A Phase 3 interventional study of Onasemnogene Abeparvovec-xioi in SMA, sponsored by Novartis Gene Therapies. Completed at 10 sites in 4 countries. Open to participants aged Up to 6 Months. Per ClinicalTrials.gov, last updated 2026-01-26.
Sponsored by Novartis Gene Therapies (part of Novartis) · Phase 3, Interventional, and Treatment
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.
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). 30 patients \< 6 months (\< 180 days) of age at the time of gene replacement therapy (Day 1) will be enrolled.
The trial 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 complete screening procedures to determine eligibility for trial enrollment. Patients who meet the entry criteria will enter the in-patient gene replacement therapy period (Day -1 to Day 3). On Day -1, patients will be admitted to the hospital for pre-treatment baseline procedures. On Day 1, patients will receive a one-time intravenous (IV) infusion of onasemnogene abeparvovec-xioi, and will undergo in-patient safety monitoring over the next 48 hours. Patients may be discharged 48 hours after the infusion, based on Investigator judgment. During the outpatient follow-up period (Days 4 to End of Trial at 18 months of age), patients will return at regularly scheduled intervals for efficacy and safety assessments until the End of Trial when the patient reaches 18 months of age. After the End of Trial visit, eligible patients will be asked to participate into the long-term follow up trial.
All post-treatment visits will be relative to the date on which gene replacement therapy is administered, until the patient is 14 months of age, after which they will be relevant to the patient's date of birth.
Novartis Gene Therapies is the lead sponsor of 8 studies on the registry; none are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 5 (100%) have results posted.
First submitted before Novartis Gene Therapies became part of Novartis.
Counted across the registry records on this site, refreshed daily.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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).
Biological: Onasemnogene Abeparvovec-xioi
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).
Also known as: AVXS-101, OAV101, Zolgensma
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.
Time frame: From Day 1 up to 18 Months of Age Visit (Up to a Maximum of Approximately 17 Months)
Event-free Survival at 14 Months of Age
Event-free survival at 14 months of age was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study by 14 months of age.
Time frame: Up to 14 months of age
| Milestone | Onasemnogene Abeparvovec-xioi |
|---|---|
| Started | 33 |
| Completed | 32 |
| Not completed | 1 |
| Withdrew: Death | 1 |
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.
| Participants | Onasemnogene Abeparvovec-xioi |
|---|---|
| Number of Participants Who Achieve Independent Sitting for at Least 10 Seconds | 14 |
Event-free survival at 14 months of age was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study by 14 months of age.
| Participants | Onasemnogene Abeparvovec-xioi |
|---|---|
| Event-free Survival at 14 Months of Age | 31 |
Collected over From Day 1 up to 30 days after the 18 months of age visit (up to a maximum of 17 months). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Onasemnogene Abeparvovec-xioi | 1/33 (3%) | 19/33 (57.6%) | 32/33 (97%) |
| Event | Onasemnogene Abeparvovec-xioi |
|---|---|
| PneumoniaInfections and infestations | 5/33 |
| PyrexiaGeneral disorders | 4/33 |
| GastroenteritisInfections and infestations | 3/33 |
| Respiratory tract infectionInfections and infestations | 3/33 |
| Upper respiratory tract infectionInfections and infestations | 3/33 |
| GastrostomySurgical and medical procedures | 2/33 |
| Feeding disorderMetabolism and nutrition disorders | 2/33 |
| BronchiolitisInfections and infestations | 2/33 |
| Lower respiratory tract infectionInfections and infestations | 2/33 |
| Respiratory syncytial virus infectionInfections and infestations | 2/33 |
| Event | Onasemnogene Abeparvovec-xioi |
|---|---|
| PyrexiaGeneral disorders | 20/33 |
| Upper respiratory tract infectionInfections and infestations | 10/33 |
| Alanine aminotransferase increasedInvestigations | 9/33 |
| Aspartate aminotransferase increasedInvestigations | 8/33 |
| VomitingGastrointestinal disorders | 8/33 |
| HypertransaminasaemiaHepatobiliary disorders | 8/33 |
| ConstipationGastrointestinal disorders | 7/33 |
| CoughRespiratory, thoracic and mediastinal disorders | 6/33 |
| DiarrhoeaGastrointestinal disorders | 5/33 |
| HypertensionVascular disorders | 4/33 |
| Age, Categorical(Participants) | Onasemnogene Abeparvovec-xioi |
|---|---|
| <=18 years | 33 |
| Between 18 and 65 years | 0 |
| >=65 years | 0 |
| Age, Continuous(months) | Onasemnogene Abeparvovec-xioi |
|---|---|
| Mean | 4.055 ± 1.2799 |
| Sex: Female, Male(Participants) | Onasemnogene Abeparvovec-xioi |
|---|---|
| Female | 19 |
| Male | 14 |
| Race and Ethnicity Not Collected(Participants) | Onasemnogene Abeparvovec-xioi |
|---|
| Region of Enrollment(participants) | Onasemnogene Abeparvovec-xioi |
|---|---|
| Belgium | 4 |
| Italy | 23 |
| United Kingdom | 4 |
| France | 2 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — 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/.
Data sharing details ↗ · www.clinicalstudydatarequest.com
Ran from Aug 2018 to primary completion in Sep 2020, 24 months.
Results were first posted in Apr 2021, 6 months after primary completion.
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This study is completed, as verified in Jan 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Onasemnogene abeparvovec