An observational study in Spinal Muscular Atrophy 1, sponsored by Novartis Gene Therapies. Active, not recruiting at 1 site in United States. Per ClinicalTrials.gov, last updated 2026-09-22.
Sponsored by Novartis Gene Therapies (part of Novartis) · Observational
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 10th largest by enrollment of the 10 observational ones that give a count, is the only one that started in 2017, is one of 10 observational ones, and was the 9th 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 2017.
This study's enrollment of 13 is below the median of 82 across 19 observational 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.
This tab as a report to print or save as PDF → With an account.
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:
This tab as a report to print or save as PDF → With an account.
This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years.
This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years. The last visit of the parent study or early discontinuation from the parent study may serve as the visit at which the informed consent form process is conducted for the AVXS 101-LT-001 long term follow-up safety study. Patients will return annually for follow up study visits for five (5) years, and then will be contacted via phone annually for ten (10) years. Additionally, patient record transfers from their local physician and/or neurologist will be requested in conjunction with the annual study visits and phone contacts for review by the investigator.
If the patient is unable to return to the original investigative site, the sponsor will arrange with the patients' local established physician to serve as an additional investigator to conduct the required assessments.
This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.).
Patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi will roll over from the previous study into the AVXS-101-LT-001 study for continuous safety monitoring for up to 15 years.
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:
1. Parent/legal guardian unable or unwilling to participate in the long term follow up safety study.
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.
Patients received treatment with onasemnogene abeparvovec-xioi in the parent study, AVXS-101-CL-101
Also known as: Zolgensma
Long-Term Safety
The primary objective is to collect long term safety data of patients with SMA Type 1 who were treated with onasemnogene abeparvovec-xioi in the AVXS-101-CL-101 gene replacement therapy clinical trial by assessing incidence of SAEs and Adverse Events of Special Interest
Time frame: 15 years
Plan to share: No
Started Sep 2017 (actual). Primary completion is estimated for Dec 2030, 158 months after the start.
How these figures are worked out, and which studies are left out of them.
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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Spinal Muscular Atrophies of Childhood →