Pregnancy-time treatment of fetal SMA: a registry of mother-child outcomes

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a longitudinal registry that follows up to 30 mother-child pairs where a pregnant person used a therapy to treat a fetus diagnosed with SMA. The study will collect medical records, interviews, and surveys to describe maternal-fetal monitoring, delivery outcomes, and postnatal health after prenatal treatment. Data are gathered retrospectively and prospectively over a seven-year period, with enrollment over three years and at least four years of follow-up. Outcomes will inform best practices for monitoring and supporting pregnancies affected by SMA treated prenatally. The study is led by St. Jude Children’s Research Hospital and collects data via REDCap.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 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).

Exclusion Criteria:

* Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.

Where it's running

• St. Jude Children's Research Hospital
  Memphis, Tennessee, United States Recruiting

Study contacts

• Principal investigator: Richard Finkel, MD — St. Jude Children's Research Hospital
• Study coordinator: Richard Finkel, MD
• Email: referralinfo@stjude.org
• Phone: 888-226-4343

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.