Bone Marrow for Hemoglobinopathy Research
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT00669305 |
Recruitment Status :
Completed
First Posted : April 30, 2008
Last Update Posted : February 25, 2020
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Condition or disease |
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Sickle Cell Anemia Thalassemia |
Study Type : | Observational |
Actual Enrollment : | 26 participants |
Observational Model: | Cohort |
Time Perspective: | Prospective |
Official Title: | Bone Marrow for Hemoglobinopathy Research |
Actual Study Start Date : | May 15, 2008 |
Actual Primary Completion Date : | August 29, 2019 |
Actual Study Completion Date : | August 29, 2019 |

Group/Cohort |
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Cohort 1
Human participants affected with sickle cell disease or thalassemia will donate bone marrow for use in experimental models
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- Percentage of successful achievement of therapeutic level in mouse models resulting from retroviral vector mediated gene transfer, gene editing or drug treatment. [ Time Frame: 4 years ]The specific hypothesis to be tested is that a gene therapy vector, gene editing strategy, or drug modality can be designed that achieves a therapeutic level of globin production in transduced cells in mouse models.
Biospecimen Retention: Samples With DNA

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Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Sampling Method: | Probability Sample |
Inclusion Criteria:
- Patients with homozygous S/S disease or doubly heterozygous for S and β thalassemia who are 2 years or older are eligible. Patients with HbE- β- thalassemia or homozygous (severe) β-thalassemia are also eligible. Patients with thalassemia include those who are transfusion dependent (major) or severely anemic but relatively transfusion independent (intermedia). Diagnostic criteria include standard hematological parameters, red cell indices, hemoglobin electrophoresis and quantitative determination of HbF and HbA2.
- Patients are eligible for participation in the protocol only if they are currently clinically stable and have been free of all acute disease manifestations for a minimum of 14 days.
- Patients may participate while continuing their current therapeutic regimen including regular transfusion therapy or hydroxyurea administration.
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In general, two categories of patients will be considered as research participants in this protocol.
- Patients who are 18 years or older and therefore able to provide informed consent will be eligible. Such individuals will be recruited from among patients followed at SJCRH. In addition, individuals followed in an outside clinic who are recruited will be asked to come to the Hematology Clinic at SJCRH to enroll and have the procedure performed. Alternatively, if a patient who is 18 or older is to undergo a diagnostic or surgical procedure under general anesthesia, and they agree to participate in the study, the bone marrow aspirate will be obtained at that time.
- Patients between the ages of 2 and 17 years who are scheduled for a diagnostic or surgical procedure at SJCRH or LeBonheur Children's Medical Center for which sedation or general anesthesia is indicated will be eligible for protocol enrollment. A bone marrow aspiration will be performed during the sedation or general anesthesia for the diagnostic or surgical procedure.
Exclusion Criteria:
- Active, acute manifestations of sickle cell disease including painful crisis, acute chest syndrome, cerebrovascular events or active infection.
- Pregnant women will not be eligible for study enrollment.
- Inability or unwillingness of the research participant or legal guardian/representative to give written informed consent will preclude enrollment on this research protocol.
- Platelet count < 150,000/mm^3
- Neutrophil count < 2000/mm^3 (unless on hydroxyurea therapy)
- Neutrophil count < 1000/mm^3 for patients on hydroxyurea therapy
- Prothrombin Time > 17 seconds
- Partial thromboplastin Time > 43 seconds
- History of excessive bleeding in the context of previous procedures including surgery and dental extractions

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00669305
United States, Tennessee | |
St. Jude Children's Research Hospital | |
Memphis, Tennessee, United States, 38105 |
Principal Investigator: | Mitchell J. Weiss, MD, PhD | St. Jude Children's Research Hospital |
Responsible Party: | St. Jude Children's Research Hospital |
ClinicalTrials.gov Identifier: | NCT00669305 |
Other Study ID Numbers: |
BMR U54HL070590 ( U.S. NIH Grant/Contract ) P01HL053749 ( U.S. NIH Grant/Contract ) 201003 ( Other Identifier: Doris Duke Foundation ) |
First Posted: | April 30, 2008 Key Record Dates |
Last Update Posted: | February 25, 2020 |
Last Verified: | February 2020 |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Genetics Blood cells Vector-mediated gene transfer Bone marrow CD34 cells |
Thalassemia Anemia, Sickle Cell Hemoglobinopathies Anemia, Hemolytic, Congenital |
Anemia, Hemolytic Anemia Hematologic Diseases Genetic Diseases, Inborn |