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Long-term Follow-up with Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
Study Summary
This trial is for people with Spinal Muscular Atrophy who have been treated with onasemnogene abeparvovec-xioi in the past. Researchers will monitor safety and efficacy of the treatment long-term.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Side effects data
From 2019 Phase 3 trial • 22 Patients • NCT03306277Trial Design
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Who is running the clinical trial?
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- I have SMA and was treated with onasemnogene abeparvovec-xioi in a Novartis study.I have SMA and was treated with onasemnogene abeparvovec-xioi in a Novartis study.
- Group 1: Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec-xioi
- All Individual Drugs Already Approved - Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- Approved for 5 Other Conditions - This treatment demonstrated efficacy for 5 other conditions.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
To what afflictions is this therapy most often given?
"This medication is most frequently used to treat patients with antibiotic-resistant infections. It has also been found to help patients with spinal muscular atrophy (sma), post-operative recovery, and motor neuron disease."
Could you give me a number for how many different hospitals this research is being conducted in?
"Ann Robert H. Lurie Children's Hospital of Chicago in Chicago, Columbia University Medical Center in New york, and Children's Hospital of Philadelphia in Philadelphia are a few of the 20 locations currently running this trial."
Could you please rank this treatment's level of risk?
"This Phase 3 trial's safety was scored a 3 by our Power team because there is both efficacy and safety data supporting the treatment."
Are we still able to enroll individuals in this experiment?
"As of September 27th, 2022, this study is no longer actively recruiting patients. Spinal muscular atrophy type iii currently has 205 clinical trials enrolling patients and 30 of those trials are actively searching for participants."
Who else is applying?
How old are they?
What site did they apply to?
What portion of applicants met pre-screening criteria?
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