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

NCT03188419 | Completed

• 2 other identifiers: 99991710417-C-N104
• Study Type: observational
• Target: 161
• Locations: 1 country
• Sites: 1

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.

Conditions

Primary T-cell Immunodeficiency Disorders; Common Variable Immunodeficiency

Keywords

Prospective Donors; Immunodeficiency

Outcome Measures

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

  1 year

Study Arms (1)

1

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

Eligibility Criteria

• Age: 4 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

* Data Analysis Only

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (1)

National Cancer Institute (NCI)

Bethesda, Maryland, 20892, United States

Location

Related Publications (2)

• 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.

  PMID: 20007560 BACKGROUND

• 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.

  PMID: 25054717 BACKGROUND

MeSH Terms

Conditions

T cell immunodeficiency primary; Common Variable Immunodeficiency; Immunologic Deficiency Syndromes

Condition Hierarchy (Ancestors)

Immune System Diseases

Study Officials

• Jennifer A Kanakry, M.D.

  National Cancer Institute (NCI)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 14, 2017
• First Posted: June 15, 2017
• Study Start: June 14, 2017
• Primary Completion: December 29, 2019
• Study Completion: February 20, 2020
• Last Updated: February 21, 2020

Record last verified: 2020-02

Locations

US (1)