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NCT03554343 Clinical Trial

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

Spinal Muscular Atrophy Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data
NCT number NCT03554343
Other study ID # B412201734396
Secondary ID
Status Completed
Phase
First received
Last updated
Start date March 5, 2018
Est. completion date February 28, 2021

Study information
Verified date March 2021
Source Centre Hospitalier Régional de la Citadelle
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

Medico-economic study of Newborn screening of Spinal Muscular Atrophy

Description:

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.

Recruitment information / eligibility
Status Completed
Enrollment 136339
Est. completion date February 28, 2021
Est. primary completion date February 28, 2021
Accepts healthy volunteers No
Gender All
Age group N/A to 7 Days
Eligibility Inclusion Criteria: - All newborns in southern Belgium Exclusion Criteria: - Newborns whose parents refuse screening

Study Design

Related Conditions & MeSH terms

Intervention

Diagnostic Test:
• test for SMN1 exon 7 deletion
Newborns are screened for SMN1 exon 7 deletion through standard NBS practice Positive case are promptly referred to reference centers

Locations
Country Name City State
Belgium CRMN, Hôpital La Citadelle Liege Wallonia

Sponsors (5)
Lead Sponsor Collaborator
Centre Hospitalier Régional de la Citadelle Association Belge contre les Maladies neuro Musculaires, AveXis, Inc., Biogen, Roche Pharma AG

Country where clinical trial is conducted

Belgium, 

References & Publications (1)

Boemer F, Caberg JH, Dideberg V, Dardenne D, Bours V, Hiligsmann M, Dangouloff T, Servais L. Newborn screening for SMA in Southern Belgium. Neuromuscul Disord. 2019 May;29(5):343-349. doi: 10.1016/j.nmd.2019.02.003. Epub 2019 Feb 15. — View Citation

Outcome
Type Measure Description Time frame Safety issue
Primary Cases detected Number of cases detected, false negative and false positive cases march 2018-March 2021
Secondary Incidence of SMA to gather epidemiological data on SMA in Wallonia-Brussels Federation (carrier frequency, number of cases/year). March 2018-March 2021
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