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
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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|
|Study Type :||Observational|
|Estimated Enrollment :||1000 participants|
|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
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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|
Inclusion Criteria - Subjects are eligible for enrollment if they meet the following criteria:
- 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.
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).
- 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.
- 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.
- History of recurrent infections (i.e., severe mouth ulcers, gingivitis and sinusitis).
- Age greater than three months.
- 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.
- 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.).
- Immune neutropenia with positive anti-neutrophil antibodies meeting criteria in 1, 3, 5 and 6.
- All SCN subjects originally enrolled in Amgen-sponsored SCN studies.
- Neutropenia known to be drug induced
- Primary myelodysplasia
- Primary leukemia
- Aplastic anemia
- Known HIV disease
- Systemic autoimmune diseases such as rheumatoid arthritis or systemic lupus erythematosus
- Chemotherapy-induced neutropenia (within the last 5 years)
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
|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 firstname.lastname@example.org|
|United States, New Jersey|
|St. Joseph's Children's Hospital||Recruiting|
|Paterson, New Jersey, United States, 07503|
|Contact: MaryAnn Bonilla 973-754-3230 email@example.com|
|Contact: Caterna Lovaglio, RN/CRA 973-754-3778 firstname.lastname@example.org|
|United States, Washington|
|University of Washington School of Medicine||Recruiting|
|Seattle, Washington, United States, 98195|
|Contact: David Chandler Dale 206-543-7215|
|Melbourne, Victoria, Australia, 3350|
|Contact: Graham Leischke, PhD +61 3 9902 9720 Graham.Lieschke@monash.edu|
|Winnipeg, Manitoba, Canada, R3E 0V9|
|Contact: Bonnie Paula Cham 204-787-4147|
|Hospital for Sick Children||Recruiting|
|Toronto, Ontario, Canada, M5G 1X8|
|Contact: Yigal Dror, MD +416-813-5630 email@example.com|
|Contact: Philippa McCaffrey +416-813-8886 firstname.lastname@example.org|
|Medizinische Hochschule Hannover||Recruiting|
|Hannover, Germany, D-30625|
|Contact: Connie Zeidler, Dr.med +49-511-532-6710 email@example.com|
|Contact: Sonja Klein Klein.Sonja@mh-hannover.de|
|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|
|Study Chair:||David Chandler Dale||University of Washington|
|Responsible Party:||David Chandler Dale, Professor of Medicine, University of Washington|
|Other Study ID Numbers:||
|First Posted:||October 19, 1999 Key Record Dates|
|Last Update Posted:||October 22, 2020|
|Last Verified:||October 2020|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
severe chronic neutropenia