FindTrial
Sign In

Onasemnogene Abeparvovec-xioi

BIOLOGICAL7 trials

Sponsors

Novartis Gene Therapies

Conditions

Gene TherapySMASMA - Spinal Muscular AtrophySpinal Muscular AtrophySpinal Muscular Atrophy 1Spinal Muscular Atrophy Type ISpinal Muscular Atrophy Type IISpinal Muscular Atrophy Type III

Phase 1

Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
TerminatedNCT03381729
Novartis Gene TherapiesSpinal Muscular Atrophy
Start: 2017-12-14End: 2021-11-18Updated: 2026-01-26

Phase 3

Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
CompletedNCT03306277
Novartis Gene TherapiesGene Therapy, SMA - Spinal Muscular Atrophy
Start: 2017-10-24End: 2019-11-12Updated: 2026-01-26
Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
CompletedNCT03461289
Novartis Gene TherapiesSMA
Start: 2018-08-16End: 2020-09-11Updated: 2026-01-26
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
CompletedNCT03505099
Novartis Gene TherapiesSpinal Muscular Atrophy
Start: 2018-04-02End: 2021-06-15Updated: 2026-01-26
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
CompletedNCT03837184
Novartis Gene TherapiesSpinal Muscular Atrophy Type I
Start: 2019-05-31End: 2021-06-29Updated: 2026-01-26
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Active, not recruitingNCT04042025
Novartis Gene TherapiesSMA, Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II +1
Start: 2020-02-10End: 2035-12-31Updated: 2025-04-25

Unknown Phase

Long-Term Follow-up Study for Patients From AVXS-101-CL-101
Active, not recruitingNCT03421977
Novartis Gene TherapiesSpinal Muscular Atrophy 1
Start: 2017-09-21End: 2030-12-02Updated: 2025-04-25

Related Papers

JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam
Journal of Neurology2024-05-1123 citations
Intravenous and Intrathecal Onasemnogene Abeparvovec Gene Therapy in Symptomatic and Presymptomatic Spinal Muscular Atrophy: Long-Term Follow-Up Study (S34.002)
Neurology2023-04-257 citations
Long-Term Follow-Up of Onasemnogene Abeparvovec Gene Therapy in Patients with Spinal Muscular Atrophy Type 1 (P7-9.006)
Neurology2023-04-256 citations
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)
Journal of Neuromuscular Diseases2023-03-1053 citations
Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.
2023-02-1335 citations
Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial
Nature Medicine2022-06-17250 citations
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.
2022-06-17199 citations
Onasemnogene abeparvovec gene therapy for spinal muscular atrophy type 1: phase 3 study update (str1ve-eu)
Journal of Neurology Neurosurgery & Psychiatry2022-05-2713 citations
Onasemnogene Abeparvovec in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update in Children with Three Copies of SMN2 (P15-5.001)
Neurology2022-05-03
Implications of circulating neurofilaments for spinal muscular atrophy treatment early in life: A case series.
2021-10-3030 citations
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.
2021-10-01182 citations
Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy
JAMA Neurology2021-05-17260 citations
Long-Term Follow-Up (LTFU) of Onasemnogene Abeparvovec Gene Therapy in Spinal Muscular Atrophy (SMA) (2301)
Neurology2021-04-134 citations
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.
2021-03-17329 citations
S11 Long-term follow-up of the phase 1 START trial of onasemnogene abeparvovec gene therapy in spinal muscular atrophy type 1
2021-01-213 citations
S12 Onasemnogene abeparvovec gene therapy for spinal muscular atrophy type 1: phase 3 study (STR1VE-US)
2021-01-212 citations
Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1 (SMA1): Phase 3 US Study (STR1VE) Update (1828)
Neurology2020-04-1419 citations
250 AVXS-101 phase 3 study in spinal muscular atrophy type 1
Journal of Neurology Neurosurgery & Psychiatry2019-11-144 citations
15.33 AVXS-101 in presymptomatic spinal muscular atrophy (SMA)
Journal of Neurology Neurosurgery & Psychiatry2019-11-141 citations
S61 Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): preliminary pulmonary and ventilatory findings from the phase 3 study (STR1VE)
2019-11-12

10 more papers not shown