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Start clinical study zolgensma ZOLGENSMA® (onasemnogene abeparvovec-xioi) was studied in children who were showing symptoms (symptomatic) of spinal muscular atrophy (SMA) before treatment. Two studies done by Lowes and Dabbous support the use of Zolgensma as a more effective treatment for SMA Type 1. 1 The RESTORE registry, an ongoing noninterventional study initiated in 2018, evaluated long-term outcomes of SMA treatments, including onasemnogene abeparvovec. May 7, 2019 · The sustained clinical impact following dosing suggests that Zolgensma effectively halts motor neuron loss and adds to the evidence of the long-term durability of Zolgensma. Mar 19, 2025 · The first clinical trial testing Zolgensma, launched in 2014, was called START (NCT02122952). In October 2019, the FDA placed a partial clinical hold on studies of intrathecal Zolgensma®, after AveXis reported safety events in a pre-clinical study. 2 years after dosing. govPhase 3, open-label, single-arm study of a single, one-time dose of onasemnogene abeparvovec-xioi (gene replacement therapy) in patients with spinal muscular atrophy who meet enrollment criteria and are genetically defined by bi-allelic deletion of survival motor neuron 1 gene (SMN1) with 2 or 3 copies of survival motor neuron 2 gene (SMN2 May 24, 2019 · The efficacy of Zolgensma in pediatric patients less than 2 years of age with SMA with bi-allelic mutations in the SMN1 gene was evaluated in STR1VE, an open-label, single-arm clinical trial (ongoing), and in START, an open-label, single-arm, ascending-dose clinical trial (completed). Participants will ‘roll over’ from their respective previous (parent) study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to Zolgensma treatment. This includes two patients who achieved the new milestone of standing with assistance during the long-term follow-up period. 14 All infants treated with Zolgensma in CL-101 were alive and event-free through 24 months of follow-up. May 24, 2019 · Zolgensma was also tested in a phase 3 trial in the U. Mar 19, 2025 · A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the treatment of older patients with spinal muscular atrophy (SMA). S. 1 years of age) than the children treated in previous clinical studies 1-6 Nearly all patients treated maintained or improved motor milestones after 52 weeks, with most Dec 31, 2019 · START was a Phase 1 study evaluating the safety and efficacy of a one-time IV infusion of Zolgensma in SMA Type 1 patients with the onset of clinical symptoms before six months of age. The first clinical trial (START) was a single center, open label with dose escalation after the first three patients received low dose. These trials aim to assess the long-term safety and effectiveness of the drug in patients who have received it through intravenous (IV) or Mar 19, 2025 · The findings, based on up to 5 years of follow-up, aligned with previously reported clinical trial data, showing no unexpected safety concerns or late-onset toxicities. These studies generally enrolled babies with type 1 SMA who were six months or younger when they received Zolgensma and had not received other SMA treatments. The first in-human trial for intravenous (IV) administration of Zolgensma STRIVE began in April 2014. Feb 24, 2025 · Zolgensma in clinical trials The approval of Zolgensma in the U. Additionally, results from the phase 3 STR1VE-US (also known as STR1VE) study were reported in April, 2021, and confirmed the findings of START. START was an open-label, single-arm, dose-escalation clinical trial that enrolled 15 patients with SMA Type 1. | A This is a long-term follow-up safety and efficacy study of children in clinical trials who were treated intravenously with Zolgensma. 3 lbs [22]. At the time of the original ICER report, data were available only from a single, small (12 patients) open-label, 2-cohort clinical trial (CL-101) of Zolgensma and its extension study (START) in Type I SMA. 1 years of age) than the children treated in previous clinical The favourable benefit-risk profile shown in this study supports the use of onasemnogene abeparvovec for treatment of symptomatic patients with genetic or clinical characteristics predictive of infantile-onset spinal muscular atrophy type 1. Many of the current clinical studies involving Zolgensma are long-term follow-up studies monitoring the safety and efficacy of the drug in individuals who received the one-time treatment, including: Apr 11, 2023 · Highlighting the remarkable durability of Zolgensma, data from LT-001, an ongoing 15-year LTFU study of patients who completed the Phase 1 START study, showed that up to 7. 