Long-term Follow-up with Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
Trial Summary
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
What data supports the effectiveness of the treatment Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy?
Research shows that Onasemnogene Abeparvovec-xioi, a gene replacement therapy, has been effective in improving outcomes for infants with spinal muscular atrophy, particularly those under two years old. Studies highlight its long-term safety and positive impact on patient and caregiver experiences.12345
Is Onasemnogene Abeparvovec-xioi safe for humans?
Onasemnogene Abeparvovec-xioi has been studied for safety in infants with spinal muscular atrophy, showing it is generally safe, but like any treatment, it may have side effects. It is the only approved gene-replacement therapy for this condition, and ongoing studies continue to monitor its long-term safety.12346
How is the drug Onasemnogene Abeparvovec-xioi unique for treating spinal muscular atrophy?
Onasemnogene Abeparvovec-xioi is unique because it is a gene replacement therapy that delivers a fully functional copy of the survival motor neuron gene, addressing the genetic root cause of spinal muscular atrophy. It is administered as a one-time intravenous infusion, which is different from other treatments that may require ongoing administration.12347
What is the purpose of this trial?
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
Research Team
Sitra Tauscher-Wisniewski, MD
Principal Investigator
Novartis Gene Therapies, Inc.
Eligibility Criteria
This trial is for patients with Spinal Muscular Atrophy who previously received the gene therapy onasemnogene abeparvovec-xioi in a Novartis-sponsored study. Participants or their guardians must consent to and follow the study's procedures.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants received treatment with IV or IT onasemnogene abeparvovec-xioi in their respective parent study
Long-term Follow-up
Continuous monitoring of safety, efficacy, and durability of response to onasemnogene abeparvovec-xioi treatment
Treatment Details
Interventions
- Onasemnogene Abeparvovec-xioi
Find a Clinic Near You
Who Is Running the Clinical Trial?
Novartis Gene Therapies
Lead Sponsor