NeutropeniaNCT00004342
International Registry for Severe Chronic Neutropenia
An observational study of Neutropenia, run by National Center for Research Resources (NCRR) at 10 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 months and older
- Conditions the study is for
- Neutropenia
- How many people
- 1,000 participants
- Where
- 10 sitesin 5 countries
- Study dates
- Jun 1994to not given
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: patients diagnosed with severe chronic neutropenia
Who may be able to join
None listed.
Who cannot join
- 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)
Trial locations and contacts
10 sites in 5 countries.
10 sites in 5 countries
United States5 sites, 5 recruiting
- Recruiting
University of Michigan
Ann Arbor, Michigan, 48109-0266
Kelly J. Walkovich, MD, 734-647-2893, kwalkovi@umich.edu
- Recruiting
Dana-Farber/Boston Children¹s Cancer and Blood Disorders Center
Boston, Massachusetts, 02115
Akiko Shimamura, MD, PhD, 617-919-6109, Akiko.Shimamura@childrens.harvard.edu
- Recruiting
St. Joseph's Children's Hospital
Paterson, New Jersey, 07503
MaryAnn Bonilla, 973-754-3230, bonillam@sjhmc.org
Caterna Lovaglio, RN/CRA, 973-754-3778, lovaglic@sjhmc.org
- Recruiting
University of Washington School of Medicine
Seattle, Washington, 98195
David Chandler Dale, 206-543-7215
- Recruiting
University of Massachusetts
Worcester, Massachusetts, 01655
Peter Newburger, MD, 508-856-4225, Peter.Newburger@umassmed.edu
Canada2 sites, 2 recruiting
- Recruiting
Hospital for Sick Children
Toronto, Ontario, M5G 1X8
Yigal Dror, MD, +416-813-5630, yigal.dror@sickkids.ca
Philippa McCaffrey, +416-813-8886, phillippa.mccaffrey@sickkids.ca
- Recruiting
CancerCare Manitoba
Winnipeg, Manitoba, R3E 0V9
Bonnie Paula Cham, 204-787-4147
Australia1 site, 1 recruiting
- Recruiting
Monash University
Melbourne, Victoria, 3350
Graham Leischke, PhD, +61 3 9902 9720, Graham.Lieschke@monash.edu
Germany1 site, 1 recruiting
- Recruiting
Medizinische Hochschule Hannover
Hanover, D-30625
Connie Zeidler, Dr.med, +49-511-532-6710, zeidler.cornelia@mh-hannover.de
Sonja Klein, Klein.Sonja@mh-hannover.de
United Kingdom1 site, 1 recruiting
- Recruiting
Leeds Teaching Hospitals, Yorkshire Regional Centre for Paediatric Oncology & Haematology
Leeds, England, LS1 3EX
Sally Kinsey, MD, +44-113-392-8191
About this study
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.
Read the 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.
- Conditions
- Neutropenia
- Keywords
- chronic neutropenia, disease-related problem/condition, hematologic disorders, neutropenia, rare disease, severe chronic neutropenia
- Medical subject headings
- Neutropenia, Hematologic Diseases, Rare Diseases, Neutropenia, severe chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 |
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 1999 (estimated)
- Study startJun 1994
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for NeutropeniaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 1,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Center for Research Resources (NCRR)
- Sponsor type
- US National Institutes of Health
- Collaborators
- University of Washington
- Responsible party
- Principal investigator, David Chandler Dale, University of Washington
- Investigators
- David Chandler Dale, Study chair, University of Washington
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004342
- Sponsor's protocol ID
- 199/11901
- Other IDs
- UW-730
- Status last verified
- Oct 2020
- First submitted
- 18 Oct 1999
- Last updated
- 22 Oct 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
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.
PubMed 7655015Welte K, Dale D. Pathophysiology and treatment of severe chronic neutropenia. Ann Hematol. 1996 Apr;72(4):158-65. doi: 10.1007/s002770050156.
PubMed 8624368Kalra 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.
PubMed 8541548
Study at a glance
From the registry record, last updated by the sponsor on 22 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- International Registry for Severe Chronic Neutropenia
- ClinicalTrials.gov ID
- NCT00004342
- Acronym / sponsor ID
- 199/11901
- Conditions
- Neutropenia
- Keywords
- chronic neutropenia, disease-related problem/condition, hematologic disorders, neutropenia, rare disease, severe chronic neutropenia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 1,000 participants (estimated)
- Lead sponsor
- National Center for Research Resources (NCRR)US National Institutes of Health
- Collaborators
- University of Washington
- Study start
- Jun 1994 (actual)
- First posted
- 19 Oct 1999 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Other
- Time perspective
- Cross-sectional
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Ages
- 3 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- patients diagnosed with severe chronic neutropenia
Key inclusion criteria (0 listed)
Key exclusion criteria (7 listed)
- Neutropenia known to be drug induced
- Primary myelodysplasia
- Primary leukemia
- Aplastic anemia
- Known HIV disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Oct 2020.
- BackgroundDale 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.
- Background: PubMed 7655015Guerra 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.
- Background: PubMed 8624368Welte K, Dale D. Pathophysiology and treatment of severe chronic neutropenia. Ann Hematol. 1996 Apr;72(4):158-65. doi: 10.1007/s002770050156.
- Background: PubMed 8541548Kalra 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dana-Farber/Boston Children¹s Cancer and Blood Disorders Center
Boston, United States
David Chandler Dale study chair
University of Washington
10 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neutropenia, run by National Center for Research Resources (NCRR) at 10 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 22 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Canada, Australia, Germany, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00004342, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Oct 2020.