Long-term Follow-up with Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

Recruiting at 31 trial locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Novartis Gene Therapies

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

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

I have SMA and was treated with onasemnogene abeparvovec-xioi in a Novartis study.

Participant/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule

I have SMA and was treated with onasemnogene abeparvovec-xioi in a Novartis study.

Exclusion Criteria

Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study

Treatment Details

Interventions

• Onasemnogene Abeparvovec-xioi (Virus Therapy)

Trial Overview The long-term safety and effectiveness of onasemnogene abeparvovec-xioi, a gene replacement therapy for SMA, are being monitored as participants transition from earlier trials for ongoing evaluation.

Participant Groups

Group I: Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec-xioi Experimental Treatment 1 Intervention

Participants received treatment with IV onasemnogene abeparvovec-xioi in an onasemnogene abeparvovec-xioi or received treatment with IT onasemnogene abeparvovec-xioi in an onasemnogene.

Who Is Running the Clinical Trial?

Novartis Gene Therapies

Lead Sponsor

Trials

9

Recruited

45,200+