Muscular Atrophy, SpinalNCT05789758
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
An observational study of Muscular Atrophy, Spinal, run by Biogen at 14 sites in 2 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 54 years, women only
- Conditions the study is for
- Muscular Atrophy, Spinal
- How many people
- 20 participants
- Where
- 14 sitesin 2 countries
- Study dates
- 15 Dec 2023to 31 Oct 2033
- How long will I take part?
- 2 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 54 years
- Women only
- No healthy volunteers
- Adult
Study population: Pregnant participants with SMA who are exposed to nusinersen from the UK Adult SMA REACH, ISMAR - US, and SMArtCARE registries will be enrolled to obtain information on effects of nusinersen on pregnancy complications and outcomes.
Who may be able to join
- Participant who is currently pregnant (or was pregnant during the relevant exposure window) and enrolled in the United States (US) network of the ISMAR registry, the United Kingdom (UK) Adult SMA REACH, or the Germany, Austria, or Switzerland network of the SMArtCARE registry
- 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
Who cannot join
- Treatment with risdiplam at any time from the first day of their LMP, 2 weeks prior to the date of conception (approximately 5 half-lives), and/or plans to receive treatment with risdiplam during pregnancy
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Trial locations and contacts
14 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
Global Biogen Clinical Trial Center contact
14 sites in 2 countries
United Kingdom10 sites, 10 recruiting
- Recruiting
University Hospitals Birmingham NHS Foundation Trust
Birmingham, West Midlands, B15 2GW
(0121) 432 3232
Nicholas Davies
- Recruiting
North Bristol NHS Trust
Bristol, BS10 5NB
0117 950 5050
Andria Merrison
- Recruiting
University College London Hospitals NHS Foundation Trust
London, Greater London, NW1 2PG
020 3456 7890
Matt Parton
- Recruiting
St George's University NHS Foundation Trust
London, Greater London, SW17 0QT
020 8672 1255
Clare Galtrey
- Recruiting
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, Tyne and Wear, NE7 7ND
0191 233 6161
Chiara Marini-Bettolo
- Recruiting
Nottingham University Hospitals Trust
Nottingham, Nottinghamshire, NG5 1PB
0115 969 1169
Saam Sedehizadeh
- Recruiting
The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust
Oswestry, Shropshire, SY10 7AG
01691 404000
Tracey Willis
- Recruiting
The Northern Care Alliance NHS Foundation Trust
Salford, Greater Manchester, M6 8HD
0161 789 7373
James Lilleker
- Recruiting
Sheffield Teaching Hospitals NHS Foundation Trust
Sheffield, South Yorkshire, S10 2SB
0114 271 7000
Channa Hewamadduma
- Recruiting
University Hospital Southampton NHS Foundation Trust
Southampton, Hampshire, SO16 6YD
023 8077 7222
Annamaria Kiss-csenki
United States4 sites, 3 recruiting
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02115
617-355-8036
Basil T. Darras
- Active, not recruiting
Research Site
Cambridge, Massachusetts, 02142
- Recruiting
Columbia University Irving Medical Center
New York, New York, 10032
212-305-1319
Michio Hirano
- Recruiting
Stanford Neuroscience Health Center
Palo Alto, California, 94304
650-725-7622
John W. Day
About this study
Official title: Spinraza (Nusinersen) SMA Pregnancy Exposure Study Within Existing SMA Registries
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:
Read more
- 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.
Read the detailed description
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).
- Conditions
- Muscular Atrophy, Spinal
- Being tested
- No Intervention
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
Administered as specified in the treatment arm.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Pregnancy Terminations
Pregnancy termination is defined as any induced or voluntary fetal loss during pregnancy.
Measured: Up to 10 years
Number of Spontaneous Abortions
Spontaneous abortion is defined as any loss of a fetus due to natural causes at <20 weeks of gestation.
Measured: Up to 10 years
Number of Fetal Deaths
Fetal deaths include early fetal loss which is fetal death occurring at ≥20 weeks but <28 weeks of gestation and late fetal loss which is fetal death occurring at ≥28 weeks of gestation.
Measured: Up to 10 years
Number of Live Births
Live births include premature births which is defined as delivery at <37 weeks of gestation, full term birth which is defined as delivery at ≥37 weeks of gestation.
Measured: Up to 10 years
Number of Neonatal, Perinatal, and Infant Deaths
Neonatal death is defined as death occurring in a newborn prior to 28 days after birth. Perinatal death is defined as death occurring at or after 28 days and prior to 12 weeks after birth. Infant death is defined as death occurring between 12 and 52 weeks after birth, inclusive.
Measured: Up to 10 years
Number of Major Congenital Malformations (MCMs)
MCMs (interchangeably referred to as congenital malformations, congenital anomalies, and birth defects) are abnormalities in structural development that are medically or cosmetically significant, are present at birth, and persist in postnatal life unless or until repaired.
Measured: Up to 10 years
Number of Infants Small for Gestational Age Birth
Birth weight will be classified as small for gestational age (<10th percentile), appropriate (10th-90th percentile), or large (>90th percentile).
Measured: Up to 10 years
Number of Ectopic and Molar Pregnancies
Ectopic pregnancy is defined as a pregnancy that occurs outside of the uterine cavity. Molar pregnancy is defined as genetically abnormal conceptions characterized by abnormal chorionic villi, trophoblastic hyperplasia, poor fetal development, and an increased risk of malignant disease development.
