A Phase 3 interventional study of onasemnogene abeparvovec-xioi in Spinal Muscular Atrophy, sponsored by Novartis Gene Therapies. Completed at 16 sites in 6 countries. Open to participants aged Up to 42 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 40th largest by enrollment of the 76 interventional ones that give a count, is one of 9 that started in 2018, is one of 18 in phase 3, and was the 52nd 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 42 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. 9 started in 2018.
This study's enrollment of 30 is close to 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.
To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in pre-symptomatic patients with SMA and 2 or 3 copies SMN2
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.
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.
Parent(s)/legal guardian(s) willing and able to complete the process and comply with study procedures and visit schedule
Patients with pre-symptomatic SMA Type 1 as determined by the following features:
a. 2 copies of SMN2 (n ≥14)
Patients with pre-symptomatic SMA Type 2 as determined by the following features:
Exclusion Criteria:
Biological mother with active viral infection as determined by screening laboratory samples (includes [HIV] or positive serology for or C)
Severe nonpulmonary/respiratory tract infection within 4 Weeks before administration of gene replacement therapy or concomitant illness that, in the opinion of the Investigator or Sponsor medical monitor, creates unnecessary risks for gene replacement therapy such as:
AntiAAV9 antibody titer >1:50 as determined by Enzyme-linked Immunosorbent Assay (ELISA) binding immunoassay
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.
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.
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 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.
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.
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.
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.
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.
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.
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.
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.
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.
One-time intravenous infusion of onasemnogene abeparvovec-xioi at 1.1 X 10\^14 vg/kg
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.
Also known as: Zolgensma
Cohort 1: Number of Participants Who Achieved Sitting Alone for at Least 30 Seconds
Defined by the Bayley Scales of Infant and Toddler Development (BSID) Gross Motor (GM) subtest performance criteria number 26, confirmed by video recording, as a participant who sits for at least 30 seconds without assistance from another person or object. The participant was allowed to use their upper extremities.
Time frame: From Day 1 up to 18 months of age visit
Cohort 2: Number of Participants Who Achieved Standing Alone for at Least 3 Seconds
Defined by the BSID GM subtest performance criteria number 40, confirmed by video recording, as a participant who stands alone for at least 3 seconds unsupported.
Time frame: From Day 1 up to 24 months of age visit
Cohort 1: 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: From Day 1 up to 14 months of age
Cohort 1: Number of Participants Who Achieved the Ability to Maintain Weight at or Above the Third Percentile Without the Need for Non-Oral or Mechanical Feeding Support
The ability to maintain weight at or above the third percentile without the need for non-oral or mechanical feeding support was defined by meeting the following criteria at each visit up to 18 months of age: * Did not receive nutrition through mechanical support (i.e., feeding tube) * Maintained weight (≥ third percentile for age and sex as defined by World Health Organization \[WHO\] guidelines) consistent with the participant's age at the assessment.
Time frame: From Day 1 up to 18 months of age
Cohort 2: Number of Participants Who Achieved the Ability to Walk Alone
Defined by the BSID GM subtest performance criteria number 43, confirmed by video recording, as a participant who takes 5 coordinated independent steps.
Time frame: From Day 1 up to 24 months of age visit
A total of 30 participants took part in the trial at 16 sites in the United States, the United Kingdom, Belgium, Canada, Australia and Japan between April 2018 and June 2021.
| Milestone | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 |
|---|---|---|
| Started | 14 | 15 |
| Received avxs-101 | 14 | 15 |
| Completed | 14 | 15 |
| Not completed | 0 | 0 |
Defined by the Bayley Scales of Infant and Toddler Development (BSID) Gross Motor (GM) subtest performance criteria number 26, confirmed by video recording, as a participant who sits for at least 30 seconds without assistance from another person or object. The participant was allowed to use their upper extremities.
| Participants | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 |
|---|---|
| Cohort 1: Number of Participants Who Achieved Sitting Alone for at Least 30 Seconds | 14 |
Defined by the BSID GM subtest performance criteria number 40, confirmed by video recording, as a participant who stands alone for at least 3 seconds unsupported.
| Participants | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 |
|---|---|
| Cohort 2: Number of Participants Who Achieved Standing Alone for at Least 3 Seconds | 15 |
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 | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 |
|---|---|
| Cohort 1: Event-free Survival at 14 Months of Age | 14 |
The ability to maintain weight at or above the third percentile without the need for non-oral or mechanical feeding support was defined by meeting the following criteria at each visit up to 18 months of age: * Did not receive nutrition through mechanical support (i.e., feeding tube) * Maintained weight (≥ third percentile for age and sex as defined by World Health Organization \[WHO\] guidelines) consistent with the participant's age at the assessment.
