Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies - Full Text View

Sponsor:	Dana-Farber Cancer Institute
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:	Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier:	NCT00153985

Purpose

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	Intervention	Phase
Hemoglobinopathies Sickle Cell Disease Thalassemia	Drug: Busulfex Drug: Fludarabine Drug: Alemtuzumab Procedure: Stem Cell Transfusion	Phase II

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Historical Control, Single Group Assignment, Safety/Efficacy Study
Official Title:	Multi-Center Study Using Allogeneic Stem Cell Transplantation Following Reduced Intensity Chemotherapy in Patients With Hemoglobinopathies

Resource links provided by NLM:

Genetics Home Reference related topics: sickle cell disease

MedlinePlus related topics: Anemia Blood Transfusion and Donation Cancer Sickle Cell Anemia Thalassemia

Drug Information available for: Busulfan Fludarabine Fludarabine monophosphate Campath Alemtuzumab

U.S. FDA Resources

Further study details as provided by Dana-Farber Cancer Institute:

Primary Outcome Measures:

To determine if the preparative regimen of busulfex, fludarabine and alemtuzumab (CAMPATH) will generate stable engraftment with donor stem cells in patients with severe hemoglobinopathy. [ Time Frame: 3 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

To assess the safety of busulfex, fludarabine and alemtuzumab [ Time Frame: 3 years ] [ Designated as safety issue: Yes ]
to describe the incidence and severity of acute or chronic graft vs. host disease. [ Time Frame: 3 years ] [ Designated as safety issue: No ]

Estimated Enrollment:	20
Study Start Date:	March 2004
Study Completion Date:	July 2009
Primary Completion Date:	March 2008 (Final data collection date for primary outcome measure)

Intervention Details:

Given once daily for 4 days

Given intravenously once daily for 4 days

One day before fludarabine and busulfex are started, alemtuzumab will be given once daily for 5 days.

Performed three days after the end of chemotherapy

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.

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00153985

Locations

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
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Beth Israel Deaconess Medical Center
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

Principal Investigator:

Catherine J. Wu, MD

Dana-Farber Cancer Institute

More Information

No publications provided

Responsible Party:	Dana-Farber Cancer Institute ( Catherine J. Wu, MD )
Study ID Numbers:	03-338
Study First Received:	September 8, 2005
Last Updated:	January 4, 2010
ClinicalTrials.gov Identifier:	NCT00153985 History of Changes
Health Authority:	United States: Institutional Review Board

Keywords provided by 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:

Antimetabolites
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Immunologic Factors
Antineoplastic Agents
Hematologic Diseases
Physiological Effects of Drugs
Anemia
Anemia, Hemolytic
Fludarabine monophosphate
Thalassemia
Immunosuppressive Agents

Pharmacologic Actions
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Therapeutic Uses
Hemoglobinopathies
Busulfan
Alemtuzumab
Myeloablative Agonists
Antineoplastic Agents, Alkylating
Fludarabine
Alkylating Agents
Anemia, Sickle Cell

ClinicalTrials.gov processed this record on February 08, 2010