Phase 3
Completed N=33
Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
SMA
Source: ClinicalTrials.gov NCT03461289 ↗
Enrolled (actual)
33
Serious AEs
57.6%
Results posted
Apr 2021
Primary outcomePrimary: Number of Participants Who Achieve Independent Sitting for at Least 10 Seconds — 14 Participants
◆ Published Evidence
Highly cited
257citations · ~51 / year
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.
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.
Linked Publications (2)
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Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial.
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Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Who Achieve Independent Sitting for at Least 10 Seconds |
14 | — |
| SECONDARY Event-free Survival at 14 Months of Age |
31 | <0.0001 sig |
Eligibility Criteria
Inclusion Criteria
- 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
Exclusion Criteria
- 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.
Data sourced from ClinicalTrials.gov (NCT03461289) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.