NCT07805278Recruiting
A Registry of Cases of Spinal Muscular Atrophy
A Registry of Cases of Spinal Muscular Atrophy Diagnosed in the Prenatal Period That Received Disease-Modifying Therapy
Source: ClinicalTrials.gov · Last updated 22 Oct 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 30 (estimated)
- First submitted
- 13 Aug 2026
- Start date
- Nov 2026
- Primary completion date
- Aug 2029
- Lead sponsor
- St. Jude Children's Research Hospital
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT07805278
What you commit to
- Study type: Observational
- Planned participants: 30
- From Nov 2026 to the primary completion date, Aug 2029
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.
Who the study is looking for
- Current or past pregnancy during which the fetus received a genetic diagnosis of SMA.
- Current or past treatment with SMA-directed therapy intended to treat the fetus during gestation (such as risdiplam, nusinersen, onasemnogene abeparvovec, or another product intended to treat SMA).
Sex: All
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| St. Jude Children's Research Hospital Coordinator of this site? Confirm its status | Memphis | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.