Participation in a Research Registry for Immune Disorders
|
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT01953016 |
|
Recruitment Status :
Recruiting
First Posted : September 30, 2013
Last Update Posted : April 20, 2021
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Background:
- People with primary immune deficiency diseases (PIDD) have weak immune systems. This makes it hard for their bodies to fight infection. The Immune Deficiency Foundation has a network to collect data about people with PIDD. It is called the United States Immunodeficiency Network. It will help doctors and scientists better understand these disorders. The goal is to get medical data for everyone with these disorders in the U.S. and Canada. Data will be stored in a registry. Researchers can use it to study if these disorders are increasing. They can also learn how the disorders are diagnosed and treated.
Objectives:
- To collect data on people with primary immune deficiency disorders.
Eligibility:
- People who have a PIDD.
Design:
- Data can be added with no record of personal identity.
- Data can be added with identity kept separate. This data will be linked to the registry by a code number.
- Data for the registry includes:
- Family history
- Disease treatment
- Disease characteristics
- Medical history
- Laboratory data
| Condition or disease |
|---|
| Primary Immunodeficiencies APECED CGD (Chronic Granulomatous Disease) Wiskott- Aldrich Syndrome SCID |
The purpose of this protocol is to provide a resource for clinical and laboratory research through enrollment of known immunodeficiency patients into a national registry, the US Immunodeficiency Network (USIDNET). The registry data will expand NIH s and the nation s knowledge base about immune deficiency disorders and genetic mutations that lead to these disorders. Additional registrants from NIH protocols will not only increase the understanding of the molecular basis of these disorders, but also will serve to document and track the incidence and progression of complications.
Objectives and specific aims
The purpose of this proposal is to create a mechanism for depositing NIH data into USIDNET. The patient Registry is designed to obtain longitudinal data on a large number of patients with primary immunodeficiency diseases, and genetic carriers of these defects in order to:
- Learn more about the phenotypic variations seen in a large number of individual patients with the same rare molecular diagnosis.
- Determine the natural history of these genetic disorders of immunity and establish genotype-phenotype correlations.
- Learn effects of various treatment protocols used in these patients over time, including unexpected side effects that may be unique to a particular diagnostic group.
- To evaluate quality of life using standard tools and correlate these with genotype and treatment history.
- To promote collaborative research amongst interested investigators by identifying a larger pool of potential research subjects than would be available at their own institutions
- To identify patients with a specific diagnosis for potential participation in multi-institutional clinical trials designed for diagnosis or therapy or their specific disease.
| Study Type : | Observational |
| Estimated Enrollment : | 1000 participants |
| Observational Model: | Cohort |
| Time Perspective: | Other |
| Official Title: | NIH Participation to USIDNET Registry |
| Actual Study Start Date : | September 30, 2013 |
| Estimated Primary Completion Date : | January 8, 2025 |
| Estimated Study Completion Date : | January 8, 2025 |
| Group/Cohort |
|---|
|
immunodeficiency
Individual of all ages, gender, and races with an immunodeficiency disorder from NIH studies, will be accepted for registration.
|
- Prevalence [ Time Frame: Ongoing ]The Registry will provide a minimum estimate of the prevalance of each disorder in US, a comprehensive clinical picture of each disorder, and a resource for clinical and laboratory research.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | Child, Adult, Older Adult |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
- INCLUSION CRITERIA
Individuals of all ages, gender, and races with an immunodeficiency disorder from NIH studies will be accepted for registration. No healthy volunteers will be enrolled.
EXCLUSION CRITERIA
Individuals with immunodeficiency associated with HIV infection, chemotherapy or other immunosuppressive therapies will not be accepted for registration unless there is clear evidence that these individuals also have a genetically determined immunodeficiency disease as well. Adult individuals who do not give informed consent will also be excluded.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01953016
| Contact: Elizabeth K Garabedian, R.N. | (301) 435-2443 | garabede@mail.nih.gov |
| United States, Maryland | |
| National Institutes of Health Clinical Center, 9000 Rockville Pike | Recruiting |
| Bethesda, Maryland, United States, 20892 | |
| Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR) 800-411-1222 ext TTY8664111010 prpl@cc.nih.gov | |
| Principal Investigator: | Elizabeth K Garabedian, R.N. | National Human Genome Research Institute (NHGRI) |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | National Human Genome Research Institute (NHGRI) |
| ClinicalTrials.gov Identifier: | NCT01953016 |
| Other Study ID Numbers: |
130199 13-HG-0199 |
| First Posted: | September 30, 2013 Key Record Dates |
| Last Update Posted: | April 20, 2021 |
| Last Verified: | March 9, 2021 |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
|
Job Syndrome/HIES GATA2 Primary Immunodeficiencies SCID DOCK8 |
|
Granulomatous Disease, Chronic Wiskott-Aldrich Syndrome Immunologic Deficiency Syndromes Immune System Diseases Phagocyte Bactericidal Dysfunction Leukocyte Disorders Hematologic Diseases |
Genetic Diseases, X-Linked Genetic Diseases, Inborn Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hemorrhagic Disorders Lymphopenia Leukopenia |