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Participation in a Research Registry for Immune Disorders

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ClinicalTrials.gov Identifier: NCT01953016
Recruitment Status : Recruiting
First Posted : September 30, 2013
Last Update Posted : February 7, 2019
Sponsor:
Collaborators:
National Institute of Allergy and Infectious Diseases (NIAID)
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Human Genome Research Institute (NHGRI) )

Tracking Information
First Submitted Date September 25, 2013
First Posted Date September 30, 2013
Last Update Posted Date February 7, 2019
Actual Study Start Date September 30, 2013
Estimated Primary Completion Date May 1, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: November 3, 2018)
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.
Original Primary Outcome Measures
 (submitted: September 25, 2013)
We will add NHGRI patients enrolled in 00-HG-0209 into a national registry of individuals with primary immune deficiency disorders to discover basic outcome data, ethnic and racial characteristics, kinds of complications etc. of these immune def... [ Time Frame: 8 years ]
Change History Complete list of historical versions of study NCT01953016 on ClinicalTrials.gov Archive Site
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 Participation in a Research Registry for Immune Disorders
Official Title NIH Participation to USIDNET Registry
Brief Summary

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
Detailed Description

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
Study Design Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Individuals of all ages, gender, and races with an immunodeficiency disorder from NIH studies, will be accepted for registration.@@@
Condition
  • Primary Immunodeficiencies
  • APECED
  • CGD (Chronic Granulomatous Disease)
  • Wiskott- Aldrich Syndrome
  • SCID
Intervention Not Provided
Study Groups/Cohorts immunodeficiency
Individual of all ages, gender, and races with an immunodeficiency disorder from NIH studies, will be accepted for registration.
Publications * Ruffner MA; USIDNET Body Weight Group, Sullivan KE. Complications Associated with Underweight Primary Immunodeficiency Patients: Prevalence and Associations Within the USIDNET Registry. J Clin Immunol. 2018 Apr;38(3):283-293. doi: 10.1007/s10875-018-0492-0. Epub 2018 Apr 4.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: August 24, 2017)
1000
Original Estimated Enrollment
 (submitted: September 25, 2013)
600
Estimated Study Completion Date May 1, 2020
Estimated Primary Completion Date May 1, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria
  • 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.

Sex/Gender
Sexes Eligible for Study: All
Ages up to 150 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Elizabeth K Garabedian, R.N. (301) 435-2443 garabede@mail.nih.gov
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT01953016
Other Study ID Numbers 130199
13-HG-0199
Has Data Monitoring Committee Not Provided
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
Responsible Party National Institutes of Health Clinical Center (CC) ( National Human Genome Research Institute (NHGRI) )
Study Sponsor National Human Genome Research Institute (NHGRI)
Collaborators
  • National Institute of Allergy and Infectious Diseases (NIAID)
  • National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Investigators
Principal Investigator: Elizabeth K Garabedian, R.N. National Human Genome Research Institute (NHGRI)
PRS Account National Institutes of Health Clinical Center (CC)
Verification Date February 5, 2019