Ignite Creation Date:
2024-05-05 @ 11:38 AM
Last Modification Date:
2024-10-26 @ 9:10 AM
Study NCT ID:
NCT00091871
Status:
RECRUITING
Last Update Posted:
2024-07-01
First Post:
2004-09-17
Brief Title:
A Longitudinal Study of Familial Hypereosinophilia FE Natural History and Markers of Disease Progression
Sponsor:
National Institute of Allergy and Infectious Diseases NIAID
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Longitudinal Study of Familial Hypereosinophilia FE Natural History and Markers of Disease Progression
Status:
RECRUITING
Status Verified Date:
2024-10-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Eosinophils are a type of white blood cell Elevated eosinophil levels can damage the heart nerves and other organs in the syndrome known as hypereosinophilic syndrome HES Some individuals have a hereditary form of HES known as familial eosinophilia FE More research on the causation and mechanisms of HES is needed in order to design more effective and less toxic therapies
This study will investigate FE and its genetic causes damage mechanisms and disease markers such as blood test abnormalities It will enroll approximately 50 individuals both adults and children from a previously studied family with FE This is a long-term study of indefinite duration
Participants will undergo yearly clinical examinations including medical history physical examination bloodwork EKG echocardiogram and pulmonary function tests with additional or more frequent examinations and tests as required In addition participants will donate blood and tissue for research purposes Both adult and child participants will donate blood At the initial evaluation adult participants will donate bone marrow During the study some adult participants will also undergo a limited number of leukaopheresis sessions in which blood is donated from one arm the blood is separated into red blood cells and other components and the red blood cells are returned into the donors other arm
This study will investigate FE and its genetic causes damage mechanisms and disease markers such as blood test abnormalities It will enroll approximately 50 individuals both adults and children from a previously studied family with FE This is a long-term study of indefinite duration
Participants will undergo yearly clinical examinations including medical history physical examination bloodwork EKG echocardiogram and pulmonary function tests with additional or more frequent examinations and tests as required In addition participants will donate blood and tissue for research purposes Both adult and child participants will donate blood At the initial evaluation adult participants will donate bone marrow During the study some adult participants will also undergo a limited number of leukaopheresis sessions in which blood is donated from one arm the blood is separated into red blood cells and other components and the red blood cells are returned into the donors other arm
Detailed Description:
Study Description
Affected and unaffected members of families with familial hypereosinophilia FE will be admitted on this protocol For affected family members a thorough clinical evaluation will be performed every 2 years with emphasis on potential sequelae of eosinophil-mediated tissue damage Blood cells bone marrow andor serum will also be collected to provide reagents such as DNA RNA and specific antibodies for use in the laboratory to address issues related to the genetic and immunologic basis of FE as well as its pathogenesis It is anticipated that affected family members will undergo a more extensive evaluation than is generally available and that the specimens collected from them will prove to be valuable reagents for laboratory studies related to eosinophilia eosinophil activation and function While the study is not designed to address the question of therapy for FE in patients for whom medical therapy is indicated for either the hypereosinophilia itself or its sequelae appropriate treatment will be instituted by our clinical service or the patients local physicians No experimental chemotherapy is involved in this protocol Unaffected family members will provide research specimens on this protocol to help determine the underlying genetic causes of FE
Objectives
Primary Objective To study the natural history of familial hypereosinophilia FE
Secondary Objectives
1 To determine the immunologic and molecular mechanisms responsible for eosinophilia eosinophil activation and pathogenesis in FE
2 To identify early clinical or laboratory markers of disease progression
Endpoints Primary Endpoint Development of eosinophilic end organ manifestations
Secondary Endpoints
1a Description of immunologic features of FE
1b Identification of genetic drivers of FE
2 Identification of clinical or laboratory markers that become abnormal prior to disease progression in FE
Affected and unaffected members of families with familial hypereosinophilia FE will be admitted on this protocol For affected family members a thorough clinical evaluation will be performed every 2 years with emphasis on potential sequelae of eosinophil-mediated tissue damage Blood cells bone marrow andor serum will also be collected to provide reagents such as DNA RNA and specific antibodies for use in the laboratory to address issues related to the genetic and immunologic basis of FE as well as its pathogenesis It is anticipated that affected family members will undergo a more extensive evaluation than is generally available and that the specimens collected from them will prove to be valuable reagents for laboratory studies related to eosinophilia eosinophil activation and function While the study is not designed to address the question of therapy for FE in patients for whom medical therapy is indicated for either the hypereosinophilia itself or its sequelae appropriate treatment will be instituted by our clinical service or the patients local physicians No experimental chemotherapy is involved in this protocol Unaffected family members will provide research specimens on this protocol to help determine the underlying genetic causes of FE
Objectives
Primary Objective To study the natural history of familial hypereosinophilia FE
Secondary Objectives
1 To determine the immunologic and molecular mechanisms responsible for eosinophilia eosinophil activation and pathogenesis in FE
2 To identify early clinical or laboratory markers of disease progression
Endpoints Primary Endpoint Development of eosinophilic end organ manifestations
Secondary Endpoints
1a Description of immunologic features of FE
1b Identification of genetic drivers of FE
2 Identification of clinical or laboratory markers that become abnormal prior to disease progression in FE
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 04-I-0286 | None | None | None |