South-seq: Deoxyribonucleic Acid (DNA) Sequencing for Newborn Nurseries in the South

NCT ID: NCT03842995

Last Updated: 2024-12-13

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

477 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-04-15

Study Completion Date

2023-12-31

Brief Summary

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2,000 infants with signs suggestive of a genetic disorder being treated at a neonatal intensive care unit (NICU) in which African-American and rural populations are highly represented will be enrolled. Whole genome sequencing (WGS) will be used to identify pathogenic variation in DNA from these infants. Stakeholders, including parents, clinicians, and community leaders, will be engaged to develop culturally adapted educational materials and to equip non-genetics providers to return WGS results. Parents will be provided with these materials through a web portal, the Genome Gateway, and will be placed into one of two arms of a randomized trial to compare the effectiveness technology-assisted WGS result delivery by non-genetics providers relative to result delivery from genetic counselors.

Detailed Description

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Barriers to widespread and routine implementation of WGS-enabled clinical care exist at several levels. Surveys of clinicians indicate discomfort in their understanding of genomics and ability to communicate results to patients, and also concern about the time required to do so. Medical geneticists and genetic counselors are disproportionately concentrated in large academic centers, and their numbers are inadequate to support the number of patients that may benefit from WGS. This limitation will have a disproportionate effect on patients in rural and/or medically underserved areas. For example, all but one of the genetic counselors in Alabama are based in Birmingham or Huntsville (lone exception is in Mobile), which means that the southern 2/3 of the state, including major rural underserved areas, have little to no local access to genetic counseling services.

These barriers are especially apparent in neonatal care. For parents of sick neonates, their first interactions with the healthcare system take place in the NICU. Neonatology training traditionally emphasizes critical care and can neglect communication, with one study reporting that 93% of fellows stated that their training in this area should be improved. There is a particular lack of training in genomic neonatal medicine, with few didactic lectures, role play sessions, simulated experiences, or hands on training in clinically relevant scenarios. When infants are diagnosed with congenital anomalies in utero, prenatal consultation with subspecialists can be confusing for genetic conditions with a spectrum of causes and outcomes, and inconsistent information given by different providers, e.g., the neonatologist and the pediatric surgeon.

A central premise underlying the proposal is that non-genetics health care providers, including those outside of academic medical centers, can be empowered to use WGS-testing in their practices. There is ample precedent for implementation of complex technology in primary care: pediatricians, internists, and family practitioners routinely use advanced imaging technologies without a deep understanding of the underlying technology. Bringing WGS-enabled genomic medicine to community health care providers requires, at the least, straightforward criteria to identify patients who may benefit, a user-friendly consent process, clearly worded laboratory reports, easily accessible patient education materials, ready access to support from medical geneticists and genetic counselors, and basic training in how WGS can be applied routinely. The study investigators seek to demonstrate that, if these factors are provided, WGS can be carried out and relevant results returned by newborn medicine providers, and that the patient experience will be at least equal to that achieved with the traditional approach of face-to-face counseling by a geneticist or genetic counselor.

In order to compare technology-assisted WGS result delivery by trained healthcare providers to formal genetic counseling by genetic counselors (standard of care), a series of surveys have been developed and will be completed online using the Genome Gateway platform/website developed for this trial. The survey time points are enrollment (specimen collection from the infant/proband), return of results (ROR) (roughly 2-3 months post-enrollment when WGS results are available), 1-month post-ROR counselling, 4-months post-ROR counselling, and 4.5 months post-ROR counselling.

Conditions

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Whole Genome Sequencing

Keywords

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Genetic Counseling NICU

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

HEALTH_SERVICES_RESEARCH

Blinding Strategy

NONE

Study Groups

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Genetic Counselor

Standard of Care. Parents/caregivers of neonates enrolled in SouthSeq will receive counseling on their child's Whole Genome Sequencing (WGS) results from Genetic Counselors

Group Type PLACEBO_COMPARATOR

Genetic Counselor

Intervention Type BEHAVIORAL

Standard of Care

Trained Healthcare Provider

Healthcare providers (e.g., neonatologists and neonatology nurse practitioners) will receive training to competently deliver Whole Genome Sequencing results to parents/caregivers of neonates enrolled in SouthSeq

Group Type EXPERIMENTAL

Trained Healthcare Provider

Intervention Type BEHAVIORAL

Neonatologists and Neonatology Nurse Practitioners that receive training to deliver whole genome sequencing results

Interventions

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Genetic Counselor

Standard of Care

Intervention Type BEHAVIORAL

Trained Healthcare Provider

Neonatologists and Neonatology Nurse Practitioners that receive training to deliver whole genome sequencing results

Intervention Type BEHAVIORAL

Eligibility Criteria

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Inclusion Criteria

* Parent or caregiver/guardian is willing to participate and answer surveys

Exclusion Criteria

* Proband has secondary findings from WGS
* Parent or caregiver is not available to participate and answer surveys
* Parent or caregiver requires language interpreter services/translated materials
Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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University of Mississippi Medical Center

OTHER

Sponsor Role collaborator

HudsonAlpha Institute for Biotechnology

OTHER

Sponsor Role collaborator

Woman's Hospital, Louisiana

OTHER

Sponsor Role collaborator

Children's Hospital New Orleans, LA

OTHER

Sponsor Role collaborator

Norton Children's Hospital

OTHER

Sponsor Role collaborator

University of Louisville

OTHER

Sponsor Role collaborator

University of Alabama at Birmingham

OTHER

Sponsor Role lead

Responsible Party

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Maria Danila, MD, MSc, MSPH

Principle Investigator

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Maria Danila, MD, MSc,MSPH

Role: STUDY_DIRECTOR

University of Alabama at Birmingham

Locations

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University of Alabama at Birmingham/Children's of Alabama

Birmingham, Alabama, United States

Site Status

Woman's Hospital

Baton Rouge, Louisiana, United States

Site Status

University of Mississippi Medical Center

Jackson, Mississippi, United States

Site Status

Countries

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United States

References

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Biesecker BB, Woolford SW, Klein WMP, Brothers KB, Umstead KL, Lewis KL, Biesecker LG, Han PKJ. PUGS: A novel scale to assess perceptions of uncertainties in genome sequencing. Clin Genet. 2017 Aug;92(2):172-179. doi: 10.1111/cge.12949. Epub 2017 Jan 30.

Reference Type BACKGROUND
PMID: 27925165 (View on PubMed)

Costal Tirado A, McDermott AM, Thomas C, Ferrick D, Harris J, Edwards A, McAllister M. Using Patient-Reported Outcome Measures for Quality Improvement in Clinical Genetics: an Exploratory Study. J Genet Couns. 2017 Oct;26(5):1017-1028. doi: 10.1007/s10897-017-0079-6. Epub 2017 Mar 9.

Reference Type BACKGROUND
PMID: 28281044 (View on PubMed)

Provided Documents

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Document Type: Study Protocol, Statistical Analysis Plan, and Informed Consent Form

View Document

Other Identifiers

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U01HG007301

Identifier Type: NIH

Identifier Source: secondary_id

View Link

IRB-300000328

Identifier Type: -

Identifier Source: org_study_id