Genomic Uniformed-Screening Against Rare Disease In All Newborns

Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age1-1
SponsorColumbia University

About this trial

The goal of this study is to learn how genomic sequencing technology can be used to effectively expand the conditions screened on newborn screening. Newborn screening ensures equity and allows all babies to have the same chance at the healthiest life. Families will be invited to have their newborn baby screened for additional conditions beyond what all babies are screened for as part of the newborn screening public health program. Families can choose to be part of the study or choose not to be part of the study and just have the routine newborn screening test. Families will also be able to choose to learn about their baby's risk for conditions that have effective treatments available but are not on the routine newborn screening panel or also learn about conditions for which there is not currently FDA approved medications but for which medications are under development or for which early intervention services or treatment of seizures may improve the child's outcome. Families will be invited to the study shortly after the baby is born and will learn the decision not to participate, and we will interview a subset of parents who agree to be interviewed. Newborns who screen positive will be referred to appropriate providers for care and will be followed through review of electronic medical records and parental follow up via phone, text, postal mail or email.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

Newborns admitted to the well-baby nurseries from the recruiting hospitals

Newborns born after 33 weeks of gestation

Newborns whose parents are English, Mandarin, or Spanish speaking

Disqualifiers

None

Trial design

Design model

Single group

Treatments tested in this trial

  • Genome sequencing-based newborn screening

    Other intervention

    Dried blood spots collected at birth for routine newborn screening will be used for genome sequencing based screening of a defined set of conditions.

Treatment groups

100,000 Participants
are divided into 1 treatment group
Group A: Enrolled in the studyExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Enrollment Rate (percentage)

Enrollment rate will be defined as: number of enrolled newborns / number of newborns approached by the research assistant.

Time frame
From study launch to end of enrollment (up to 5 years)
2

Successful Sequencing Rate (percentage)

Successful sequencing rate will be defined as: number of successful sequencing / number of enrolled newborns.

Time frame
Up to 6 months after the end of enrollment
3

Screen Positive Rate (percentage)

Screen positive rate will be defined as: number of newborns with a positive screening / number of successful sequencing.

Time frame
Up to 6 months after the end of enrollment
4

True Positive Rate (percentage)

True positive rate will be defined as: number of confirmed diagnosis / number of screen positive.

Time frame
Up to 6 months after the end of enrollment

Secondary outcomes

1

Score on Decision Regret Scale

The Decision Regret Scale measures distress or remorse after a health care decision. This 5-items scale ranges from 0 (no regrets - best outcome) to 100 (highest regret - worst outcome). Scores above 1 and below 25 are considered as an indication of mild regret. Scores above 25 are considered as an indication of moderate to severe regret.

Time frame
Up to 6 months after the end of enrollment

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Columbia University

Lead sponsor

Illumina, Inc.

Collaborator

GeneDx

Collaborator

New York State Department of Health

Collaborator