Measured: Up to 10 years
Number of Maternal Deaths
Maternal deaths during pregnancy, labor, delivery, or up to 12 weeks after delivery will be reported.
Measured: Up to 10 years
Number of Infants With Abnormal Postnatal Growth and Development and Neurobehavioral Impairment
Abnormal postnatal growth and development and neurobehavioral impairment in infants up to 24 months of age will be reported.
Measured: Up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2023
- Study start15 Dec 2023
- Primary completion31 Oct 2032 (estimated)
- Study completion31 Oct 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Atrophy, SpinalStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 20 participants (estimated)
- Target duration
- 2 Years
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05789758
- Sponsor's protocol ID
- CH-SPN-12180
- Other IDs
- 232SM405
- Status last verified
- Mar 2026
- First submitted
- 16 Mar 2023
- Last updated
- 9 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Spinraza (Nusinersen) SMA Pregnancy Exposure Study Within Existing SMA Registries
- ClinicalTrials.gov ID
- NCT05789758
- Acronym / sponsor ID
- CH-SPN-12180
- Conditions
- Muscular Atrophy, Spinal
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 15 Dec 2023 (actual)
- Primary completion
- 31 Oct 2032 (estimated)
- Study completion
- 31 Oct 2033 (estimated)
- First posted
- 29 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 2 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Nusinersen Treated Participants | – | Other: No InterventionPregnant 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. |
Who may qualify
- Ages
- 18 to 54 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- Pregnant participants with SMA who are exposed to nusinersen from the UK Adult SMA REACH, ISMAR - US, and SMArtCARE registries will be enrolled to obtain information on effects of nusinersen on pregnancy complications and outcomes.
Key inclusion criteria (3 listed)
- Participant who is currently pregnant (or was pregnant during the relevant exposure window) and enrolled in the United States (US) network of the ISMAR registry, the United Kingdom (UK) Adult SMA REACH, or the Germany, Austria, or Switzerland network of the SMArtCARE registry
- 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
Key exclusion criteria (2 listed)
- Treatment with risdiplam at any time from the first day of their LMP, 2 weeks prior to the date of conception (approximately 5 half-lives), and/or plans to receive treatment with risdiplam during pregnancy
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No InterventionAdministered as specified in the treatment arm. | Other | Nusinersen Treated Participants |
Primary outcome
Number of Pregnancy Terminations
Time frame: Up to 10 years
Pregnancy termination is defined as any induced or voluntary fetal loss during pregnancy.
Number of Spontaneous Abortions
Time frame: Up to 10 years
Spontaneous abortion is defined as any loss of a fetus due to natural causes at <20 weeks of gestation.
Number of Fetal Deaths
Time frame: Up to 10 years
Fetal deaths include early fetal loss which is fetal death occurring at ≥20 weeks but <28 weeks of gestation and late fetal loss which is fetal death occurring at ≥28 weeks of gestation.
Number of Live Births
Time frame: Up to 10 years
Live births include premature births which is defined as delivery at <37 weeks of gestation, full term birth which is defined as delivery at ≥37 weeks of gestation.
Number of Neonatal, Perinatal, and Infant Deaths
Time frame: Up to 10 years
Neonatal death is defined as death occurring in a newborn prior to 28 days after birth. Perinatal death is defined as death occurring at or after 28 days and prior to 12 weeks after birth. Infant death is defined as death occurring between 12 and 52 weeks after birth, inclusive.
Number of Major Congenital Malformations (MCMs)
Time frame: Up to 10 years
MCMs (interchangeably referred to as congenital malformations, congenital anomalies, and birth defects) are abnormalities in structural development that are medically or cosmetically significant, are present at birth, and persist in postnatal life unless or until repaired.
Number of Infants Small for Gestational Age Birth
Time frame: Up to 10 years
Birth weight will be classified as small for gestational age (<10th percentile), appropriate (10th-90th percentile), or large (>90th percentile).
Number of Ectopic and Molar Pregnancies
Time frame: Up to 10 years
Ectopic pregnancy is defined as a pregnancy that occurs outside of the uterine cavity. Molar pregnancy is defined as genetically abnormal conceptions characterized by abnormal chorionic villi, trophoblastic hyperplasia, poor fetal development, and an increased risk of malignant disease development.
Number of Maternal Deaths
Time frame: Up to 10 years
Maternal deaths during pregnancy, labor, delivery, or up to 12 weeks after delivery will be reported.
Number of Infants With Abnormal Postnatal Growth and Development and Neurobehavioral Impairment
Time frame: Up to 10 years
Abnormal postnatal growth and development and neurobehavioral impairment in infants up to 24 months of age will be reported.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2026.
- Individual participant dataSharing planned: Yes. In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Related trials for Muscular Atrophy, Spinal
RecruitingPhase 4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Muscular Atrophy, Spinal
Enrolling by invitationPhase 1/2
Gene Therapy for IGHMBP2-Related Diseases
SMARD1, CMT2S
Not yet recruitingEarly Phase 1
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
Spinal Muscular Atrophy (SMA)
RecruitingPhase 4
ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)
Spinal Muscular Atrophy
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Global Biogen Clinical Trial Center contact
Medical Director study director
Biogen
14 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Muscular Atrophy, Spinal, run by Biogen at 14 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 9 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United Kingdom, United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact US Biogen Clinical Trial Center (866-633-4636, clinicaltrials@biogen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Pregnancy Terminations (time frame: Up to 10 years). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Mar 2026.