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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
Sponsor:
Collaborator:
University of Washington
Information provided by (Responsible Party):
David Chandler Dale, University of Washington

Tracking Information
First Submitted Date October 18, 1999
First Posted Date October 19, 1999
Last Update Posted Date October 22, 2020
Actual Study Start Date June 1994
Primary Completion Date Not Provided
Current Primary Outcome Measures Not Provided
Original Primary Outcome Measures Not Provided
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title International Registry for Severe Chronic Neutropenia
Official Title Not Provided
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.

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 Observational
Study Design Observational Model: Other
Time Perspective: Cross-Sectional
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Blood, Bone marrow, saliva
Sampling Method Non-Probability Sample
Study Population patients diagnosed with severe chronic neutropenia
Condition Neutropenia
Intervention Not Provided
Study Groups/Cohorts
  • 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
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Unknown status
Estimated Enrollment
 (submitted: June¬†23,¬†2005)
1000
Original Enrollment Same as current
Study Completion Date Not Provided
Primary Completion Date Not Provided
Eligibility 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.

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)
Sex/Gender
Sexes Eligible for Study: All
Ages 3 Months and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Australia,   Canada,   Germany,   United Kingdom,   United States
Removed Location Countries Italy
 
Administrative Information
NCT Number NCT00004342
Other Study ID Numbers 199/11901
UW-730
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Current Responsible Party David Chandler Dale, University of Washington
Original Responsible Party Not Provided
Current Study Sponsor National Center for Research Resources (NCRR)
Original Study Sponsor Same as current
Collaborators University of Washington
Investigators
Study Chair: David Chandler Dale University of Washington
PRS Account National Center for Research Resources (NCRR)
Verification Date October 2020