A Phase 3 interventional study of Onasemnogene Abeparvovec-xioi in SMA - Spinal Muscular Atrophy and Gene Therapy, sponsored by Novartis Gene Therapies. Completed at 16 sites in United States. Open to participants aged Up to 180 Days. Per ClinicalTrials.gov, last updated 2026-01-26.
Sponsored by Novartis Gene Therapies (part of Novartis) · Phase 3, Interventional, and Treatment
Among 121 studies in Muscular Atrophy, Spinal with a site in United States — 21 open to participants now, 39 under way, 7 of them phase 3. Muscular Atrophy, Spinal · United States.
Among the 121 studies in Muscular Atrophy, Spinal with a site in United States, this one is the 50th largest by enrollment of the 76 interventional ones that give a count, is one of 4 that started in 2017, is one of 18 in phase 3, and was the 48th registered.
282 studies on the registry are indexed under Muscular Atrophy, Spinal; 73 are open to participants now.
Browse Muscular Atrophy, Spinal studies →21 of the 121 studies in Muscular Atrophy, Spinal with a site in United States are open now. Recruiting first, the newest first:
Johns Hopkins University · Sites in United States
St. Jude Children's Research Hospital · Sites in United States
Biogen · Sites in United States
Biogen · Sites in United States, China
Novartis Pharmaceuticals · Sites in United States
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 58 Muscular Atrophy, Spinal studies recruiting now, 28 (48%) set no upper age limit and 27 (47%) stop at 75 or younger; 46 take children under 18; 11 take healthy volunteers.
This study: Up to 180 Days · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
121 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 18 · Phase 2 27 · Phase 3 20 · Phase 4 6
First in 1986; this study’s year is marked. 4 started in 2017.
This study's enrollment of 22 is below the median of 29 across 156 interventional studies indexed under Muscular Atrophy, Spinal.
Median enrollment 33 across the 76 interventional studies, 75 across the 43 observational. Of the 76 that completed or were terminated, 33 (43%) have results posted.
This tab as a report to print or save as PDF → With an account.
61 of 121 have an industry lead sponsor. The 10 most frequent:
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.
Novartis Gene Therapies has 4 studies filed under Muscular Atrophy, Spinal — fewer than ten, so there is no comparison to draw.
7 of the studies in Muscular Atrophy, Spinal with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05067790Phase 3BiogenA Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND) | 2027-09-24 |
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
| NCT07223320Teachers College, Columbia UniversityHABIT-ILE + FST in Children With SMA | 2026-12-31 |
| NCT07400198Jacqueline MontesGait and Bone Health in SMA | 2027-06-30 |
| NCT06532474St. Jude Children's Research HospitalExploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies | 2027-09 |
Among the 121 studies in Muscular Atrophy, Spinal with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
Phase 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). Fifteen (15) participants \< 6 months (\< 180 days) of age at the time of gene replacement therapy (Day 1) will be enrolled.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
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.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
One-time Intravenous administration of onasemnogene abeparvovec-xioi at the therapeutic dose.
Biological: Onasemnogene Abeparvovec-xioi
Non-replicating recombinant adeno-associated virus serotype 9 (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.
Also known as: Zolgensma
Achievement of Independent Sitting for at Least 30 Seconds
Independent sitting is defined as sitting up straight with head erect for at least 30 seconds. This endpoint is a co-primary endpoint. The two co-primary efficacy endpoints were assessed in sequence: The endpoint of functional independent sitting was assessed first and, only when this assessment met statistical significance, was the endpoint of event-free survival assessed.
Time frame: Up to 18 months
Event-free Survival
Survival is defined by the avoidance of combined endpoint of either death or permanent ventilation, which is defined by tracheostomy or by the requirement of ≥ 16 hours of respiratory assistance per day for ≥ 14 consecutive days in the absence of an acute reversible illness, excluding perioperative ventilation. Permanent ventilation is considered a surrogate for death. An acute reversible illness is defined as any condition other than SMA that results in increased medical intervention. The endpoint is a co-primary endpoint. The two co-primary efficacy endpoints were assessed in sequence: The endpoint of functional independent sitting was assessed first and, only when this assessment met statistical significance was the survival endpoint assessed.
Time frame: 14 months
Ability to Thrive
Ability to thrive is defined as achieving all of the following at 18 months of age: * does not receive nutrition through mechanical support or other non-oral method * ability to tolerate thin liquids as demonstrated through a formal swallowing test * maintains weight This is a co-secondary endpoint. The two co-secondary endpoints were assessed in sequence: The endpoint of ability to thrive was assessed first and, only when this assessment met statistical significance was the endpoint of ventilatory support independence assessed.
Time frame: 18 months
Ventilatory Support Independence
Ventilatory support independence is defined as requiring no daily ventilator support/usage at 18 months of age, excluding acute reversible illness and perioperative ventilation, through assessment of actual usage data captured from the device (Phillips Trilogy BiPAP device). This endpoint is derived solely from the Phillips Trilogy BiPAP device. This is a co-secondary endpoint. The two co-secondary endpoints were assessed in sequence: The endpoint of ability to thrive was assessed first and, only when this assessment met statistical significance was the endpoint of ventilatory support independence assessed.
