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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT00004342
Registration number
NCT00004342
Ethics application status
Date submitted
18/10/1999
Date registered
19/10/1999
Date last updated
22/10/2020
Titles & IDs
Public title
International Registry for Severe Chronic Neutropenia
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Scientific title
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Secondary ID [1]
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UW-730
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Secondary ID [2]
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199/11901
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Neutropenia
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Condition category
Condition code
Blood
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Other blood disorders
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Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
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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Comparator / control treatment
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Control group
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Outcomes
Eligibility
Key inclusion criteria
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.
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Minimum age
3
Months
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
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)
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Study design
Purpose
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Duration
Cross-sectional
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Selection
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Timing
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Statistical methods / analysis
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Recruitment
Recruitment status
Unknown status
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/06/1994
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
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Sample size
Target
1000
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Monash University - Melbourne
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Recruitment postcode(s) [1]
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3350 - Melbourne
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Recruitment outside Australia
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United States of America
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State/province [1]
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Massachusetts
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United States of America
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State/province [2]
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Michigan
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United States of America
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State/province [3]
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New Jersey
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United States of America
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State/province [4]
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Washington
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Country [5]
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Canada
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State/province [5]
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Manitoba
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Country [6]
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Canada
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State/province [6]
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Ontario
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Country [7]
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Germany
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State/province [7]
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Hannover
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Country [8]
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United Kingdom
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State/province [8]
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England
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Funding & Sponsors
Primary sponsor type
Government body
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Name
National Center for Research Resources (NCRR)
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Address
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Other collaborator category [1]
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Other
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Name [1]
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University of Washington
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Address [1]
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT00004342
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Trial related presentations / 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.
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Public notes
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Contacts
Principal investigator
Name
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David Chandler Dale
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Address
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University of Washington
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT00004342
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