Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion - Join Clinical Trial NCTNCT03306277
How to Join This Clinical Trial - NCTNCT03306277
Learn how to participate in this PHASE3 trial studying Onasemnogene abeparvovec-xioi for Spinal muscular atrophy, SMA Type 1. Current status: COMPLETED.
Am I Eligible for This Clinical Trial?
This clinical research study is looking for participants with Spinal muscular atrophy, SMA Type 1. Eligibility requirements include specific age ranges, health status, and medical history. Contact the study team to learn if you qualify.
- Condition Being Studied
- Spinal muscular atrophy, SMA Type 1
- Treatment Being Tested
- Onasemnogene abeparvovec-xioi
- Study Phase
- PHASE3 - Large-scale efficacy study
- Enrollment Status
- COMPLETED
- Study Identifier
- NCTNCT03306277 - ClinicalTrials.gov Identifier
- Sponsored By
- Study sponsor
What to Expect as a Participant
Participating in this clinical trial involves:
- Initial screening to determine eligibility
- Regular study visits and health assessments
- Receiving the study treatment or placebo
- Medical monitoring and follow-up care
- Contributing to medical research that may help others
- Potential access to new treatments before they're widely available
Clinical Trial Benefits and Compensation
Participants in this clinical research study may receive:
- Close medical monitoring by healthcare professionals
- Access to potential new treatments
- Compensation for time and travel (varies by study)
- No-cost study-related medical care
- The opportunity to help advance medical knowledge
Where Is This Clinical Trial Located?
This study is enrolling participants at multiple locations. Find a study site near you:
How to Enroll in This Study
To learn more about participating in this PHASE3 clinical trial for Spinal muscular atrophy, SMA Type 1:
- Review the eligibility criteria with your healthcare provider
- Contact the study team for a pre-screening interview
- Schedule an in-person screening visit if eligible
- Review and sign the informed consent form
- Begin participation in the clinical trial
Why Choose Quri.ai as Your Clinical Trial Matching Service
Quri.ai is a free clinical trial matching service that helps patients connect with research sites. We make it easier to:
- Find clinical trials that match your condition
- Get matched to the right study location near you
- Connect directly with study coordinators
- Understand if you qualify with simplified eligibility screening
- Navigate the enrollment process with personalized support
- Access new treatments through clinical research participation