Summary

Location
at UCLA
Dates
study started
estimated completion

Description

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.

Official Title

A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101

Keywords

Spinal Muscular Atrophy Type I Spinal Muscular Atrophy Type II Spinal Muscular Atrophy Type III SMA Gene replacement Muscular Atrophy Muscular Atrophy, Spinal Spinal Muscular Atrophies of Childhood Atrophy Onasemnogene Abeparvovec-xioi Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec-xioi

Eligibility

You can join if…

  • 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

You CAN'T join if...

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

Locations

  • David Geffen School of Medicine at UCLA
    Los Angeles California 90095 United States
  • Stanford University Medical Center
    Palo Alto California 94304 United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Novartis Gene Therapies
ID
NCT04042025
Phase
Phase 4
Study Type
Interventional
Last Updated