Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Recruiting in Palo Alto (17 mi)
+105 other locations
Age: Any Age
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Academic
Recruiting
Sponsor: Novartis Pharmaceuticals
No Placebo Group
Trial Summary
What is the purpose of this trial?Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an autosomal recessive, early childhood disease with an incidence of 1:10,000 live births. SMA is the leading cause of infant mortality due to genetic diseases.
The purpose of this registry is to assess the long term outcomes of patients with SMA in the context of advances in treatment options and also to characterize and assess long-term safety and effectiveness of OAV-101.
Eligibility Criteria
Inclusion Criteria
Patients with SMA, genetically confirmed on or after 24 May 2018.
Appropriate consent/assent has been obtained for participation in the registry
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Prospective observational registryExperimental Treatment2 Interventions
This is a prospective, multi center, multinational, non-interventional observational registry.
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
University of California Davis Health SystemSacramento, CA
CHRISTUS HealthSan Antonio, TX
Penn State HersheyHershey, PA
Yale-New Haven Health SystemNew Haven, CT
More Trial Locations
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Who Is Running the Clinical Trial?
Novartis PharmaceuticalsLead Sponsor
AveXis, Inc.Lead Sponsor
United BioSource, LLCIndustry Sponsor