MedPath

NIAID Centralized Sequencing Protocol

Recruiting
Conditions
Primary Immunodeficiency
Atopy
Autoimmunity
Autoinflammation
Registration Number
NCT03206099
Lead Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Brief Summary

Background:

Genetic testing called "sequencing" helps researchers look at DNA. Genes are made of DNA and are the instructions for our bodies to function. We all have thousands of genes. DNA variants are differences in genes between two people. We all have lots of variants. Most are harmless and some cause differences like blue or brown eyes. A few variants can cause health problems.

Objective:

To understand the genetics of immune disorders various health conditions, as well as outcomes of clinical genomics and genetic counseling services performed under this protocol.

Eligibility:

Participants in other NIH human subjects research protocols - either at the NIH Clinical Center (CC) or at Children s National Health System (CNHS) - (aged 0-99 years), and, in select cases, their biological relatives

Design:

Researchers will study participant s DNA extracted from blood, saliva, or another tissue sample, including previously collected samples we may have stored at the NIH. Researchers will look at participant s DNA in great detail. We are looking for differences in the DNA sequence or structure between participants and other people.

Participants will receive results that:

* Are important to their health

* Have been confirmed in a clinical lab

* Suggest that they could be at risk for serious disease that may affect your current or future medical management.

Some genetic information we return to participants may be of uncertain importance.

If genetic test results are unrelated to the participant s NIH evaluations, then we will not typically report:

* Normal variants

* Information about progressive, fatal conditions that have no effective treatment

* Carrier status (conditions you don t have but could pass on)

The samples and data will be saved for future research.

Personal data will be kept as private as possible.

If future studies need new information, participants may be contacted.

Detailed Description

Investigators at the National Institute of Allergy and Infectious Diseases (NIAID) use next-generation sequencing technologies to help determine genetic contributions to immune diseases. These efforts have increased rates of molecular diagnosis for a subset of NIAID

participants as well as uncovered fundamental insights into the cellular and signaling pathways in host defense and immune regulation.

Despite these successes, analysis and interpretation of genomic data remain a substantial challenge. Simply, researchers do not understand the functional and clinical consequences of most human genetic variation. This is true at NIAID and across the intramural research program. Making progress in this area requires a coordinated, systematic, and transparent approach to clinical genomics research.

This protocol is specific to genetic testing and explicitly aims to both strengthen clinical care and enhance research throughout participating programs at the NIH. Probands will provide biological specimens for genetic testing and will be required to be enrolled on a primary protocol, which will execute the primary clinical and research evaluations. This protocol serves as a vehicle for a

programmatic effort that includes standardized phenotyping, test ordering through the Clinical Research Information System (CRIS), sample collection and isolation, nucleic acid analysis, bioinformatics, clinical interpretation, reporting in CRIS, genetic counseling, and supporting effective use of genomics as a research tool throughout the intramural program. Genetic testing results and data (upon request) will be shared with the research teams for protocols on which a given participant is co-enrolled. Overall, increased process standardization will support data integrity and efficiency while still accommodating the need for investigator flexibility.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
20000
Inclusion Criteria

Not provided

Read More
Exclusion Criteria

Not provided

Read More

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Identifying novel genetic defects associated with immune disordersUpon analysis of genomic data

Identifying novel genetic defects associated with immune disorders

Identifying novel clinical phenotypes associated with established genetic defectsUpon analysis of genomic data

Identifying novel clinical phenotypes associated with established genetic defects

Identifying established genetic disorders of the immune systemUpon analysis of genomic data

Identifying established genetic disorders of the immune system, as well as known genetic disorders outside of the immune system in some cases

Secondary Outcome Measures
NameTimeMethod
Evidence base for how to improve clinical genomic services on this protocol and related programs.5.1.1. Enrollment/Baseline Report Comprehension Survey and Semi-Structured Phone Interviews

Studies of the processes and outcomes of the clinical genomics and genetic counseling services performed under this protocol. These studies will use surveys, interviews, and other social and behavioral research methods to collect data from study participants about their perceptions, experiences, and attitudes related to their condition and participation in this protocol. The goal of these additional studies will be to improve the services provided under protocol 17-I-0122 and to generate an evidence base for other investigators conducting similar studies.

Trial Locations

Locations (2)

National Institutes of Health Clinical Center

🇺🇸

Bethesda, Maryland, United States

Children's National Health System

🇺🇸

Washington, District of Columbia, United States

© Copyright 2025. All Rights Reserved by MedPath