Actively Recruiting

Phase Not Applicable
Age: 0 - 2Years
All Genders
Healthy Volunteers
NCT06549218

Genetic Newborn Screening for Rare Diseases Within the Screen4Care Project

Led by University Hospital Freiburg · Updated on 2026-05-04

20000

Participants Needed

8

Research Sites

104 weeks

Total Duration

On this page

Sponsors

U

University Hospital Freiburg

Lead Sponsor

I

Innovative Medicines Initiative

Collaborating Sponsor

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

Genetic Newborn Screening for Rare Diseases Within the Screen4Care Project

Who Can Participate

Age: 0 - 2Years
All Genders
Healthy Volunteers

Eligibility Criteria

Eligible

You may qualify if you...

  • Newborns born in one of the participating hospitals and birth centres
  • Informed consent signed by both parents or legal guardians to participate in genetic newborn screening (TREAT-panel)
  • Participation in the TREAT-panel study for whole genome sequencing
  • Symptoms suggestive of a genetic disease within the first 2 years of life for whole genome sequencing
  • Informed consent signed by both parents or legal guardians to participate in both genetic newborn screening (TREAT-panel) and whole genome sequencing
Not Eligible

You will not qualify if you...

  • Missing informed consent of parents or legal guardians

AI-Screening

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Trial Site Locations

Total: 8 locations

1

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

Dijon, France, 21079

Completed

2

Charité University Medicine Berlin

Berlin, Germany, 13353

Actively Recruiting

3

Clinic for Neuropediatrics and Muscular Diseases, Freiburg University Medical Center

Freiburg im Breisgau, Germany, 79106

Actively Recruiting

4

University Medical Center Göttingen, Clinic for Neurology

Göttingen, Germany, 37075

Actively Recruiting

5

Ospedale Pediatrivo Bambino Gesu IRCCS

Rome, Lazio, Italy, 00165

Actively Recruiting

6

Unit Medical Genetics, Azienda Ospedaliero-Universitaria Sant'Anna

Ferrara, Italy, 44122

Completed

7

Azienda Ospedaliero Universitaria di Modena, Neonatology Unit

Modena, Italy, 41100

Actively Recruiting

8

San Pietro Fatebenefratelli Hospital

Roma, Italy, 00189

Actively Recruiting

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Research Team

A

Alessandra Ferlini, Professor

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

SCREENING

Number of Arms

1

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