Zolgensma Intrathecal (onasemnogene abeparvovec-xioi intrathecal) / Novartis 
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5 Trials

   

Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Zolgensma Intrathecal (onasemnogene abeparvovec-xioi intrathecal) / Novartis
2021-006709-31: Phase IIIb, open-label, multi-center study to evaluate safety, tolerability and efficacy study of OAV101 administered intrathecally to participants with spinal muscular atrophy (SMA) who have discontinued treatment with nusinersen or risdiplam

Ongoing
3
28
Europe
OAV101, OAV101, Solution for injection, Zolgensma
Novartis Pharma AG, Novartis Pharma AG
Spinal Muscular Atrophy, Spinal Muscular Atrophy, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
2021-003474-31: Safety and efficacy of Intrathecal OAV101 in Pediatric Patients with Spinal Muscular Atrophy (SMA) (STEER)

Not yet recruiting
3
125
Europe
OAV101, Okrido, prednisolone, OAV101, Solution for injection, Oral solution, Zolgensma, Okrido 6mg/mL oral solution, Prednisolone Sodium Phosphate Oral Solution
Novartis Pharma AG, Novartis Pharma AG
Spinal Muscular Atrophy, Spinal Muscular Atrophy, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
STRENGTH, NCT05386680: Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

Completed
3
27
Europe, Canada, Japan, US, RoW
OAV101, AVXS-101, Zolgensma
Novartis Pharmaceuticals
Spinal Muscular Atrophy
11/24
11/24
STEER, NCT05089656: Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

Active, not recruiting
3
127
Europe, US, RoW
OAV101, Zolgensma, AVXS-101, Sham control
Novartis Pharmaceuticals
Type 2 Spinal Muscular Atrophy
11/24
02/25
NCT04042025 / 2019-002611-26: Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

Active, not recruiting
3
85
Europe, Canada, Japan, US, RoW
Onasemnogene Abeparvovec-xioi, Zolgensma
Novartis Gene Therapies
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
12/35
12/35

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