NCT00059423

Natural History Study for BEN

Completed Last updated 22 March 2024

What this trial tests

trial in Neutropenia in 178 participants. Completed in 26 June 2020.

Timeline

3 June 2003

Primary endpoint
23 April 2019

26 June 2020

Quick facts

Lead sponsor	National Heart, Lung, and Blood Institute (NHLBI)
Status	Completed
Study type	OBSERVATIONAL
Enrollment	178
Start date	3 June 2003
Primary completion	23 April 2019
Estimated completion	26 June 2020
Sites	1 location across United States

Conditions studied

• Neutropenia — all drugs for Neutropenia →
• Agranulocytosis — all drugs for Agranulocytosis →
• Hematologic Diseases — all drugs for Hematologic Diseases →
• Leukocyte Disorders — all drugs for Leukocyte Disorders →

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Who can join

Adults 5 to 99, any sex, with Neutropenia or Agranulocytosis. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

In recent decades, hematologists have noticed that persons of African descent sometimes have lower white blood cell counts of a certain type, called granulocytes. These cells help to fight infections. The lower number of granulocytes in this situation does not appear to lead to more infections, and these individuals do not have any symptoms. This condition is called benign ethnic neutropenia (BEN), and is observed in a small percentage of individuals of African descent. This study will investigate the condition by studying people with and without BEN.

The goals of this study are to:

1. identify individuals of African descent with BEN.
2. determine the effects of two drugs, G-CSF and dexamethasone, on granulocyte production and movement.
3. determine whether there are differences in those with and without BEN in the way genes are stimulated after the administration of G-CSF and dexamethasone.

Study participants will be asked to interview with the research team, undergo physical exams, donate a blood sample, and receive G-CSF by injection, followed by dexamethasone (orally) about three weeks later. They also will be required to undergo apheresis three times, a procedure in which blood is drawn from a donor and separated into its components. Some components are retained for research analyses, such as granulocytes, and small amount of blood; the remainder is returned by transfusion to the donor. This procedure will be required of participants before they receive G-CSF, the day after they receive G-CSF, and the day after they receive dexamethasone. Gene messages (mRNA will be isolated from granulocytes, and analyzed to better understand granulocyte growth and movement.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Analyses of genome wide association data, cytokines, and gene expression in African-Americans with benign ethnic neutropenia.
   Charles BA, Hsieh MM, Adeyemo AA, Shriner D, et al · · 2018 · cited 38× · PMID 29596498 · DOI 10.1371/journal.pone.0194400
2. Natural history of benign ethnic neutropenia in individuals of African ancestry.
   Lakhotia R, Aggarwal A, Link ME, Rodgers GP, et al · · 2019 · cited 17× · PMID 30909074 · DOI 10.1016/j.bcmd.2019.01.009