A Registry of Cases of Spinal Muscular Atrophy
ConditionSpinal Muscular Atrophy
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorSt. Jude Children's Research Hospital
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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).
Disqualifiers
Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.
Trial population
Participants who meet the eligibility criteria and consent to participate.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
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 delivery2
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 delivery3
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 deliverySponsors and contacts
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Source ClinicalTrials.gov