Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 (SPR1NT)
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ClinicalTrials.gov Identifier: NCT03505099 |
Recruitment Status :
Completed
First Posted : April 23, 2018
Results First Posted : January 11, 2022
Last Update Posted : September 7, 2022
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Tracking Information | |||||||
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First Submitted Date ICMJE | April 13, 2018 | ||||||
First Posted Date ICMJE | April 23, 2018 | ||||||
Results First Submitted Date ICMJE | December 14, 2021 | ||||||
Results First Posted Date ICMJE | January 11, 2022 | ||||||
Last Update Posted Date | September 7, 2022 | ||||||
Actual Study Start Date ICMJE | April 2, 2018 | ||||||
Actual Primary Completion Date | June 15, 2021 (Final data collection date for primary outcome measure) | ||||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
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Change History | |||||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE | Not Provided | ||||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||
Descriptive Information | |||||||
Brief Title ICMJE | Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 | ||||||
Official Title ICMJE | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy With Multiple Copies of SMN2 | ||||||
Brief Summary | To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in pre-symptomatic patients with SMA and 2 or 3 copies SMN2 | ||||||
Detailed Description | Phase 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). Patients with SMN1 point mutations or the SMN2 gene modifier mutation (c.859G>C) may enroll but will not be included in the efficacy analysis sets. The study includes a screening period, a gene replacement therapy period, and a follow-up period. During the screening period (Days -30 to -2), patients whose parent(s)/legal guardian(s) provide informed consent will undergo screening procedures to determine eligibility for study enrollment. Patients who meet the entry criteria will enter the in-patient gene replacement therapy period (Day -1 to Day 2). On Day -1, patients will be admitted to the hospital for pre-treatment baseline procedures. On Day 1, patients will receive a single, one-time intravenous (IV) infusion of onasemnogene abeparvovec-xioi, and will undergo in-patient safety monitoring for a minimum of 24 hours post infusion. Patients may be discharged 24 hours (48 hours in Japan) after the infusion, based on Investigator judgment. During the outpatient follow-up period (Days 3 to End of Study at 18 or 24 of age, dependent upon respective SMN2 copy number), patients will return at regularly scheduled intervals for efficacy and safety assessments until the End of Study when the patient reaches 18 months of age (SMN2 = 2) or 24 months of age (SMN2 = 3). After the End of Study visit, eligible patients will be invited to rollover into a long-term follow up study. |
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Study Type ICMJE | Interventional | ||||||
Study Phase ICMJE | Phase 3 | ||||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Intervention Model Description: Open-label, single arm Masking: None (Open Label)Primary Purpose: Treatment |
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Condition ICMJE | Spinal Muscular Atrophy | ||||||
Intervention ICMJE | Biological: onasemnogene abeparvovec-xioi
A non-replicating recombinant AAV9 containing the complimentary deoxyribonucleic acid (cDNA) of the human SMN gene under the control of the cytomegalovirus (CMV) enhancer/chicken-β-actin-hybrid promoter (CB). The AAV inverted terminal repeat (ITR) has been modified to promote intramolecular annealing of the transgene, thus forming a double-stranded transgene ready for transcription.
Other Name: Zolgensma
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Study Arms ICMJE | Experimental: onasemnogene abeparvovec-xioi
One-time intravenous infusion of onasemnogene abeparvovec-xioi at 1.1 X 10^14 vg/kg
Intervention: Biological: onasemnogene abeparvovec-xioi
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||
Recruitment Status ICMJE | Completed | ||||||
Actual Enrollment ICMJE |
30 | ||||||
Original Estimated Enrollment ICMJE |
44 | ||||||
Actual Study Completion Date ICMJE | June 15, 2021 | ||||||
Actual Primary Completion Date | June 15, 2021 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | up to 42 Days (Child) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||
Listed Location Countries ICMJE | Australia, Belgium, Canada, Japan, United Kingdom, United States | ||||||
Removed Location Countries | Germany, Israel, Italy, Korea, Republic of, Spain, Taiwan | ||||||
Administrative Information | |||||||
NCT Number ICMJE | NCT03505099 | ||||||
Other Study ID Numbers ICMJE | AVXS-101-CL-304 2017-004087-35 ( EudraCT Number ) JapicCTI-184203 ( Registry Identifier: JapicCTI ) COAV101A12303 ( Other Identifier: Novartis Pharmaceuticals ) |
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Has Data Monitoring Committee | Yes | ||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Novartis ( Novartis Gene Therapies ) | ||||||
Original Responsible Party | AveXis, Inc. | ||||||
Current Study Sponsor ICMJE | Novartis Gene Therapies | ||||||
Original Study Sponsor ICMJE | AveXis, Inc. | ||||||
Collaborators ICMJE | PRA Health Sciences | ||||||
Investigators ICMJE |
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PRS Account | Novartis | ||||||
Verification Date | August 2022 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |