Current Environment:

Summary

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.

Conditions

Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA

Recruitment Status

Enrolling by invitation

Eligibility Criteria

Inclusion Criteria:

Any participant with SMA who received onasemnogene abeparvovec-xioi gene replacement therapy in an Novartis Gene Therapies, Inc. sponsored clinical study
Participant/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule

Exclusion Criteria:

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

Intervention

Intervention Type

Intervention Name

Biological

Onasemnogene Abeparvovec-xioi

Phase

Phase 4

Gender

All

Min Age

N/A

Max Age

N/A

Download Date

June 9, 2021

Principal Investigator

N/A

Primary Contact Information

For more information on this trial, visit clinicaltrials.gov.

Contact

For more information and to contact the study team: