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International Registry for Severe Chronic Neutropenia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00004342
Recruitment Status : Unknown
Verified October 2020 by David Chandler Dale, University of Washington.
Recruitment status was:  Recruiting
First Posted : October 19, 1999
Last Update Posted : October 22, 2020
University of Washington
Information provided by (Responsible Party):
David Chandler Dale, University of Washington

Brief Summary:

OBJECTIVES: I. Document the clinical course of severe chronic neutropenia (SCN).

II. Monitor and assess long term safety of primary treatment in SCN patients in the United States, Canada, Europe, and Australia.

III. Study the incidence and outcome of adverse events such as osteoporosis, splenomegaly, cytogenetic abnormalities, myelodysplastic syndrome, and leukemia.

IV. Evaluate growth and development and hematologic parameters. V. Monitor for clinically significant changes in primary treatment response over time.

VI. Establish a physician network to increase the understanding of SCN. VII. Establish a demographic database to allow for future research.

Condition or disease

Detailed Description:


Patients are treated by the referring physician as medically indicated. Clinical data are collected at baseline and then every 6 months.

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Study Type : Observational
Estimated Enrollment : 1000 participants
Observational Model: Other
Time Perspective: Cross-Sectional
Actual Study Start Date : June 1994

Adult Neutropenic Subject
Adult subjects with diagnosis of severe chronic neutropenia
Minor Neutropenic Subject
Children under 18 years of age who are diagnosed with severe chronic neutropenia
Parent of Minor Neutropenic Subjects
Parent of minor subjects (i.e., children under 18 years of age) who are diagnosed with severe chronic neutropenia

Biospecimen Retention:   Samples With DNA
Blood, Bone marrow, saliva

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
patients diagnosed with severe chronic neutropenia

Inclusion Criteria - Subjects are eligible for enrollment if they meet the following criteria:

  1. A confirmed diagnosis of severe chronic neutropenia based on documented absolute neutrophil counts of less than 0.5x109/L on at least three occasions in the three months prior to enrollment.
  2. For subjects with presumed cyclic neutropenia, documentation of at least two neutrophil cycles is preferred. Documentation should include the nadirs with neutrophil counts of less than 200 followed by a clear increase in the counts generally to at least 500 to 1000 followed by a second nadir, usually expected to occur at about three weeks after the first nadir, i.e., cycling with a three week periodicity. Documentation with at least six weeks of counts and two expected nadirs is preferred.

    Cases not showing clear oscillations will be categorized as congenital (if neutropenia or neutropenic complications appear to have occurred from birth) or idiopathic (if all symptoms in evidence point to an acquired disorder occurring after the first year of life).

  3. Bone marrow aspiration consistent with the diagnosis of congenital, cyclic or idiopathic neutropenia. In all of these conditions, it is expected that the marrow aspirate evaluation at the time of neutropenia will show a deficiency of mature neutrophils. An exception is myelokathexis, a condition with large accumulations of neutrophils with pycnotic nuclei in the marrow. Bone marrow aspirates may show some dyspoiesis of the neutrophil lineage, but abnormalities of erythropoiesis or platelet formation are, in general, inconsistent with the diagnosis of SCN.
  4. Normal cytogenetic evaluation. The only exception being cases of well documented severe congenital neutropenia with preferably previously documented normal cytogenetic evaluation will now be enrolled in the Registry at the time of evolution to leukemia.
  5. History of recurrent infections (i.e., severe mouth ulcers, gingivitis and sinusitis).
  6. Age greater than three months.
  7. Independent of hematological parameters, subjects with the following diagnoses may be included: Shwachman-Diamond syndrome (SDS), glycogen storage disease type 1b (GSD1b), Barth syndrome, and Cohen's syndrome.
  8. Subjects with moderately severe chronic neutropenia (i.e., ANC less than 1.0x109/L) and recurrent severe infections (i.e., deep tissue infections of subcutaneous areas, lungs, liver, etc.).
  9. Immune neutropenia with positive anti-neutrophil antibodies meeting criteria in 1, 3, 5 and 6.
  10. All SCN subjects originally enrolled in Amgen-sponsored SCN studies.

Exclusion Criteria

  1. Neutropenia known to be drug induced
  2. Primary myelodysplasia
  3. Primary leukemia
  4. Aplastic anemia
  5. Known HIV disease
  6. Systemic autoimmune diseases such as rheumatoid arthritis or systemic lupus erythematosus
  7. Chemotherapy-induced neutropenia (within the last 5 years)

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): NCT00004342

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United States, Massachusetts
Dana-Farber/Boston Children¹s Cancer and Blood Disorders Center Recruiting
Boston, Massachusetts, United States, 02115
Contact: Akiko Shimamura, MD, PhD    617-919-6109    Akiko.Shimamura@childrens.harvard.edu   
University of Massachusetts Recruiting
Worcester, Massachusetts, United States, 01655
Contact: Peter Newburger, MD    508-856-4225    Peter.Newburger@umassmed.edu   
United States, Michigan
University of Michigan Recruiting
Ann Arbor, Michigan, United States, 48109-0266
Contact: Kelly J. Walkovich, MD    734-647-2893    kwalkovi@umich.edu   
United States, New Jersey
St. Joseph's Children's Hospital Recruiting
Paterson, New Jersey, United States, 07503
Contact: MaryAnn Bonilla    973-754-3230    bonillam@sjhmc.org   
Contact: Caterna Lovaglio, RN/CRA    973-754-3778    lovaglic@sjhmc.org   
United States, Washington
University of Washington School of Medicine Recruiting
Seattle, Washington, United States, 98195
Contact: David Chandler Dale    206-543-7215      
Australia, Victoria
Monash University Recruiting
Melbourne, Victoria, Australia, 3350
Contact: Graham Leischke, PhD    +61 3 9902 9720    Graham.Lieschke@monash.edu   
Canada, Manitoba
CancerCare Manitoba Recruiting
Winnipeg, Manitoba, Canada, R3E 0V9
Contact: Bonnie Paula Cham    204-787-4147      
Canada, Ontario
Hospital for Sick Children Recruiting
Toronto, Ontario, Canada, M5G 1X8
Contact: Yigal Dror, MD    +416-813-5630    yigal.dror@sickkids.ca   
Contact: Philippa McCaffrey    +416-813-8886    phillippa.mccaffrey@sickkids.ca   
Medizinische Hochschule Hannover Recruiting
Hannover, Germany, D-30625
Contact: Connie Zeidler, Dr.med    +49-511-532-6710    zeidler.cornelia@mh-hannover.de   
Contact: Sonja Klein       Klein.Sonja@mh-hannover.de   
United Kingdom
Leeds Teaching Hospitals, Yorkshire Regional Centre for Paediatric Oncology & Haematology Recruiting
Leeds, England, United Kingdom, LS1 3EX
Contact: Sally Kinsey, MD    +44-113-392-8191      
Sponsors and Collaborators
National Center for Research Resources (NCRR)
University of Washington
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Study Chair: David Chandler Dale University of Washington
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Responsible Party: David Chandler Dale, Professor of Medicine, University of Washington
ClinicalTrials.gov Identifier: NCT00004342    
Other Study ID Numbers: 199/11901
First Posted: October 19, 1999    Key Record Dates
Last Update Posted: October 22, 2020
Last Verified: October 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 David Chandler Dale, University of Washington:
chronic neutropenia
disease-related problem/condition
hematologic disorders
rare disease
severe chronic neutropenia
Additional relevant MeSH terms:
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Leukocyte Disorders
Hematologic Diseases