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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
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )

Brief Summary:

Background:

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.

Objective:

To find out how genetic diseases and the ways they are inherited affect the breadth of options for allo HSCT donors.

Eligibility:

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.

Design:

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

Detailed Description:

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.

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Study Type : Observational
Actual Enrollment : 161 participants
Observational Model: Cohort
Time Perspective: Retrospective
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


Group/Cohort
1
Retrospective chart review of patients with inherited immunodeficiency diseases requiring allogeneic hematopoietic stem cell transplant (allo HSCT)



Primary Outcome Measures :
  1. 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



Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 85 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Retrospective chart review of patients who with inherited immunodeficiency diseases requiring allogeneic hematopoietic stem cell transplant (allo HSCT)at the NIH Clinical Center.
Criteria
  • Data Analysis Only

Information from the National Library of Medicine

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


Locations
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United States, Maryland
National Cancer Institute (NCI)
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
National Cancer Institute (NCI)
Investigators
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Principal Investigator: Jennifer A Kanakry, M.D. National Cancer Institute (NCI)
Publications:
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Responsible Party: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT03188419    
Other Study ID Numbers: 999917104
17-C-N104
First Posted: June 15, 2017    Key Record Dates
Last Update Posted: February 21, 2020
Last Verified: February 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) ):
Prospective Donors
Immunodeficiency
Additional relevant MeSH terms:
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Immunologic Deficiency Syndromes
Common Variable Immunodeficiency
Immune System Diseases