At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- No Intervention (other)
- How long the study runs
- Study runs about 118 months (dates as stated)
- About the drug or intervention
- No Intervention — other: Administered as specified in the treatment arm.
- Patient visit burden
- Not specified by the sponsor
In plain English
This study looks at how nusinersen (Spinraza) affects people with spinal muscular atrophy (SMA) who took it before or during pregnancy, and the health of their babies. Spinal muscular atrophy is a genetic condition linked to chromosome 5q that affects the muscles. The study is funded by Biogen.
Who can take part
- You are pregnant (or were pregnant during the relevant time) and are in a registry: the US ISMAR registry, the UK Adult SMA REACH registry, or the SMArtCARE registry (Germany, Austria or Switzerland)
- You have genetic test records showing spinal muscular atrophy linked to chromosome 5q
- Records show you had nusinersen up to 14 months before your last menstrual period before conception, 14.5 months before conception, and/or at any time during pregnancy
Who may not be able to
- You had risdiplam treatment from the first day of your last menstrual period or 2 weeks before conception, or plan to have risdiplam during pregnancy
- Other rules may also apply — ask the trial team
What taking part involves
- • Not stated — ask the trial team
Time commitment: Not stated — ask the trial team about visits, how long the study lasts, and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Observing health over time
- Ages
- 18 Years to 54 Years
- Who
- Female
- Number of participants
- 20
- Started
- 2023-12-15
- Last checked
- 2026-03
Plain English Summary
What is this study?
- • Testing a new treatment for muscular atrophy, spinal
- • Clinical study - 20 participants
- • 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
Who can take part?
- • Ages 18 Years to 54 Years
- • Diagnosed with muscular atrophy, spinal
- • Female only
Where?
- • London - University College London Hospitals NHS Foundation Trust
- • London - St George's University NHS Foundation Trust
- • Salford - The Northern Care Alliance NHS Foundation Trust
- • Southampton - University Hospital Southampton NHS Foundation Trust
- • +6 more UK sites
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
More detail
The primary objectives of the study are to prospectively evaluate pregnancy complications and outcomes in participants with SMA, birth outcomes and adverse effects in infants born to participants with SMA, who were exposed to nusinersen up to 14 months prior to the first day of their last menstrual period (LMP) before conception, 14.5 months before the date of conception, and/or at any time during their pregnancy. The secondary objective of the study is to evaluate pregnancy outcomes in participants with SMA exposed to nusinersen as compared with participants without SMA who were not exposed to nusinersen (e.g., participants from external, general population comparators).
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 18 Years - 54 Years
- Who can join: Female only
What the study is looking for
- ✓Genetic documentation of spinal muscular atrophy linked to chromosome 5q (5q SMA)
Who cannot take part
- ✗NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
See the full criteria
Where Is This Study? (10 UK sites)
University College London Hospitals NHS Foundation Trust
London NW1 2PG, United Kingdom
St George's University NHS Foundation Trust
London SW17 0QT, United Kingdom
The Northern Care Alliance NHS Foundation Trust
Salford M6 8HD, United Kingdom
University Hospital Southampton NHS Foundation Trust
Southampton SO16 6YD, United Kingdom
Nottingham University Hospitals Trust
Nottingham NG5 1PB, United Kingdom
The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust
Oswestry SY10 7AG, United Kingdom
Sheffield Teaching Hospitals NHS Foundation Trust
Sheffield S10 2SB, United Kingdom
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne NE7 7ND, United Kingdom
University Hospitals Birmingham NHS Foundation Trust
Birmingham B15 2GW, United Kingdom
North Bristol NHS Trust
Bristol BS10 5NB, United Kingdom
How to Get in Touch
US Biogen Clinical Trial Center
Sponsor contactCONTACT
Global Biogen Clinical Trial Center
Sponsor contactCONTACT