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A Pilot Trial to Assess the Feasibility and Safety of GCSF Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major


N/A
18 Years
N/A
Not Enrolling
Both
Beta Thalassemia Major, Congenital Anemias

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Trial Information

A Pilot Trial to Assess the Feasibility and Safety of GCSF Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major


Inclusion Criteria:



- Subjects must be 18 years or older

- Subjects may be of either gender or of any ethnic background

- Subjects must have a confirmed diagnosis of ßthalassemia major and have been enrolled
in a hypertransfusion program with a confirmed annual transfusion of ≥ or = to 100
mL/kg/yr AND ≥ or = to 8 Transfusions of blood per year over a minimum of two years.

- Patients must be off hydroxyurea (HU) or erythropoietin (EPO) treatment for at least
three months prior to entry onto the study

- Subjects must have a performance score of Karnofsky > or = to 70 of the time of entry
into the study.

- Subjects must have a normal EKG and a normal chest xray

- Each patient must be willing to participate as a research subject and must sign an
informed consent form.

- Subjects must be splenectomized or have no palpable spleen

- Negative pregnancy test, if female

Exclusion Criteria:

- Active infections including Hepatitis B and C, HTLV 1 and 2, West Nile Virus, and HIV
1

- Female patient pregnant or breast feeding

- Patients with uncontrolled seizure disorders

- Allergy to GCSF or bacterial E. coli products

- History of sickle cell disease or sickle trait

- History of thrombosis or known thrombophilia

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To investigate the feasibility and safety of peripheral blood hematopoietic progenitor cell (HPC) mobilization with GCSF in patients with βthalassemia major and to determine the yield of CD34+ HPCs mobilized.

Outcome Time Frame:

conclusion of study

Safety Issue:

Yes

Principal Investigator

Farid Boulad, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Memorial Sloan-Kettering Cancer Center

Authority:

United States: Institutional Review Board

Study ID:

08-030

NCT ID:

NCT00658385

Start Date:

April 2008

Completion Date:

February 2011

Related Keywords:

  • Beta Thalassemia Major
  • Congenital Anemias
  • GCSF
  • Anemia
  • Beta-Thalassemia
  • Thalassemia

Name

Location

Memorial Sloan-Kettering Cancer CenterNew York, New York  10021