- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00658385
Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major
A Pilot Trial to Assess the Feasibility and Safety of GCSF Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major
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.
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Not Applicable
Contacts and Locations
Study Locations
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New York
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New York, New York, United States, 10065
- Memorial Sloan Kettering Cancer Center
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: 1
GCSF (human recombinant granulocyte colony stimulating factor)Neupogen(Amgen), Filgrastim, 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. |
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Participants With no Serious Adverse Events
Time Frame: Up to 14 Days
|
The entered value represents the number of participants with the absence of serious adverse events.
G-CSF mobilization will be considered safe if there are no more than 1 of 5 patients with SAEs
|
Up to 14 Days
|
Collaborators and Investigators
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start
Primary Completion (ACTUAL)
Study Completion (ACTUAL)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ESTIMATE)
Study Record Updates
Last Update Posted (ESTIMATE)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 08-030
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.
Clinical Trials on Beta Thalassemia Major
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CelgeneTerminatedBeta Thalassemia Intermedia | Beta Thalassemia MajorFrance, United Kingdom, Italy, Greece
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M.D. Anderson Cancer CenterWithdrawnSickle Cell Disease | Sickle Beta Thalassemia | Beta Thalassemia Major | Sickle Cell-SS Disease | Sickle Beta 0 Thalassemia | Sickle Beta Plus ThalassemiaUnited States
-
CorrectSequence Therapeutics Co., LtdNot yet recruitingBeta-Thalassemia MajorChina
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First Affiliated Hospital of Guangxi Medical UniversityUnknown
-
Editas Medicine, Inc.RecruitingHemoglobinopathies | Thalassemia Major | Thalassemia Intermedia | Transfusion Dependent Beta ThalassemiaUnited States, Canada
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Aga Khan UniversityCompletedBeta Thalassemia Major
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China Medical University HospitalCompleted
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EmeraMedCompletedBeta Thalassemia MajorAlbania
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Tanta UniversityCompletedBeta Thalassemia MajorEgypt
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Nanfang Hospital of Southern Medical UniversityGuangdong Yike Gene Science and Technology CO.,LtdUnknownBeta Thalassemia MajorChina
Clinical Trials on GCSF, Central venous line placement, Stem cell Collection (leukapheresis)
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Centre Chirurgical Marie LannelongueNot yet recruiting
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City of Hope Medical CenterNational Cancer Institute (NCI)RecruitingAcute Myeloid Leukemia | Hodgkin Lymphoma | Myelofibrosis | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Non-Hodgkin Lymphoma | Myelodysplastic Syndrome | Hematopoietic and Lymphoid System Neoplasm | Myeloproliferative Neoplasm | Chronic Myeloid Leukemia, BCR-ABL1 PositiveUnited States
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University of Wisconsin, MadisonCompletedTetralogy of FallotUnited States
-
TriStar HealthWithdrawn
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Nationwide Children's HospitalWithdrawnCrohn's Disease
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National Heart, Lung, and Blood Institute (NHLBI)National Cancer Institute (NCI); National Marrow Donor Program; Blood and Marrow...CompletedMultiple MyelomaUnited States
-
M.D. Anderson Cancer CenterNovartisCompleted
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Dana-Farber Cancer InstituteMassachusetts General Hospital; Beth Israel Deaconess Medical CenterTerminated
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National Cancer Institute (NCI)RecruitingDOCK8 DeficiencyUnited States
-
University of Alabama at BirminghamRecruiting