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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

ACTIVE_NOT_RECRUITING

Eligibility Criteria

Inclusion Criteria:

* Any participant with SMA who received onasemnogene abeparvovec-xioi gene replacement therapy in a Novartis Gene Therapies-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

PHASE3

Gender

ALL

Min Age

N/A

Max Age

N/A

Download Date

2024-02-22

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:

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi NCT04042025