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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
Scientific title
Secondary ID [1] 0 0
UW-730
Secondary ID [2] 0 0
199/11901
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Neutropenia 0 0
Condition category
Condition code
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
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
Comparator / control treatment
Control group

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.
Minimum age
3 Months
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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)

Study design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
UNKNOWN
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/06/1994
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
1000
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Monash University - Melbourne
Recruitment postcode(s) [1] 0 0
3350 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Massachusetts
Country [2] 0 0
United States of America
State/province [2] 0 0
Michigan
Country [3] 0 0
United States of America
State/province [3] 0 0
New Jersey
Country [4] 0 0
United States of America
State/province [4] 0 0
Washington
Country [5] 0 0
Canada
State/province [5] 0 0
Manitoba
Country [6] 0 0
Canada
State/province [6] 0 0
Ontario
Country [7] 0 0
Germany
State/province [7] 0 0
Hannover
Country [8] 0 0
United Kingdom
State/province [8] 0 0
England

Funding & Sponsors
Primary sponsor type
Government body
Name
National Center for Research Resources (NCRR)
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
University of Washington
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/study/NCT00004342
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.
Public notes

Contacts
Principal investigator
Name 0 0
David Chandler Dale
Address 0 0
University of Washington
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT00004342