Implementation of Whole Genome Sequencing as Screening in a Diverse Cohort of Healthy Infants

NCT ID: NCT05161169

Last Updated: 2025-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

500 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-12-21

Study Completion Date

2025-06-30

Brief Summary

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This research study is exploring the use of genomic sequencing in the newborn period to screen healthy babies for current and future health risks. The study will enroll a diverse cohort of 500 healthy infants and their parents from Boston, MA; New York City, NY; and Birmingham, AL. A small blood sample will be collected from each infant, and whole genome sequencing will be performed in 1/2 of the cohort following a randomized controlled trial design. 3 months later, the randomization status and sequencing results will be shared with parents and pediatricians. Investigators will study the medical, behavioral, and economic outcomes of genomic sequencing to better understand how this technology can be implemented in outpatient primary care settings.

Detailed Description

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The objective of this research protocol is to assess the impacts of genomic sequencing in healthy infants from ethnically and racially diverse communities as part of routine pediatric care.

Investigators will enroll a cohort of 500 healthy, ethnically and racially diverse infants from Boston, Massachusetts; New York City, New York; and Birmingham, Alabama, with planned expansion to other U.S. cities and recruitment sites. As part of this study, a stakeholder board comprised of diverse community members will provide early and regular feedback throughout the study on anticipated and ongoing community reaction to the work with sensitivity to historical injustices and cultural diversity

Primary care pediatricians from each recruitment site will be enrolled for a brief genomics education curriculum. Only infants whose healthcare providers have joined the study will be enrolled.

A small blood sample will be obtained from each enrolled infant. Participants will randomized (1:1) to receive either a family history report or a family history report plus whole genome sequencing.

Genome sequencing data will be analyzed for pathogenic and likely pathogenic variants in genes associated with childhood-onset disease risks, as well as highly actionable adult-onset disease risks. If infants have a dominant risk identified, parents may choose to be screened as part of the study.

The study team will disclose the infant's randomization status and study results during a consultation with each family, and results will be sent to the infant's pediatrician.

Parents will be surveyed at three time points over the 12 months after enrollment: baseline, immediately post-disclosure (approximately 3 months after enrollment), and 6 months post-disclosure. Surveys will assess psychosocial impacts of newborn sequencing.

Chart reviews will be performed to assess the medical outcomes and healthcare utilization costs of newborn genome sequencing.

Conditions

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Genetic Predisposition to Disease Hereditary Diseases

Keywords

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genome sequencing newborn screening preventive medicine

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Randomized controlled trial (control group vs. genome sequencing intervention)
Primary Study Purpose

SCREENING

Blinding Strategy

DOUBLE

Participants Caregivers

Study Groups

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Sequencing cohort

Infants receive genome sequencing with analysis of approximately 1000 genes associated with childhood-onset and highly actionable adult-onset disease risks. Pathogenic and likely pathogenic variants are reported to the child's parents and pediatrician. Participants also receive a detailed family history report and standard well-child care.

Group Type EXPERIMENTAL

Genome Sequencing

Intervention Type GENETIC

20 times read depth (20x) next-generation whole genome sequencing with comprehensive analysis.

Control cohort

Infants receive a detailed family history report plus standard well-child care.

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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Genome Sequencing

20 times read depth (20x) next-generation whole genome sequencing with comprehensive analysis.

Intervention Type GENETIC

Eligibility Criteria

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Inclusion Criteria

Infant participants

* Has not previously had exome or genome sequencing
* Age 0-12 months
* Seen for well-baby pediatric care at a recruiting site
* Primary healthcare provider completed the genomics education program
* At least one parent or guardian able to participate in the study

Parent participants

* Biological parent or legal guardian of an infant participating in the study
* 18 years of age or older
* Unimpaired decision-making capacity
* English or Spanish speaking
* Available to have genetic counseling and provide consent for testing the infant

Exclusion Criteria

* Parents are unwilling to have genomic reports placed in the medical record or sent to their primary care pediatrician
* Any infant in which clinical considerations preclude collecting blood via heel stick
Minimum Eligible Age

0 Months

Maximum Eligible Age

12 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Baylor College of Medicine

OTHER

Sponsor Role collaborator

Boston Children's Hospital

OTHER

Sponsor Role collaborator

Broad Institute of MIT and Harvard

OTHER

Sponsor Role collaborator

Dartmouth-Hitchcock Medical Center

OTHER

Sponsor Role collaborator

Harvard Pilgrim Health Care

OTHER

Sponsor Role collaborator

Howard University

OTHER

Sponsor Role collaborator

HudsonAlpha Institute for Biotechnology

OTHER

Sponsor Role collaborator

Massachusetts General Hospital

OTHER

Sponsor Role collaborator

Icahn School of Medicine at Mount Sinai

OTHER

Sponsor Role collaborator

University of Alabama at Birmingham

OTHER

Sponsor Role collaborator

National Center for Advancing Translational Sciences (NCATS)

NIH

Sponsor Role collaborator

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

NIH

Sponsor Role collaborator

Brigham and Women's Hospital

OTHER

Sponsor Role lead

Responsible Party

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Robert C. Green, MD, MPH

Professor of Medicine (Genetics)

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Robert C. Green, MD, MPH

Role: PRINCIPAL_INVESTIGATOR

Brigham and Women's Hospital

Ingrid A. Holm, MD, MPH

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital

Locations

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Beaumont - Corewell Health East

Royal Oak, Michigan, United States

Site Status

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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The BabySeq2 Project

Identifier Type: -

Identifier Source: org_study_id