Sun May Arise on SMA : Newborn Screening of Spinal Muscular Atrophy in Belgium
NCT ID: NCT03554343
Last Updated: 2021-03-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
136339 participants
OBSERVATIONAL
2018-03-05
2021-02-28
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Natural History Study for Patients With Nemaline Myopathy in Belgium
NCT07201636
Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy
NCT02391831
Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy
NCT06321965
Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
NCT05768048
European Registry of Patients With Infantile-onset Spinal Muscular Atrophy
NCT03339830
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
ECOLOGIC_OR_COMMUNITY
PROSPECTIVE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
All newborn from Southern Belgium
All newborns except newborns for which parents refuse newborn screening will be tested for exon 7 deletion in survival motor neuron 1 (SMN1)
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
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
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
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
Exclusion Criteria
7 Days
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
AveXis, Inc.
INDUSTRY
Biogen
INDUSTRY
Roche Pharma AG
INDUSTRY
Association Belge contre les Maladies neuro Musculaires
UNKNOWN
Centre Hospitalier Régional de la Citadelle
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Laurent Servais
Professor
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Laurent Servais, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
CRMN
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
CRMN, Hôpital La Citadelle
Liège, Wallonia, Belgium
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
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.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
B412201734396
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.