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

Common Variable Immunodeficiency Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03188419
• Other study ID #: 999917104
• Secondary ID: 17-C-N104
• Status: Completed
• Start date: June 14, 2017
• Est. completion date: February 20, 2020

Study information

• Verified date: February 2020
• Source: National Institutes of Health Clinical Center (CC)
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 161
• Est. completion date: February 20, 2020
• Est. primary completion date: December 29, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 85 Years
• Eligibility: - Data Analysis Only

Related Conditions & MeSH terms

• Common Variable Immunodeficiency
• Immunologic Deficiency Syndromes
• Primary T-cell Immunodeficiency Disorders

Locations

Country	Name	City	State
United States	National Cancer Institute (NCI)	Bethesda	Maryland

Sponsors (1)

Lead Sponsor	Collaborator
National Cancer Institute (NCI)

Country where clinical trial is conducted

United States, 

References & Publications (2)

Gragert L, Eapen M, Williams E, Freeman J, Spellman S, Baitty R, Hartzman R, Rizzo JD, Horowitz M, Confer D, Maiers M. HLA match likelihoods for hematopoietic stem-cell grafts in the U.S. registry. N Engl J Med. 2014 Jul 24;371(4):339-48. doi: 10.1056/NEJMsa1311707. — View Citation

Hsieh MM, Kang EM, Fitzhugh CD, Link MB, Bolan CD, Kurlander R, Childs RW, Rodgers GP, Powell JD, Tisdale JF. Allogeneic hematopoietic stem-cell transplantation for sickle cell disease. N Engl J Med. 2009 Dec 10;361(24):2309-17. doi: 10.1056/NEJMoa0904971. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To determine the impact of genetic diseases and their modes of inheritance on the breadth of allo HSCT donor options	To determine the impact of genetic diseases and their modes of inheritance on the breadth of allo HSCT donor options	1 year