This study is currently recruiting participants.
Number
000464-H
Sponsoring Institute
National Heart, Lung and Blood Institute (NHLBI)
Recruitment Detail
Type: Enrolling by Invitation Gender: Male & Female Min Age: 4 Years Max Age: N/A
Referral Letter Required
Yes
Population Exclusion(s)
None
Keywords
donor cell leukemia; Bone Marrow Transplantation; increased leukemia among sickle anemia cell patients; malignant neoplasms in sickle cell anemia patients; barriers to transplantation for sickle cell anemia patients; Natural History
Recruitment Keyword(s)
Condition(s)
Leukemia; Malignant Neoplasms; sickle cell anemia disease
Investigational Drug(s)
Investigational Device(s)
Intervention(s)
Supporting Site
National Heart, Lung, and Blood Institute
Sickle cell disease (SCD) is a serious genetic blood disorder. It is often treated with a stem cell transplant. Researchers want to learn more about SCD. They want to use the existing research blood and bone marrow cells collected during transplant procedures to sequence participants DNA. This will help them learn if people with SCD may have a genetic mutation in their blood stem cells due to inflammation and their bone marrow working so hard to replace their red blood cells. They also want to learn if the mutations increase the risk of developing a blood cancer after blood stem cell transplant, gene therapy, and gene editing for SCD.
Objective:
To learn if the development of certain blood diseases is due to genetic mutations in blood stem cells.
Eligibility:
People aged 4 and older with SCD or beta-thalassemia who received curative treatments at NIH, and their donors.
Design:
Participants will not have any study visits.
Participants had blood and/or tissue samples collected in previous studies. They will be asked to allow those samples to be used for this research. The samples will be used to sequence their DNA. Data from their medical records will also be used. If they say no, then their samples and data will not be used.
Data and samples will be kept secure. Samples will be given an ID number. Data will be kept in a password-protected database.
Participants will not receive the results of tests performed on their samples. If they want to have genetic testing done, they will be given information on where they can get those tests.
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ELIGIBILITY CRITERIA: Patients with SCD or beta thalassemia who enrolled on curative therapies protocols and their donors. Biospecimens and data will be used from research participants with SCD who underwent curative therapies at the NIH and their donors: nonmyeloablative HLA-matched sibling HSCT (protocols 03-H-0170, 14-H-0077), nonmyeloablative haploidentical HSCT (protocols 09-H-0225, 17-H-0069), or myeloablative gene therapy (14-H-0155). Coded biospecimens and data will also be available from patients who enrolled in nonmyeloablative HLA-matched sibling or nonmyeloablative haploidentical HSCT at Johns Hopkins Hospital.
Patients with SCD or beta thalassemia who enrolled on curative therapies protocols and their donors. Biospecimens and data will be used from research participants with SCD who underwent curative therapies at the NIH and their donors: nonmyeloablative HLA-matched sibling HSCT (protocols 03-H-0170, 14-H-0077), nonmyeloablative haploidentical HSCT (protocols 09-H-0225, 17-H-0069), or myeloablative gene therapy (14-H-0155). Coded biospecimens and data will also be available from patients who enrolled in nonmyeloablative HLA-matched sibling or nonmyeloablative haploidentical HSCT at Johns Hopkins Hospital.
Principal Investigator
Referral Contact
For more information: