Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major

This study has been completed.
Sponsor:
Information provided by:
Memorial Sloan-Kettering Cancer Center
ClinicalTrials.gov Identifier:
NCT00658385
First received: April 10, 2008
Last updated: February 11, 2011
Last verified: February 2011
  Purpose

Betathalassemia major is a disease of the blood and bone marrow. You were born with it and it has made you unable to make normal hemoglobin and red cells. You have been receiving red blood cell transfusions all your life. These transfusions do not cure your disease. The problem with transfusions is that they contain a lot of iron. With time iron builds up in your body and will eventually hurt some of your organs . Because of this buildup of iron , you are taking medicine that helps your body get rid of the extra iron.

Today, the only other treatment is bone marrow or stem cell transplant. It can only be done when a matched donor is available. This is most often a brother, sister, or parent. Bone marrow transplant may cure betathalassemia major. If you have a transplant and it is successful, you will no longer have the disease. Without a matched sibling or parent, the standard treatment is to keep having transfusions.

In the near future, we will be testing a new treatment for making normal hemoglobin and normal red blood cells. We have recreated the healthy hemoglobin gene in a test tube. We are able to use it and put it back into cells. This is called gene therapy. We have been able to put this gene into the stem cells of mice with thalassemia. These mice were cured. We now plan to take that gene and put it into stem cells from people who have betathalassemia major. We will then inject those stem cells back into that person's blood.

In general, we can obtain more stem cells from the blood of a person than from the bone marrow . In order to do so, we must give that person a blood growth factor. The growth factor stimulates the bone marrow to make more stem cells. That growth factor is called granulocyte colony stimulating factor (GCSF), or Filgrastim.

The purpose of this trial is to find out if the drug GCSF has any side effects on you, and if you will make more stem cells in response to it. This trial is not a gene therapy trial. This trial will not help your thalassemia.


Condition Intervention
Beta Thalassemia Major
Congenital Anemias
Genetic: GCSF, Central venous line placement, Stem cell Collection (leukapheresis)

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Pilot Trial to Assess the Feasibility and Safety of GCSF Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major

Resource links provided by NLM:


Further study details as provided by Memorial Sloan-Kettering Cancer Center:

Primary Outcome Measures:
  • 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. [ Time Frame: conclusion of study ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • To determine whether the collected HPCs are amenable to transduction with a lentiviral vector encoding the normal ßglobin gene. [ Time Frame: conclusion of study ] [ Designated as safety issue: No ]

Enrollment: 5
Study Start Date: April 2008
Study Completion Date: February 2011
Primary Completion Date: February 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
GCSF (human recombinant granulocyte colony stimulating factor)Neupogen(Amgen), Filgrastim, Central venous line placement, Stem cell Collection (leukapheresis)
Genetic: GCSF, Central venous line placement, Stem cell Collection (leukapheresis)

Daily injections under the skin of a GCSF. This is done for 5 to 6 days. On days 1, 3,5, and if need on day 6. To collect stem cells, we need good access to this blood. If the patient has good veins, we do this by placing an IV on each one of their arms. The peripheral blood stem cell collection is usually an outpatient procedure and takes about 3 to 4 hours. You will have blood work and a physical exam on days one, three, and five while you are getting GCSF.

These will be done again 24 hours after your stem cells are collected.


  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00658385

Locations
United States, New York
Memorial Sloan-Kettering Cancer Center
New York, New York, United States, 10065
Sponsors and Collaborators
Memorial Sloan-Kettering Cancer Center
Investigators
Principal Investigator: Farid Boulad, MD Memorial Sloan-Kettering Cancer Center
  More Information

Additional Information:
No publications provided

Responsible Party: Farid Boulad, MD, Memorial Sloan-Kettering Cancer Center
ClinicalTrials.gov Identifier: NCT00658385     History of Changes
Other Study ID Numbers: 08-030
Study First Received: April 10, 2008
Last Updated: February 11, 2011
Health Authority: United States: Institutional Review Board

Keywords provided by Memorial Sloan-Kettering Cancer Center:
GCSF

Additional relevant MeSH terms:
Anemia
Beta-Thalassemia
Thalassemia
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Lenograstim
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 17, 2014