A Phase 3 interventional study of Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II and Spinal Muscular Atrophy Type III, sponsored by Novartis Gene Therapies. Active, not recruiting at 31 sites in 9 countries. Per ClinicalTrials.gov, last updated 2026-09-24.
Sponsored by Novartis Gene Therapies (part of Novartis) · Phase 3, Interventional, and Treatment
Among 23 studies in Spinal Muscular Atrophies of Childhood with a site in United States — 3 open to participants now, 7 under way, 2 of them phase 3. Spinal Muscular Atrophies of Childhood · United States.
Among the 23 studies in Spinal Muscular Atrophies of Childhood with a site in United States, this one is the 4th largest by enrollment of the 13 interventional ones that give a count, is the only one that started in 2020, is one of 4 in phase 3, and was the 12th registered.
61 studies on the registry are indexed under Spinal Muscular Atrophies of Childhood; 18 are open to participants now.
Browse Spinal Muscular Atrophies of Childhood studies →3 of the 23 studies in Spinal Muscular Atrophies of Childhood with a site in United States are open now. Recruiting first, the newest first:
Jacqueline Montes · Sites in United States
Scholar Rock, Inc. · Sites in United States, Belgium, France and 4 more countries
Columbia University · 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 12 Spinal Muscular Atrophies of Childhood studies recruiting now, 5 (42%) set no upper age limit and 7 (58%) stop at 75 or younger; 11 take children under 18; 2 take healthy volunteers.
This study: 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.
23 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 4 · Phase 2 5 · Phase 3 4
First in 1986; this study’s year is marked. 1 started in 2020.
This study's enrollment of 85 is above the median of 15 across 41 interventional studies indexed under Spinal Muscular Atrophies of Childhood.
Median enrollment 40 across the 13 interventional studies, 88 across the 10 observational. Of the 15 that completed or were terminated, 7 (47%) have results posted.
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9 of 23 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 Spinal Muscular Atrophies of Childhood — fewer than ten, so there is no comparison to draw.
1 of the studies in Spinal Muscular Atrophies of Childhood with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07400198Jacqueline MontesGait and Bone Health in SMA | 2027-06-30 |
Among the 23 studies in Spinal Muscular Atrophies of Childhood with a site in United States, from their registry updates:
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This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
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.
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.
Participants received treatment with IV onasemnogene abeparvovec-xioi in an onasemnogene abeparvovec-xioi or received treatment with IT onasemnogene abeparvovec-xioi in an onasemnogene.
Biological: Onasemnogene Abeparvovec-xioi
Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the cytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec-xioi administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.
Also known as: Zolgensma
Number of Participants Who Reach Developmental Milestones
Assessed via the developmental milestone checklist, formed of 10 yes/no questions. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone.
Time frame: Up to 5 years
Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
The HFMSE was devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE is formed of 33 assessments rated from 0 (unable to perform functional task) to 2 (able to perform functional task unassisted). Higher scores on the total scale of 0-66 indicates higher levels of motor ability.
Time frame: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Pulmonary Assessment Results and Require Ventilatory Support
Participants will receive pulmonary assessments by a pulmonologist or appropriate clinician. Respiratory device data will be reviewed for participants receiving non-invasive ventilatory support.
Time frame: Up to 15 years
Number of Participants Who Experience Swallowing Dysfunction and Require Nutritional Support
Assessed via the swallowing function questionnaire, formed of 4 yes/ no questions and 1 body weight question.
Time frame: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change from Baseline in Physical Examination Findings
The physical examination includes review of the following systems: head, ears, eyes, nose and throat, lungs/thorax, cardiovascular, abdomen, musculoskeletal, neurologic, dermatologic, lymphatic, and genitourinary. In addition, visual inspection of the spine, back, shoulders, and hips looking for spinal curvature and asymmetry will be carried out. Joints will be assessed for loss of mobility and contractures.
