Breadth of Donor Options for People With Inherited Diseases Requiring Allogeneic Hematopoietic Stem Cell Transplant in the Era of Alternative Donor Transplants Using Post-Transplantation Cyclophosphamide
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|ClinicalTrials.gov Identifier: NCT03188419|
Recruitment Status : Completed
First Posted : June 15, 2017
Last Update Posted : February 21, 2020
People who have certain immune system diseases often need a procedure called allo HSCT. This is short for allogeneic hematopoietic stem cell transplant. This might cure people with these diseases. Many people who need allo HSCT need donors who are relatives with similar genes. But the disease may also affect those in the donor pool. This may mean there are fewer options for people with inherited diseases. Researchers want to collect data on how transplant candidates and their donors are found.
To find out how genetic diseases and the ways they are inherited affect the breadth of options for allo HSCT donors.
Records from studies that have already been done. These will be for people ages 4 and older who were evaluated for allo HSCT or to be donors.
Participants already signed a consent form for their records to be shared.
Researchers will study the participant data.
Data will be stored in an electronic system. Researchers will use passwords to protect the data.
|Condition or disease|
|Primary T-cell Immunodeficiency Disorders Common Variable Immunodeficiency|
This protocol is a retrospective review of donor search results for patients with inherited immunodeficiency diseases requiring allogeneic hematopoietic stem cell transplant (allo HSCT).
The study will involve collecting information related to the donor search for transplant candidates and their prospective donors, using records in CRIS, Crimson, the HLA lab, and records in the transplant coordinator office (such as shipping logs of HLA typing kits). The study will not involve the use of specimens or participant contact.
The participants whose records will be reviewed will be those who were evaluated for allo HSCT or donation on an NIH primary immunodeficiency transplant protocol at a time when haplo donors were eligible (March 30, 2012 to present for GATA2, January 1, 2015 for CGD, October 6, 2015 to present for 16-C-0003, and May 21, 2014 to present for DOCK8).
The Principal Investigators on the included protocols have granted permission to conduct this study and have verified that none of the original protocols or informed consent documents preclude such a review of clinical data.
|Study Type :||Observational|
|Actual Enrollment :||161 participants|
|Official Title:||Retrospective Study of the Breadth of Donor Options for Patients With Inherited Diseases Requiring Allogeneic Hematopoietic Stem Cell Transplant in the Era of Alternative Donor Transplants Using Post-Transplantation Cyclophosphamide|
|Actual Study Start Date :||June 14, 2017|
|Actual Primary Completion Date :||December 29, 2019|
|Actual Study Completion Date :||February 20, 2020|
Retrospective chart review of patients with inherited immunodeficiency diseases requiring allogeneic hematopoietic stem cell transplant (allo HSCT)
- To determine the impact of genetic diseases and their modes of inheritance on the breadth of allo HSCT donor options [ Time Frame: 1 year ]To determine the impact of genetic diseases and their modes of inheritance on the breadth of allo HSCT donor options
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): NCT03188419
|United States, Maryland|
|National Cancer Institute (NCI)|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Jennifer A Kanakry, M.D.||National Cancer Institute (NCI)|