Recruiting

SMA with Medication

Sponsor:

St. Jude Children's Research Hospital

Code:

NCT07805278

Conditions

Spinal Muscular Atrophy

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Study Details

Brief summary:

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.

Conditions

Spinal Muscular Atrophy

Study ID

NCT07805278

Start date

Sep, 2026

Status verified date

Sep, 2026

Completion date

Aug, 2033

Anticipated

Primary completion date

Aug, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

30 participants

Anticipated

Interventions and Outcome Measures

Primary outcome measure

  • Maternal-fetal monitoring outcomes from instances of healthy women dosed with risdiplam to treat their fetus diagnosed with Spinal Muscular Atrophy (SMA) [ 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 [ Time Frame: At enrollment and shortly after delivery ]
  • Delivery outcomes associated with prenatally treated SMA [ Time Frame: At delivery ]

Central Contacts and Locations

Central contacts

Richard Finkel, MD

888-226-4343referralinfo@stjude.org

Locations

St. Jude Children's Research Hospital

Recruiting

Memphis, Tennessee, United States, 38105

Contacts

Richard Finkel, MD

888-226-4343referralinfo@stjude.org

Principal Investigator:

Richard Finkel, MD

More Information

Sponsor

St. Jude Children's Research Hospital

Last update posted

Sep 8, 2026

Last verified

Sep, 2026

Keywords

  • Spinal Muscular Atrophy (SMA)
  • Registry Study
  • Fetuses Diagnosed with SMA
  • Current or Past Pregnancy
  • SMA Treatment During Gestation
  • Risdiplam
  • Nusinersen
  • Onasemnogene Abeparvovec
  • Prenatal SMA
  • Fetal SMA
  • Prenatal risdiplam
  • Fetal risdiplam

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by St. Jude Children's Research Hospital on 2026-09-08.