Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
STRIVE-EU
Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
3 other identifiers
interventional
33
4 countries
10
Brief Summary
Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in patients with spinal muscular atrophy (SMA) Type 1 who meet enrollment criteria and are genetically defined by a biallelic pathogenic mutation of the survival motor neuron 1 gene (SMN1) with one or two copies of survival motor neuron 2 gene (SMN2). Up to 30 patients \< 6 months (\< 180 days) of age at the time of gene replacement therapy (Day 1) will be enrolled.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Aug 2018
10 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 4, 2018
CompletedFirst Posted
Study publicly available on registry
March 12, 2018
CompletedStudy Start
First participant enrolled
August 16, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 11, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
September 11, 2020
CompletedResults Posted
Study results publicly available
April 2, 2021
CompletedJanuary 26, 2026
January 1, 2026
2.1 years
March 4, 2018
March 9, 2021
January 7, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Number of Participants Who Achieve Independent Sitting for at Least 10 Seconds
Independent sitting is defined by the World Health Organization Multicentre Growth Reference Study, confirmed by video recording, as a participant who sits up straight with head erect for at least 10 seconds; participant does not use arms or hands to balance body or support position.
From Day 1 up to 18 Months of Age Visit (Up to a Maximum of Approximately 17 Months)
Secondary Outcomes (1)
Event-free Survival at 14 Months of Age
Up to 14 months of age
Study Arms (1)
Onasemnogene Abeparvovec-xioi
EXPERIMENTALOnasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the human survival motor neuron (SMN) gene under the control of the cytomegalovirus (CMV) enhancer/chicken β-actin-hybrid promoter (CB).
Interventions
Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the human survival motor neuron (SMN) gene under the control of the cytomegalovirus (CMV) enhancer/chicken β-actin-hybrid promoter (CB).
Eligibility Criteria
You may qualify if:
- Patients with SMA Type 1 as determined by diagnosis of SMA based on gene mutation analysis with biallelic SMN1 mutations (deletion or point mutations) and one or two copies of SMN2 \[inclusive of the known SMN2 gene modifier mutation (c.859G\>C)\]
- Patients must be \< 6 months (\< 180 days) of age at the time of onasemnogene abeparvovec-xioi infusion
- Patients must have a swallowing evaluation test performed prior to administration of gene replacement therapy
You may not qualify if:
- Previous, planned or expected scoliosis repair surgery/procedure prior to 18 months of age
- Use of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry \< 95% saturation at screening
- Use or requirement of non-invasive ventilatory support for 12 or more hours daily in the two weeks prior to dosing
- Patient with signs of aspiration based on a swallowing test or whose weight-for-age falls below the 3rd percentile based on World Health Organization (WHO) Child Growth Standards and unwilling to use an alternative method to oral feeding
- Participation in recent SMA treatment clinical trial (with the exception of observational cohort studies or non-interventional studies) or receipt of an investigational or commercial compound, product or therapy administered with the intent to treat SMA (eg, nusinersen, valproic acid,) at any time prior to screening for this trial.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (10)
University Hospital Ghent Neuromuscular reference center
Ghent, Belgium
Neuropédiatrie - Centre de Référence des Maladies Neuromusculaires
Liège, Belgium
Hôpital Armand Trousseau
Paris, France
Istituto Gianninia Gaslini
Genova, Italy
Policlinico "G. Martino"
Messina, Italy
Carlo Besta Neurological Research Institute
Milan, Italy
University of Milan
Milan, Italy
Policlinico Gemelli
Rome, Italy
Great Ormond Street Hospital for Children
London, United Kingdom
The John Walton Muscular Dystrophy Research Centre MRC Centre for Neuromuscular Diseases at Newcastle
Newcastle upon Tyne, United Kingdom
Related Publications (2)
Mercuri E, Muntoni F, Baranello G, Masson R, Boespflug-Tanguy O, Bruno C, Corti S, Daron A, Deconinck N, Servais L, Straub V, Ouyang H, Chand D, Tauscher-Wisniewski S, Mendonca N, Lavrov A; STR1VE-EU study group. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial. Lancet Neurol. 2021 Oct;20(10):832-841. doi: 10.1016/S1474-4422(21)00251-9.
PMID: 34536405DERIVEDDay JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH. Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy. Drug Saf. 2021 Oct;44(10):1109-1119. doi: 10.1007/s40264-021-01107-6. Epub 2021 Aug 12.
PMID: 34383289DERIVED
Related Links
MeSH Terms
Interventions
Limitations and Caveats
A comparison of the results from this study to the results from the natural history observational study (Pediatric Neuromuscular Clinical Research Network (PNCR), Finkel et al, 2014) are included in the Novartis Clinical Trial Results, as a historical control. These full results are available via this link: https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=17805.
Results Point of Contact
- Title
- EMEA Medical Information
- Organization
- Novartis Gene Therapies EU Limited
Study Officials
- STUDY CHAIR
AveXis Medinfo
Sponsor GmbH
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 4, 2018
First Posted
March 12, 2018
Study Start
August 16, 2018
Primary Completion
September 11, 2020
Study Completion
September 11, 2020
Last Updated
January 26, 2026
Results First Posted
April 2, 2021
Record last verified: 2026-01
Data Sharing
- IPD Sharing
- Will share
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.