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

NCT05789758 | Recruiting FDA Drug

• 2 other identifiers: CH-SPN-12180232SM405
• Study Type: observational
• Target: 20
• Locations: 2 countries
• Sites: 14

Brief 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. 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

Muscular Atrophy, Spinal

Outcome Measures

Primary Outcomes (10)

• Number of Pregnancy Terminations

  Pregnancy termination is defined as any induced or voluntary fetal loss during pregnancy.

  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.

  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.

  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.

  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.

  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.

  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).

  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.

  Up to 10 years

• Number of Maternal Deaths

  Maternal deaths during pregnancy, labor, delivery, or up to 12 weeks after delivery will be reported.

  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.

  Up to 10 years

Study Arms (1)

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.

Other: No Intervention

Interventions

No Intervention OTHER

Administered as specified in the treatment arm.

Nusinersen Treated Participants

Eligibility Criteria

• Age: 18 Years - 54 Years
• Healthy Volunteers: No
• Age Groups: Adult (18-64)
• Sampling Method: Non-Probability Sample

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.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Biogen: lead

Study Sites (14)

Stanford Neuroscience Health Center

Palo Alto, California, 94304, United States

RECRUITING

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

Research Site

Cambridge, Massachusetts, 02142, United States

ACTIVE NOT RECRUITING

Columbia University Irving Medical Center

New York, New York, 10032, United States

RECRUITING

University College London Hospitals NHS Foundation Trust

London, Greater London, NW1 2PG, United Kingdom

RECRUITING

St George's University NHS Foundation Trust

London, Greater London, SW17 0QT, United Kingdom

RECRUITING

The Northern Care Alliance NHS Foundation Trust

Salford, Greater Manchester, M6 8HD, United Kingdom

RECRUITING

University Hospital Southampton NHS Foundation Trust

Southampton, Hampshire, SO16 6YD, United Kingdom

RECRUITING

Nottingham University Hospitals Trust

Nottingham, Nottinghamshire, NG5 1PB, United Kingdom

RECRUITING

The Robert Jones and Agnes Hunt Orthopaedic Hospital NHS Foundation Trust

Oswestry, Shropshire, SY10 7AG, United Kingdom

RECRUITING

Sheffield Teaching Hospitals NHS Foundation Trust

Sheffield, South Yorkshire, S10 2SB, United Kingdom

RECRUITING

The Newcastle upon Tyne Hospitals NHS Foundation Trust

Newcastle upon Tyne, Tyne and Wear, NE7 7ND, United Kingdom

RECRUITING

University Hospitals Birmingham NHS Foundation Trust

Birmingham, West Midlands, B15 2GW, United Kingdom

RECRUITING

North Bristol NHS Trust

Bristol, BS10 5NB, United Kingdom

RECRUITING

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Study Officials

• Medical Director

  Biogen

  STUDY DIRECTOR

Central Study Contacts

US Biogen Clinical Trial Center

CONTACT

866-633-4636; clinicaltrials@biogen.com

Global Biogen Clinical Trial Center

CONTACT

clinicaltrials@biogen.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 2 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 16, 2023
• First Posted: March 29, 2023
• Study Start: December 15, 2023
• Primary Completion (Estimated): October 31, 2032
• Study Completion (Estimated): October 31, 2033
• Last Updated: March 9, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share

Locations

US (4); GB (10)