| Participants | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 |
|---|---|
| Cohort 1: Number of Participants Who Achieved the Ability to Maintain Weight at or Above the Third Percentile Without the Need for Non-Oral or Mechanical Feeding Support | 13 |
Defined by the BSID GM subtest performance criteria number 43, confirmed by video recording, as a participant who takes 5 coordinated independent steps.
| Participants | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 |
|---|---|
| Cohort 2: Number of Participants Who Achieved the Ability to Walk Alone | 14 |
Collected over Cohort 1: Treatment-emergent adverse events (TEAEs) were collected from Day 1 up to the 18 months of age visit. Serious adverse events (SAEs) were collected from signing of informed consent to 30 days after the last study visit (up to a maximum of approximately 20 months). Cohort 2: TEAEs were collected from Day 1 up to the 24 months of age visit. SAEs were collected from signing of informed consent to 30 days after the last study visit (up to a maximum of approximately 26 months).. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | 0/14 (0%) | 5/14 (35.7%) | 14/14 (100%) |
| Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | 0/15 (0%) | 3/15 (20%) | 15/15 (100%) |
| Event | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 |
|---|---|---|
| Middle ear effusionEar and labyrinth disorders | 1/14 | 0/15 |
| Inguinal herniaGastrointestinal disorders | 1/14 | 0/15 |
| Croup infectiousInfections and infestations | 1/14 | 0/15 |
| PyelonephritisInfections and infestations | 1/14 | 0/15 |
| HypercalcaemiaMetabolism and nutrition disorders | 1/14 | 0/15 |
| Sleep apnoea syndromeRespiratory, thoracic and mediastinal disorders | 1/14 | 0/15 |
| Ear infectionInfections and infestations | 0/14 | 1/15 |
| PharyngitisInfections and infestations | 0/14 | 1/15 |
| LethargyNervous system disorders | 0/14 | 1/15 |
| Event | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 |
|---|---|---|
| PyrexiaGeneral disorders | 7/14 | 11/15 |
| Upper respiratory tract infectionInfections and infestations | 5/14 | 9/15 |
| TeethingGastrointestinal disorders | 2/14 | 5/15 |
| ConstipationGastrointestinal disorders | 4/14 | 1/15 |
| DiarrhoeaGastrointestinal disorders | 3/14 | 4/15 |
| Aspartate aminotransferase increasedInvestigations | 3/14 | 4/15 |
| CoughRespiratory, thoracic and mediastinal disorders | 1/14 | 4/15 |
| Gastrooesophageal reflux diseaseGastrointestinal disorders | 3/14 | 3/15 |
| VomitingGastrointestinal disorders | 3/14 | 2/15 |
| Viral upper respiratory tract infectionInfections and infestations | 3/14 | 1/15 |
| Age, Categorical(Participants) | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | Total |
|---|---|---|---|
| <=18 years | 14 | 15 | 29 |
| Between 18 and 65 years | 0 | 0 | 0 |
| >=65 years | 0 | 0 | 0 |
| Sex: Female, Male(Participants) | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | Total |
|---|---|---|---|
| Female | 10 | 9 | 19 |
| Male | 4 | 6 | 10 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | Total |
|---|---|---|---|
| Hispanic or Latino | 4 | 2 | 6 |
| Not Hispanic or Latino | 10 | 13 | 23 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race/Ethnicity, Customized(Participants) | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | Total |
|---|---|---|---|
| Asian | 2 | 2 | 4 |
| American Indian or Alaska Native | 0 | 1 | 1 |
| Black or African American | 1 | 0 | 1 |
| White | 7 | 10 | 17 |
| Other | 4 | 2 | 6 |
| SMN2 gene modifier mutation (c.859G>C) Present(Participants) | Cohort 1: Bi-allelic Deletions of SMN1 and 2 Copies of SMN2 | Cohort 2: Bi-allelic Deletions of SMN1 and 3 Copies of SMN2 | Total |
|---|---|---|---|
| Count of participants | 0 | 0 | 0 |
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 Apr 2018 to primary completion in Jun 2021, 38 months.
Results were first posted in Jan 2022, 6 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 38 months is above 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.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
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Studies of the drug tested here: Onasemnogene abeparvovec