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

Novartis

Status and phase: Active, not recruiting; Phase 3

Conditions: Spinal Muscular Atrophy Type III; Spinal Muscular Atrophy Type I; SMA; Spinal Muscular Atrophy Type II

Treatments: Biological: Onasemnogene Abeparvovec-xioi

Study type: Interventional

Funder types: Industry

Identifiers

NCT04042025

COAV101A12103 (Other Identifier)

205305 (Other Identifier)

2019-002611-26 (EudraCT Number)

AVXS-101-LT-002

Details and patient eligibility

About

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.

Enrollment: 85 patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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85 participants in 1 patient group

Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec-xioi

Other group

Description:

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

Treatment:

Biological: Onasemnogene Abeparvovec-xioi