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Onasemnogene abeparvovec-xioi (Zolgensma) intrathecal in sitting non-ambulatory SMA patients
NCT03381729Phase 1Terminated
- Sponsor
- Novartis
- Start
- Dec 2017
- Primary completion
- Nov 2021
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 1
01
Overview
Phase 1, open-label trial of onasemnogene abeparvovec in spinal muscular atrophy (SMA) (3 arms), sponsored by Novartis. Primary endpoints: Number of Participants Who Achieved the Ability to Stand Alone (Age 6 to <24 Months Only) at 12 months, Change From Baseline in Hammersmith Functional Motor Scale-Expanded Score at Month 12 (Age 24 to <60 Months Only) and 1 more. Started Dec 2017; status: terminated.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
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