Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies

July 24, 2013 updated by: Catherine Wu, MD, Dana-Farber Cancer Institute

Multi-Center Study Using Allogeneic Stem Cell Transplantation Following Reduced Intensity Chemotherapy in Patients With Hemoglobinopathies

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

2

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Winship Cancer Institute-Emory University
    • Louisiana
      • Shreveport, Louisiana, United States, 71130
        • Feist-Weiller Cancer Center-LSU
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Dana-Farber Cancer Institute
      • Boston, Massachusetts, United States, 02115
        • Beth Israel Deaconess Medical Center
      • Boston, Massachusetts, United States, 02115
        • Massachusetts General Hospital
    • Ohio
      • Columbus, Ohio, United States, 43210
        • Ohio State University College of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
3 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
3 years
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.
3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Dana-Farber Cancer Institute

Collaborators

Massachusetts General Hospital
Beth Israel Deaconess Medical Center
Emory University
Brigham and Women's Hospital
Ohio State University
Feist-Weiller Cancer Center at Louisiana State University Health Sciences

Investigators

  • Principal Investigator: Catherine J. Wu, MD, Dana-Farber Cancer Institute

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2004

Primary Completion (Actual)

March 1, 2008

Study Completion (Actual)

July 1, 2009

Study Registration Dates

First Submitted

September 8, 2005

First Submitted That Met QC Criteria

September 8, 2005

First Posted (Estimate)

September 12, 2005

Study Record Updates

Last Update Posted (Estimate)

July 30, 2013

Last Update Submitted That Met QC Criteria

July 24, 2013

Last Verified

July 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 03-338

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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