator, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier: NCT00153985    
Other Study ID Numbers: 03-338
First Posted: September 12, 2005    Key Record Dates
Results First Posted: March 12, 2013
Last Update Posted: July 30, 2013
Last Verified: July 2013
Keywords provided by Catherine Wu, MD, Dana-Farber Cancer Institute:
Hemoglobinopathies
Sickle cell anemia
sickle cell-hemoglobin C disease
sickle cell-B-thalassemia
transfusion-dependant thalassemia
 allogeneic transplant
nonmyeloablative transplant
Stem cell transfusion
graft vs. host disease
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Thalassemia
Hemoglobinopathies
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Genetic Diseases, Inborn
Fludarabine
Alemtuzumab
 Busulfan
Antineoplastic Agents
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antineoplastic Agents, Immunological
Alkylating Agents
Antineoplastic Agents, Alkylating
Myeloablative Agonists