Genetic Newborn Screening for Rare Diseases Within the Screen4Care Project

NCT ID: NCT06549218

Last Updated: 2025-07-29

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

RECRUITING

Clinical Phase

NA

Total Enrollment

20000 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-12-03

Study Completion Date

2025-12-31

Brief Summary

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The main objective of the genetic newborn screening part of the Screen4Care-project is to shorten the path to rare disease diagnosis and to facilitate early intervention. Therefore, genetic newborn screening for currently treatable rare diseases (TREAT-panel approach) will be offered to families expecting a baby. Whole genome sequencing (WGS) will be offered as additional diagnostic approach to newborns participating in Screen4Care TREAT-panel approach, if they develop symptoms suggestive of a genetic disease.

To evaluate to what extend genetic newborn screening has an impact on participating infants and their families, a follow-up with standardised questionnaires will be performed for all participating families.

Detailed Description

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Conditions

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Newborn Screening

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

SCREENING

Blinding Strategy

NONE

Study Groups

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newborn screening

All newborns participating in the study will receive a genetic newborn screening for predefined treatable diseases. Newborns participating in the TREAT-panel developing symptoms suggestive of a genetic disease during the first 2 years of life can receive whole genome sequencing.

Group Type OTHER

newborn genetic screening and whole genome sequencing

Intervention Type DIAGNOSTIC_TEST

newborn genetic screening (panel of treatable diseases); whole genome sequencing (if newborn develops symptoms suggestive of a genetic disease)

Interventions

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newborn genetic screening and whole genome sequencing

newborn genetic screening (panel of treatable diseases); whole genome sequencing (if newborn develops symptoms suggestive of a genetic disease)

Intervention Type DIAGNOSTIC_TEST

Eligibility Criteria

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Inclusion Criteria

* TREAT-panel:

* newborns
* Infants born in one of the participating hospitals and birth centres
* Informed consent signed by both parents/legal guardian to participate in genetic newborn screening (TREAT-panel)
* Whole genome sequencing:

* Participation in the TREAT-panel study
* Symptoms suggestive of a genetic disease within the first 2 years of life
* Informed consent signed by both parents/legal guardian to participate in genetic newborn screening (TREAT-panel) and the whole genome sequencing

Exclusion Criteria

* Missing informed consent of parents/legal guardian
Maximum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Innovative Medicines Initiative

OTHER

Sponsor Role collaborator

Università degli Studi di Ferrara

OTHER

Sponsor Role collaborator

Ospedale Pediatrico Bambin Gesù

OTHER

Sponsor Role collaborator

University of Siena

OTHER

Sponsor Role collaborator

Centre Hospitalier Universitaire Dijon

OTHER

Sponsor Role collaborator

Real Genix

UNKNOWN

Sponsor Role collaborator

University Hospital Goettingen

OTHER

Sponsor Role collaborator

Centro Nacional de Análisis Genómico

UNKNOWN

Sponsor Role collaborator

Genoox

UNKNOWN

Sponsor Role collaborator

Schwarzwald-Baar Clinic

UNKNOWN

Sponsor Role collaborator

Municipal Hospital Karlsruhe

UNKNOWN

Sponsor Role collaborator

University Hospital Freiburg

OTHER

Sponsor Role lead

Responsible Party

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Jan Kirschner

Prof. Dr.

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Alessandra Ferlini, Professor

Role: PRINCIPAL_INVESTIGATOR

Unit Medical Genetics, Azienda Ospedaliero-Universitaria Sant'Anna

Locations

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Centre de Génétique et Centre de Référence Anomalies du Développement et Syndromes Malformatifs, Hôpital d'Enfants

Dijon, , France

Site Status RECRUITING

Clinic for Neuropediatrics and Muscular Diseases, Freiburg University Medical Center

Freiburg im Breisgau, , Germany

Site Status RECRUITING

Ospedale Pediatrivo Bambino Gesu IRCCS

Rome, Lazio, Italy

Site Status RECRUITING

Unit Medical Genetics, Azienda Ospedaliero-Universitaria Sant'Anna

Ferrara, , Italy

Site Status RECRUITING

Azienda Ospedaliero Universitaria di Modena, Neonatology Unit

Modena, , Italy

Site Status RECRUITING

San Pietro Fatebenefratelli Hospital

Roma, , Italy

Site Status RECRUITING

Countries

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France Germany Italy

Central Contacts

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Alessandra Ferlini, Professor

Role: CONTACT

+39 0532 974439

Facility Contacts

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Laurence Faivre, Professor

Role: primary

+ 33 380 295 313

Emeline Davoine

Role: backup

Janbernd Kirschner, Professor

Role: primary

+49 761 270-43650

Kathrin Freyler, Dr.

Role: backup

Enrico S Bertini

Role: primary

+39 0668592104

Alessandra Ferlini, Professor

Role: primary

+39 0532 974439

Fernanda Fortunato

Role: backup

Prof. Alberto Berardi

Role: primary

Dr Licia Lugli

Role: backup

Prof. Marco Bonito, UOC Obstetrics and Gynecology

Role: primary

Dott.ssa Maria Eleonora Scapillati, UOC Pediatrics and Neonatology

Role: backup

Other Identifiers

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101034427

Identifier Type: -

Identifier Source: org_study_id

NCT06528548

Identifier Type: -

Identifier Source: nct_alias

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