An observational study in Spinal Muscular Atrophy, sponsored by St. Jude Children's Research Hospital. Recruiting at 1 site in United States. Per ClinicalTrials.gov, last updated 2026-10-22.
Sponsored by St. Jude Children's Research Hospital · Observational
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 32nd largest by planned enrollment of the 43 observational ones that give a count, is one of 8 that started in 2026, is one of 43 observational ones, reaches primary completion later than 28 of the 38 other live ones, and was the 121st 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
Biogen · Sites in United States
Biogen · Sites in United States, China
Novartis Pharmaceuticals · Sites in United States
Biogen · Sites in United States, Australia, Brazil and 4 more countries
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: all sexes.
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. 8 started in 2026.
This study's planned enrollment of 30 is below the median of 139 across 114 observational 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.
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61 of 121 have an industry lead sponsor. The 10 most frequent:
St. Jude Children's Research Hospital is the lead sponsor of 440 studies on the registry; 102 are open to participants now.
Of its 61 completed or terminated interventional studies of FDA-regulated products, 37 (61%) have results posted.
St. Jude Children's Research Hospital has 2 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:
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Because no long-term record of prenatal Spinal Muscular Atrophy (SMA) treatment exists, this study will address an important gap in the understanding of the safety and benefit of treatment while in the womb. The registry will help guide best practices for monitoring pregnant women and their fetus with SMA, if treated with a medication while in the womb, and to follow the baby after birth for signs of safety and added benefit birth and support future research and study development.
Primary Objectives
Secondary Objectives
This is a longitudinal retrospective/prospective registry study with the expected sample size of 30 mother-child dyads. The study will collect data elements from cases of fetuses diagnosed with SMA in utero and where their mothers self-administered a disease-modifying therapy (DMT) to treat the affected fetus.
Information will be collected from the participant medical records, interviews, and surveys or questionnaires. Survey information will be collected at enrollment, shortly after delivery, and then on 6-month intervals starting 6 months after delivery. The questionnaires will be sent via email through an application called REDCap and may be completed by the participant (or someone helping them) directly or by interview with the support of a research staff member (remotely).
The total study duration is 7 years, consisting of a 3-year accrual period and minimum 4-year follow-up period.
Participants who meet the eligibility criteria and consent to participate.
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.
Maternal-fetal monitoring outcomes from instances of healthy women dosed with risdiplam to treat their fetus diagnosed with Spinal Muscular Atrophy (SMA)
Maternal-fetal monitoring outcome endpoints will be obtained from questionnaires and medical chart review. Analyses will be conducted using SAS software and will be summarized using descriptive statistics.
Time frame: At enrollment and shortly after delivery
Pregnancy outcomes from instances of healthy women dosed with risdiplam to treat their fetus diagnosed with SMA
Pregnancy outcome endpoints will be obtained from questionnaires and medical chart review. Analyses will be conducted using SAS software and will be summarized using descriptive statistics.
Time frame: At enrollment and shortly after delivery
Delivery outcomes associated with prenatally treated SMA
Delivery endpoints will be obtained from questionnaires and medical chart review. Analyses will be conducted using SAS software and will be summarized using descriptive statistics.
Time frame: At delivery
Start is estimated for Nov 2026; primary completion for Aug 2029, 33 months later.
Of 58 observational studies under Muscular Atrophy, Spinal, half reached primary completion within 88 months of starting (the median), counting those still running; a quarter within 36. This study's planned 33 months is below that.
Start moved twice since we started tracking on Sep 25, 2026: Sep 2026 → Nov 2026, last on Oct 22, 2026.
Earlier changes are on the record history on ClinicalTrials.gov ↗
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
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