A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies
NCT ID: NCT05789758
Last Updated: 2025-10-01
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
20 participants
OBSERVATIONAL
2023-12-15
2033-10-31
Brief Summary
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This is known as an "observational" study, which collects health information about study participants without changing their medical care. The pregnant participants for this study will be found using 3 different groups of SMA study research centers:
* ISMAR-US (International SMA Registry in the United States)
* UK Adult SMA-REACH (Adult SMA Research and Clinical Hub in the United Kingdom)
* SMArtCARE (Austria, Germany, and Switzerland)
The main goal of this study is to collect birth and health information from 3 groups of participants and their babies. These groups are:
* Those who received nusinersen 14 months before the first day of their last period before getting pregnant
* Those who received nusinersen 14.5 months before the day they got pregnant
* Those who received nusinersen during any time in their pregnancy
The main questions researchers want to learn about in this study are:
* Loss of pregnancy overall
* Loss of pregnancy before the baby was 20 weeks old
* Loss of pregnancy after the baby becomes 20 weeks old
* Live births
* Loss of the baby after birth
* Babies who have problems in their body that develop during pregnancy
* Babies who are small for their age while in the participant's uterus
* Pregnancy that happens outside of the uterus
* How many participants die during pregnancy, while the baby is being born, and up to 12 weeks after delivering the baby
* Babies who develop problems in their body after birth
Researchers will also compare this information to people without SMA who have not received nusinersen.
This study will be done as follows:
* Information will start being collected when the participant decides to join the study.
* Participants will be contacted at each trimester (about every 3 months) to learn about their health and pregnancy.
* Participants' doctors will be contacted at each trimester, when the participants are about 6 or 7 months pregnant, and about 4 weeks after the delivery of the baby.
* The babies' doctors will be contacted when the baby is 1, 2, 6, 12, 18, and 24 months old.
* Each participant will be in the study until the end of their pregnancy and for up to 12 weeks after delivery. Each baby will be in the study for up to 2 years after birth.
* The study overall will last at least 10 years from when the first participant joins the study.
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Nusinersen Treated Participants
Pregnant participants with SMA who are exposed to nusinersen during the relevant window defined as 14 months prior to the first day of the participant's last menstrual period before conception, 14.5 months before conception and anytime during pregnancy and are enrolled in the registries, International Spinal Muscular Atrophy Registry (ISMAR), Adult SMA REACH and SMArtCARE will be followed prospectively up to 3 months post-delivery, the infants will be followed up to 2 years post-delivery and the available data is collected retrospectively.
No Intervention
Administered as specified in the treatment arm.
Interventions
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No Intervention
Administered as specified in the treatment arm.
Eligibility Criteria
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Inclusion Criteria
* Genetic documentation of spinal muscular atrophy linked to chromosome 5q (5q SMA)
* Documentation that the participant was exposed to nusinersen up to 14 months prior to the first day of their LMP before conception, 14.5 months before conception, and/or at any time during their pregnancy
Exclusion Criteria
18 Years
54 Years
FEMALE
No
Sponsors
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Biogen
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Biogen
Locations
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Stanford Neuroscience Health Center
Palo Alto, California, United States
Research Site
Cambridge, Massachusetts, United States
St George's University NHS Foundation Trust
London, Greater London, United Kingdom
The Northern Care Alliance NHS Foundation Trust
Salford, Greater Manchester, United Kingdom
University Hospital Southampton NHS Foundation Trust
Southampton, Hampshire, United Kingdom
Nottingham University Hospitals Trust
Nottingham, Nottinghamshire, United Kingdom
The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust
Oswestry, Shropshire, United Kingdom
Sheffield Teaching Hospitals NHS Foundation Trust
Sheffield, South Yorkshire, United Kingdom
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, Tyne and Wear, United Kingdom
University Hospitals Birmingham NHS Foundation Trust
Birmingham, West Midlands, United Kingdom
North Bristol NHS Trust
Bristol, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Role: primary
Other Identifiers
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232SM405
Identifier Type: OTHER
Identifier Source: secondary_id
CH-SPN-12180
Identifier Type: -
Identifier Source: org_study_id
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