Time frame: Up to 18 months
22 participants were recruited across 16 study centers in the United States.
| Milestone | Onasemnogene Abeparvovec-xioi |
|---|---|
| Started | 22 |
| Completed | 19 |
| Not completed | 3 |
| Withdrew: Death | 1 |
| Withdrew: Adverse event | 1 |
| Withdrew: Withdrawal by subject | 1 |
Independent sitting is defined as sitting up straight with head erect for at least 30 seconds. This endpoint is a co-primary endpoint. The two co-primary efficacy endpoints were assessed in sequence: The endpoint of functional independent sitting was assessed first and, only when this assessment met statistical significance, was the endpoint of event-free survival assessed.
| Participants | Onasemnogene Abeparvovec-xioi |
|---|---|
| Achievement of Independent Sitting for at Least 30 Seconds | 13 |
Survival is defined by the avoidance of combined endpoint of either death or permanent ventilation, which is defined by tracheostomy or by the requirement of ≥ 16 hours of respiratory assistance per day for ≥ 14 consecutive days in the absence of an acute reversible illness, excluding perioperative ventilation. Permanent ventilation is considered a surrogate for death. An acute reversible illness is defined as any condition other than SMA that results in increased medical intervention. The endpoint is a co-primary endpoint. The two co-primary efficacy endpoints were assessed in sequence: The endpoint of functional independent sitting was assessed first and, only when this assessment met statistical significance was the survival endpoint assessed.
| Participants | Onasemnogene Abeparvovec-xioi |
|---|---|
| Event-free Survival | 20 |
Ability to thrive is defined as achieving all of the following at 18 months of age: * does not receive nutrition through mechanical support or other non-oral method * ability to tolerate thin liquids as demonstrated through a formal swallowing test * maintains weight This is a co-secondary endpoint. The two co-secondary endpoints were assessed in sequence: The endpoint of ability to thrive was assessed first and, only when this assessment met statistical significance was the endpoint of ventilatory support independence assessed.
| Participants | Onasemnogene Abeparvovec-xioi |
|---|---|
| Ability to Thrive | 9 |
Ventilatory support independence is defined as requiring no daily ventilator support/usage at 18 months of age, excluding acute reversible illness and perioperative ventilation, through assessment of actual usage data captured from the device (Phillips Trilogy BiPAP device). This endpoint is derived solely from the Phillips Trilogy BiPAP device. This is a co-secondary endpoint. The two co-secondary endpoints were assessed in sequence: The endpoint of ability to thrive was assessed first and, only when this assessment met statistical significance was the endpoint of ventilatory support independence assessed.
| Participants | Onasemnogene Abeparvovec-xioi |
|---|---|
| Ventilatory Support Independence | 18 |
Collected over Up to 18 months. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Onasemnogene Abeparvovec-xioi | 1/22 (4.5%) | 10/22 (45.5%) | 22/22 (100%) |
| Event | Onasemnogene Abeparvovec-xioi |
|---|---|
| Respiratory distressRespiratory, thoracic and mediastinal disorders | 4/22 |
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 2/22 |
| BronchiolitisInfections and infestations | 2/22 |
| PneumoniaInfections and infestations | 2/22 |
| Respiratory syncytial virus bronchiolitisInfections and infestations | 2/22 |
| Alanine aminotransferase increasedInvestigations | 1/22 |
| Aspartate aminotransferase increasedInvestigations | 1/22 |
| Human metapneumovirus test positiveInvestigations | 1/22 |
| Transaminases increasedInvestigations | 1/22 |
| CyanosisCardiac disorders | 1/22 |
| Event | Onasemnogene Abeparvovec-xioi |
|---|---|
| PyrexiaGeneral disorders | 12/22 |
| Upper respiratory tract infectionInfections and infestations | 11/22 |
| ConstipationGastrointestinal disorders | 9/22 |
| ScoliosisMusculoskeletal and connective tissue disorders | 9/22 |
| CoughRespiratory, thoracic and mediastinal disorders | 7/22 |
| Aspartate aminotransferase increasedInvestigations | 6/22 |
| Respiratory distressRespiratory, thoracic and mediastinal disorders | 6/22 |
| Use of accessory respiratory musclesRespiratory, thoracic and mediastinal disorders | 5/22 |
| Alanine aminotransferase increasedInvestigations | 5/22 |
| Respiration abnormalRespiratory, thoracic and mediastinal disorders | 5/22 |
| Age, Categorical(Participants) | Onasemnogene Abeparvovec-xioi |
|---|---|
| <=18 years | 22 |
| Between 18 and 65 years | 0 |
| >=65 years | 0 |
| Age, Continuous(months) | Onasemnogene Abeparvovec-xioi |
|---|---|
| Mean | 3.7 ± 1.6 |
| Sex: Female, Male(Participants) | Onasemnogene Abeparvovec-xioi |
|---|---|
| Female | 12 |
| Male | 10 |
| Ethnicity (NIH/OMB)(Participants) | Onasemnogene Abeparvovec-xioi |
|---|---|
| Hispanic or Latino | 4 |
| Not Hispanic or Latino | 18 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Onasemnogene Abeparvovec-xioi |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 2 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 3 |
| White | 11 |
| More than one race | 0 |
| Unknown or Not Reported | 6 |
| Region of Enrollment(participants) | Onasemnogene Abeparvovec-xioi |
|---|---|
| United States | 22 |
| Patient reported hospitalizations(participants) | Onasemnogene Abeparvovec-xioi |
|---|---|
| Yes | 17 |
| No | 5 |
| Weight at baseline(kg) | Onasemnogene Abeparvovec-xioi |
|---|---|
| Mean | 5.8 ± 1.1 |
1 further baseline measures are reported on the registry.
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 Oct 2017 to primary completion in Nov 2019, 24 months.
Results were first posted in Jul 2020, 8 months after primary completion.
Of 18 phase 3 interventional studies under Muscular Atrophy, Spinal, half reached primary completion within 33 months of starting (the median), counting those still running; a quarter within 24 and three quarters within 63. This study's 24 months is below that.
How these figures are worked out, and which studies are left out of them.
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