Onasemnogene Abeparvovec-brve for Spinal Muscular Atrophy
What You Need to Know Before You Apply
What is the purpose of this trial?
The primary purpose is to address critical evidence in the treatment landscape for Spinal Muscular Atrophy (SMA), specifically focusing on the intrathecal formulation of onasemnogene abeparvovec-brve (ITVISMA®). U.S. Pragmatic Multicenter Study (STREAM).
Are You a Good Fit for This Trial?
This trial is for people aged 2 and older with a confirmed genetic diagnosis of spinal muscular atrophy (SMA). Participants can be children, teens, or adults who are either able to walk on their own or need help walking. They must have at least one year of medical records showing their motor function.Inclusion Criteria
What Are the Treatments Tested in This Trial?
Interventions
- Onasemnogene Abeparvovec-brve
Trial Overview
The study looks at the real-world safety and effectiveness of a single dose of intrathecal onasemnogene abeparvovec-brve (ITVISMA®) in treating SMA across multiple centers in the U.S.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Participants with spinal muscular atrophy, including: * independently ambulatory children/adolescents (≥ 2 years to \< 18 years); * independently ambulatory adults (≥ 18 years); * Non-ambulatory adults (≥ 18 years)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Novartis Pharmaceuticals
Lead Sponsor
Dr. Vas Narasimhan
Novartis Pharmaceuticals
Chief Executive Officer since 2018
MD from Harvard Medical School
Dr. Shreeram Aradhye
Novartis Pharmaceuticals
Chief Medical Officer since 2021
MD
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