- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00004342
International Registry for Severe Chronic Neutropenia
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.
Study Overview
Status
Conditions
Detailed Description
PROTOCOL OUTLINE:
Patients are treated by the referring physician as medically indicated. Clinical data are collected at baseline and then every 6 months.
Study Type
Enrollment (Anticipated)
Contacts and Locations
Study Locations
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Victoria
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Melbourne, Victoria, Australia, 3350
- Recruiting
- Monash University
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Contact:
- Graham Leischke, PhD
- Phone Number: +61 3 9902 9720
- Email: Graham.Lieschke@monash.edu
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Manitoba
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Winnipeg, Manitoba, Canada, R3E 0V9
- Recruiting
- CancerCare Manitoba
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Contact:
- Bonnie Paula Cham
- Phone Number: 204-787-4147
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Ontario
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Toronto, Ontario, Canada, M5G 1X8
- Recruiting
- Hospital for Sick Children
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Contact:
- Yigal Dror, MD
- Phone Number: +416-813-5630
- Email: yigal.dror@sickkids.ca
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Contact:
- Philippa McCaffrey
- Phone Number: +416-813-8886
- Email: phillippa.mccaffrey@sickkids.ca
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Hannover, Germany, D-30625
- Recruiting
- Medizinische Hochschule Hannover
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Contact:
- Connie Zeidler, Dr.med
- Phone Number: +49-511-532-6710
- Email: zeidler.cornelia@mh-hannover.de
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Contact:
- Sonja Klein
- Email: Klein.Sonja@mh-hannover.de
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England
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Leeds, England, United Kingdom, LS1 3EX
- Recruiting
- Leeds Teaching Hospitals, Yorkshire Regional Centre for Paediatric Oncology & Haematology
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Contact:
- Sally Kinsey, MD
- Phone Number: +44-113-392-8191
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Massachusetts
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Boston, Massachusetts, United States, 02115
- Recruiting
- Dana-Farber/Boston Children¹s Cancer and Blood Disorders Center
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Contact:
- Akiko Shimamura, MD, PhD
- Phone Number: 617-919-6109
- Email: Akiko.Shimamura@childrens.harvard.edu
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Worcester, Massachusetts, United States, 01655
- Recruiting
- University of Massachusetts
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Contact:
- Peter Newburger, MD
- Phone Number: 508-856-4225
- Email: Peter.Newburger@umassmed.edu
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Michigan
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Ann Arbor, Michigan, United States, 48109-0266
- Recruiting
- University of Michigan
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Contact:
- Kelly J. Walkovich, MD
- Phone Number: 734-647-2893
- Email: kwalkovi@umich.edu
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New Jersey
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Paterson, New Jersey, United States, 07503
- Recruiting
- St. Joseph's Children's Hospital
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Contact:
- MaryAnn Bonilla
- Phone Number: 973-754-3230
- Email: bonillam@sjhmc.org
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Contact:
- Caterna Lovaglio, RN/CRA
- Phone Number: 973-754-3778
- Email: lovaglic@sjhmc.org
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Washington
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Seattle, Washington, United States, 98195
- Recruiting
- University of Washington School of Medicine
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Contact:
- David Chandler Dale
- Phone Number: 206-543-7215
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
Description
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.
Exclusion Criteria
- 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)
Study Plan
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
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Adult Neutropenic Subject
Adult subjects with diagnosis of severe chronic neutropenia
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Minor Neutropenic Subject
Children under 18 years of age who are diagnosed with severe chronic neutropenia
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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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Collaborators and Investigators
Collaborators
Investigators
- Study Chair: David Chandler Dale, University of Washington
Publications and helpful links
General Publications
- Dale DC, Bonilla MA, Boxer L, et al.: Development of AML/MDS in a subset of patients (PTS) with severe chronic neutropenia (SCN). Blood 84(10 suppl 1): 518a, 1994.
- Guerra J, Withers DA, Boxer LM. Myb binding sites mediate negative regulation of c-myb expression in T-cell lines. Blood. 1995 Sep 1;86(5):1873-80.
- Welte K, Dale D. Pathophysiology and treatment of severe chronic neutropenia. Ann Hematol. 1996 Apr;72(4):158-65. doi: 10.1007/s002770050156.
- Kalra R, Dale D, Freedman M, Bonilla MA, Weinblatt M, Ganser A, Bowman P, Abish S, Priest J, Oseas RS, Olson K, Paderanga D, Shannon K. Monosomy 7 and activating RAS mutations accompany malignant transformation in patients with congenital neutropenia. Blood. 1995 Dec 15;86(12):4579-86.
Study record dates
Study Major Dates
Study Start (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 199/11901
- UW-730
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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