Time frame: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Vital Signs Measurements
Vital sign measurements will include blood pressure, respiratory rate, pulse, axillary temperature, and pulse oximetry.
Time frame: Up to 5 years
Change From Baseline in Height Measurements
Time frame: Up to 5 years
Change From Baseline in Weight Measurements
Time frame: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Clinical Laboratory Assessments
Blood samples will be collected for hematology (including complete blood cell count) and chemistry.
Time frame: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Cardiac Assessments
Cardiac assessments will include a 12-lead electrocardiogram, transthoracic echocardiogram and Troponin-I.
Time frame: Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Observational Phase Questionnaire Results
The observational phase questionnaire includes 7 yes/no questions. Observation categories include: adverse events, hospitalizations, concomitant medications, ventilatory support and feeding support.
Time frame: Year 6 to Year 15
Number of Participants Who Experience at Least One Serious Adverse Event (SAE)
An SAE is defined as any adverse event (appearance of \[or worsening of any pre existing\]) undesirable sign(s), symptom(s), or medical conditions(s) which meets any one of the following criteria: * Fatal * Life-threatening * Results in persistent or significant disability/incapacity * Constitutes a congenital abnormality or birth defect * Requires in-patient hospitalization or prolongation of existing hospitalization * Is medically significant e.g. defined as an event that jeopardizes the participant or may require medical or surgical intervention to prevent one of the outcomes listed above
Time frame: Up to 15 years
Number of Participants Who Experience at Least One Adverse Event of Special Interest (AESI)
An AESI is defined as an AE occurring during any study phase that fulfills one of the following criteria: * Hepatotoxicity * Thrombotic microangiopathy * Cardiac adverse events * Dorsal root ganglia toxicity * New malignancies * New incidence of a neurologic disorder * New incidence of an autoimmune disorder * New incidence of hematologic disorder
Time frame: Up to 15 years
Change From Baseline in Bayley Scales of Infant and Toddler Development
Third Edition (Bayley-III) to be performed in all patients up to 42 months, 15 days of age.
Time frame: Up to 42 months, 15 days of age
Change From Baseline in Revised Upper Limb Module (RULM) Score
RULM score is based on a scale from 0 to 37 where lower scores reflect poorer upper limb functional ability.
Time frame: Up to 5 years
Change From Baseline in Cogstate Computerized Cognitive Battery Performed in Age 48 Months and Older
The Cogstate Computerized Cognitive Battery consists of the Identification Test (scored 0 (best) to 1.5708 (worst)), the International Shopping List Test (scored 0 (worst) to 999 (best)), the International Shopping List Test-Delayed Recall (scored 0 (worst) to 999 (best)), the One Card Learning Test (scored 0 (worst) to 1.5708 (best)), and the One Back Test (scored 0 (worst) to 1.5708 (best)).
Time frame: Up to 5 years
Change From Baseline in Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5) Performed in All Participants 5 to 21 Years of Age
The CELF-5 Following Directions and Sentence Repetition subtests use scoring that varies based on age, but will be administered to participants 5-21 years of age. The Following Directions subtest will be scored from 0-33 with higher score being more advanced and the Recalling Sentences subtest will be scored from 0-78 with higher score being more advanced.
Time frame: Up to 5 years
Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND)
ACEND score is based on a scale from 1 to 41 where higher scores represent a better caregiver experience
Time frame: Up to 5 years
Number of Participants With Concomitant Medications Overall and by Type of Medications
Time frame: Up to 5 years
Number of Participants With Other SMA Therapies Overall and by Type of Medications
Time frame: Year 6 to Year 15
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 www.clinicalstudydatarequest.com.
Data sharing details ↗ · clinicalstudydatarequest.com
Started Feb 2020 (actual). Primary completion is estimated for Dec 2035, 190 months after the start.
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This study is active, not recruiting, as verified in Sep 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
Spinal Muscular Atrophies of Childhood →