7 lbs and 46. Mar 4, 2024 · The SMART study supplements a growing body of evidence on the use of Zolgensma in a patient population older and heavier (1. May 24, 2019 · The efficacy of Zolgensma in pediatric patients less than 2 years of age with SMA with bi-allelic mutations in the SMN1 gene was evaluated in STR1VE, an open-label, single-arm clinical trial (ongoing), and in START, an open-label, single-arm, ascending-dose clinical trial (completed). Both tested the gene therapy in babies up to 6 months of age with type 1 SMA. Importantly, it also appeared to result in improved and sustained muscle function. START Trial Results Page last checked: 22nd November 2022 Last updated: 5th April 2017 In April 2017, the completed trial data indicated that what was then called AVXS-101 appeared to be safe and well tolerated by the young SMA Type 1 children being treated. Following extensive preclinical investigation, Zolgensma ® was evaluated in two clinical trials of symptomatic infantile onset SMA. During animal tests, Zolgensma® was observed to be associated with dorsal root ganglia (DRG) mononuclear cell inflammation and neuronal cell body degeneration or loss in some cases. May 17, 2021 · In this analysis of data from the ongoing, long-term follow-up of the START clinical trial, a single intravenous dose of onasemnogene abeparvovec in patients with SMA type 1 continued to have a monitorable and manageable safety profile for up to 6. The study concluded in 2019 and enrolled 15 patients who received a single infusion of a low dose (3 infants) or a high dose (12 infants) of Zolgensma. -based Phase 3 clinical trial called STR1VE (NCT03306277). . 7–48 months, with 7 STRONG Phase 1 and 2 Trial Results Page last checked: 19th April 2023 Last updated: 19th April 2023 Recently published data from the STRONG clinical trial of Zolgensma indicate that the gene therapy is safe and can be effective when delivered intrathecally, meaning directly into the fluid bathing the brain and spinal cord. Mar 20, 2023 · Basel, March 20, 2023 — Novartis today presented new data which underscore the transformational and sustained benefit of Zolgensma ® (onasemnogene abeparvovec), an essential one-time gene therapy for the treatment of spinal muscular atrophy (SMA). Sep 7, 2022 · Details for study NCT03505099, | ClinicalTrials. Apr 11, 2023 · Sitra Tauscher-Wisniewski, MD, vice president clinical development and analytics, Novartis Gene Therapies, talked about the new data findings from a study on gene therapy Zolgensma at the 2023 MDA conference. Apr 30, 2019 · The completed Phase 1 clinical trial (NCT02122952) was conducted using a single lot of ZOLGENSMA drug product, administered to two cohorts of subjects. Dec 3, 2018 · The AVXS-101, now known as ZOLGENSMA ® (onasemnogene abeparvovec-xxxx)[1], filing is supported by data from the START trial which demonstrated a dramatic increase in survival and transformative improvement in achievement of developmental milestones compared to the natural history of SMA Type 1[2 Zolgensma: SMA Type I. Mar 20, 2023 · Highlighting the remarkable durability of Zolgensma, data from LT-001, an ongoing 15-year LTFU study of patients who completed the Phase 1 START study, showed that up to 7. 5 years post-dosing, children who were treated after presenting symptoms of SMA maintained all previously achieved motor milestones. The SMART study is assessing the safety, tolerability, and efficacy of intravenous infusions of Zolgensma in patients with SMA that weigh between 18. The key evidence used during its assessment for use in the UK was from the phase 1 START trial and phase 3 STR1VE-US. These findings differed from the clinical course described in natural history studies of patients given supportive care. Feb 18, 2025 · Clinical trial results Zolgensma has been tested in many clinical trials. About Zolgensma ® Zolgensma ® (onasemnogene abeparvovec-xioi; AVXS-101) is an investigational gene therapy currently in development as a one-time infusion for SMA Type 1. An additional 12 patients received a high dose. The clinical evidence included in the review of onasemnogene abeparvovec is presented in 3 sections. govPhase 3, open-label, single-arm, single-dose, study of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with spinal muscular atrophy (SMA) Type 1 who meet enrollment criteria and are genetically defined by nonfunctional survival motor neuron 1 gene (SMN1) with 1 or 2 copies of survival motor neuron 2 gene (SMN2 Nov 2, 2017 · Preclinical studies of SMN gene-replacement therapy