Actively Recruiting
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
Led by Biogen · Updated on 2026-03-09
20
Participants Needed
14
Research Sites
515 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
In this study, researchers will know more about the effects of nusinersen, also known as Spinraza®, in pregnant participants with spinal muscular atrophy, also known as SMA. This is a drug available for doctors to prescribe for people with SMA. Due to the current treatment options that exist, people with SMA may now reach the age where they can become pregnant. But, there is not enough information known yet about what the effects of nusinersen may be on pregnant people with SMA or on their babies. 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.
CONDITIONS
Official Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Currently pregnant or was pregnant during the relevant exposure window and enrolled in ISMAR-US, UK Adult SMA REACH, or SMArtCARE registry
- Genetic confirmation of spinal muscular atrophy linked to chromosome 5q (5q SMA)
- Documented exposure to nusinersen up to 14 months before last menstrual period or at any time during pregnancy
You will not qualify if you...
- Treatment with risdiplam from 2 weeks before conception or plans to receive risdiplam during pregnancy
- Treatment with risdiplam at any time from last menstrual period through pregnancy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 14 locations
1
Stanford Neuroscience Health Center
Palo Alto, California, United States, 94304
Actively Recruiting
2
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
3
Research Site
Cambridge, Massachusetts, United States, 02142
Active, Not Recruiting
4
Columbia University Irving Medical Center
New York, New York, United States, 10032
Actively Recruiting
5
University College London Hospitals NHS Foundation Trust
London, Greater London, United Kingdom, NW1 2PG
Actively Recruiting
6
St George's University NHS Foundation Trust
London, Greater London, United Kingdom, SW17 0QT
Actively Recruiting
7
The Northern Care Alliance NHS Foundation Trust
Salford, Greater Manchester, United Kingdom, M6 8HD
Actively Recruiting
8
University Hospital Southampton NHS Foundation Trust
Southampton, Hampshire, United Kingdom, SO16 6YD
Actively Recruiting
9
Nottingham University Hospitals Trust
Nottingham, Nottinghamshire, United Kingdom, NG5 1PB
Actively Recruiting
10
The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust
Oswestry, Shropshire, United Kingdom, SY10 7AG
Actively Recruiting
11
Sheffield Teaching Hospitals NHS Foundation Trust
Sheffield, South Yorkshire, United Kingdom, S10 2SB
Actively Recruiting
12
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, Tyne and Wear, United Kingdom, NE7 7ND
Actively Recruiting
13
University Hospitals Birmingham NHS Foundation Trust
Birmingham, West Midlands, United Kingdom, B15 2GW
Actively Recruiting
14
North Bristol NHS Trust
Bristol, United Kingdom, BS10 5NB
Actively Recruiting
Research Team
U
US Biogen Clinical Trial Center
CONTACT
G
Global Biogen Clinical Trial Center
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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