A Registry of Cases of Spinal Muscular Atrophy

Part of paid clinical trials in Memphis, Tennessee.

Sponsor
St. Jude Children's Research Hospital
Study ID
NCT07805278
Status
Not Yet Recruiting

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Conditions

  • Spinal Muscular Atrophy

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Study Details

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.

Key Dates

First listed
Sep 4, 2026
Start date
Sep 30, 2026
Status verified
Sep 2026
Primary completion
Aug 31, 2029
Completion
Aug 31, 2033

Study Design

Enrollment
30 participants (estimated)

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 ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
St. Jude Children's Research HospitalMemphisTennessee38105
Richard Finkel, MD
[email protected]
888-226-4343
Richard Finkel, MD (PRINCIPAL_INVESTIGATOR)

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By research site
St. Jude Children's Research Hospital· Memphis, TN

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