in the SMNΔ7 mouse model showed improvements in survival and motor function with early treatment, presumably at a time when motor neurons are May 24, 2022 · This study describes the clinical efficacy and tolerability of gene replacement therapy with onasemnogene abeparvovec over a 3-month period in 9 SMA type 1 patients aged 1. It tested the intravenous, or into-the-vein, therapy in 15 babies with SMA type 1 — the most common, and one of the most severe forms of the disease. The START trial (NCT02122952) was a phase 1, open-label, ascending-dose clinical trial in patients with SMA type 1. Jul 1, 2021 · The secondary objective is to determine whether developmental milestones achieved in the START phase 1 clinical trial were maintained and new milestones gained. This and the Phase 1 START follow-up trial led to the eventual approval of this one time gene therapy in both the US and Europe for children with SMA Type 1. ZOLGENSMA was also studied in symptomatic children in a trial called START, which studied safety and dosing. The second section includes indirect evidence selected from the literature, or ZOLGENSMA® (onasemnogene abeparvovec-xioi) has been clinically proven to stop the progression of spinal muscular atrophy (SMA) and stop motor neuron loss with just one dose. Results from both studies show the continued effectiveness and durability of Zolgensma, which continues to display a favourable benefit-to-risk profile. Patients were symptomatic with onset of clinical symptoms before 6 months of age. Jan 6, 2025 · Investigators recently published study data from the phase 3b SMART study (NCT04851873), the first trial to evaluate intravenous (IV) onasemnogene abeparvovec (Zolgensma; Novartis), an FDA-approved therapy for spinal muscular atrophy (SMA), in patients with the disease weighing 8. 5 – 9. The first section, Systematic Review, includes pivotal studies provided in the sponsor’s submission to CADTH and Health Canada, as well as those studies that were selected according to an a priori protocol. was supported by data from a Phase 1 clinical trial called START (NCT02122952) and a U. The key trial looked at safety and efficacy and was called STR1VE. Details for study NCT03306277, | ClinicalTrials. Overall, 65 patients aged <6 months at time of Zolgensma treatment were analyzed. Onasemnogene abeparvovec-xioi, previously AVXS-101 ( Zolgensma™) is a gene therapy developed by Novartis Gene Therapies (previously AveXis), designed to address the genetic root cause of SMA by replacing the function of the missing or faulty SMN1 gene. All patients in this study who received Zolgensma were alive, free of permanent ventilation and continued to maintain developmental milestones. After reviewing each literarture study, we noticed positive results regarding the prescribing of Zolgensma. All of the participants in LT001 received an intravenous (into the blood) injection of Zolgensma after the onset of SMA symptoms. 5-21 kg. Administered during a single, intravenous (IV) infusion, Zolgensma delivers a new working copy of the SMN1 gene into a patient’s cells Mar 14, 2022 · The study retrospectively assessed the percentage of patients who achieved each endpoint and all three endpoints at predetermined times or at the end of the study (24 months of age in START and 18 months of age in STR1VE-EU and STR1VE-US). called STR1VE, with interim data presented at the 2019 MDA Clinical & Scientific Conference and the 2019 Academy of Neurology Annual Meeting validating the START trial results. 2 During the time of LT-001, three The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of AVXS-101 as a treatment of spinal muscular atrophy Type 1 (SMN1). The trial studied the safety and efficacy of ZOLGENSMA in children with 2 and 3 copies of the SMN2 backup Findings In this ongoing, long-term follow-up safety study of 13 infants with symptomatic spinal muscular atrophy type 1 treated with a single low or therapeutic dose of onasemnogene abeparvovec in the START phase 1 clinical trial, a favorable safety profile was observed for up to 6. ZOLGENSMA was studied in children who were not showing symptoms (presymptomatic) of SMA in a trial called SPR1NT. ONASEMNOGENE ABEPARVOVEC (Zolgensma): A Gene Therapy for Spinal Muscular Atrophy Onasemnogene Abeparvovec, also known as Zolgensma, is a groundbreaking gene therapy drug being studied in clinical trials for the treatment of Spinal Muscular Atrophy (SMA). This review analyzed the challenges and considerations for Zolgensma and determined if they outweighed the efficacy of treatment. m9xb7 m1m zujxw 62 mhe o57s g1u 4nt59jb ts jx94