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Completed

NCT Number: NCT03554343

Sun May Arise on SMA : Newborn Screening of Spinal Muscular Atrophy in Belgium

Medico-economic study of Newborn screening of Spinal Muscular Atrophy

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Key information

Conditions

Spinal Muscular Atrophy Central Nervous System Diseases Motor Neuron Disease Muscular Atrophy, Spinal Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases

Age range

Up to 7 day

Sex eligibility

All sexes

Study type

Observational

Primary location

CRMN, Hôpital La Citadelle

Liège, Wallonia, 4000, Belgium

About this study

The aim of this project is to demonstrate the feasibility and the medico-economic impact of a spinal muscular atrophy neonatal screening in a European country. We propose a 3-year-neonatal screening program in Southern Belgium (Wallonia-Brussels Federation) where there is 59.000 newborns/year, among whom 6 are affected by spinal muscular atrophy.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • All newborns in southern Belgium

Exclusion criteria

  • Newborns whose parents refuse screening

Treatment and study plan

test for SMN1 exon 7 deletion

Diagnostic Test

Newborns are screened for SMN1 exon 7 deletion through standard NBS practice Positive case are promptly referred to reference centers

Primary outcomes

  1. Cases detected

    Time frame: march 2018-March 2021

    Number of cases detected, false negative and false positive cases

Secondary outcomes

  1. Incidence of SMA

    Time frame: March 2018-March 2021

    to gather epidemiological data on SMA in Wallonia-Brussels Federation (carrier frequency, number of cases/year).

Sponsors and collaborators

Lead sponsor

Centre Hospitalier Régional de la Citadelle

Other

Collaborators

  • Association Belge contre les Maladies neuro Musculaires
  • AveXis, Inc.
  • Biogen
  • Roche Pharma AG

Registry information

Official study title

Sun May Arise on SMA : Newborn Screening of SMA in Belgium

Important dates

Study start
2018
Primary completion
2021
Study completion
2021
First posted
Jun 13, 2018
Registry last updated
Mar 10, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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