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CompletedNCT03188419Updated Feb 21, 2020

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

An observational study in Primary T-cell Immunodeficiency Disorders and Common Variable Immunodeficiency, sponsored by National Cancer Institute (NCI). Completed at 1 site in United States. Open to participants aged 4 Years to 85 Years. Per ClinicalTrials.gov, last updated 2020-02-21.

Sponsored by National Cancer Institute (NCI) · Observational

Study type
Observational
Model
Cohort
Time perspective
Retrospective
Enrollment
161
Ages
4 Years to 85 Years
Sex
All
01

Study 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.

Read the 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.

02

Conditions studied

  • Primary T-cell Immunodeficiency Disorders
  • Common Variable Immunodeficiency

Keywords

  • Prospective Donors
  • Immunodeficiency
03

Who can participate

Ages eligible
4 Years to 85 Years
Sexes eligible
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.

Eligibility criteria

  • Data Analysis Only
04

Study design

Observational model
Cohort
Time perspective
Retrospective
Enrollment
161 participants (actual)

Groups and cohorts

  • 1

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

05

What researchers measure

Primary outcomes

  1. 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

    Time frame: 1 year

06

Study locations

1 site
  • National Cancer Institute (NCI)
    Bethesda, Maryland 20892, United States
07

References and documents

Publications

  • 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. PubMed 20007560 ↗
  • 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. PubMed 25054717 ↗
08

Registry details

Key details

Study ID
NCT03188419
Lead sponsor
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Jun 15, 2017
Start date
Jun 14, 2017
Primary completion
Dec 29, 2019
Completion
Feb 20, 2020
Last update
Feb 21, 2020

Study contacts

Jennifer A Kanakry, M.D.
principal investigator · National Cancer Institute (NCI)

Oversight

FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Feb 2020. You cannot join it, but the record below documents what was studied.

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