Evaluating pregnancy outcomes in women with spinal muscular atrophy exposed to Spinraza

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study aims to assess pregnancy complications and outcomes in women with spinal muscular atrophy (SMA) who have been exposed to nusinersen, also known as Spinraza, during specific timeframes related to conception and pregnancy. It will compare these outcomes with those of women without SMA who were not exposed to nusinersen. The study will utilize existing SMA registries in the US, UK, and parts of Europe to gather data on both maternal and infant health outcomes. By focusing on this unique population, the study seeks to provide insights into the safety and effects of nusinersen during pregnancy.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

* 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:

* 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

Where it's running

• Stanford Neuroscience Health Center
  Palo Alto, California, United States Recruiting
• Boston Children's Hospital
  Boston, Massachusetts, United States Recruiting
• Columbia University Irving Medical Center
  New York, New York, United States Recruiting
• University College London Hospitals NHS Foundation Trust
  London, Greater London, United Kingdom Recruiting
• St George's University NHS Foundation Trust
  London, Greater London, United Kingdom Recruiting
• The Northern Care Alliance NHS Foundation Trust
  Salford, Greater Manchester, United Kingdom Recruiting

Study contacts

• Study coordinator: US Biogen Clinical Trial Center
• Email: clinicaltrials@biogen.com
• Phone: 866-633-4636

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Intrathecal Zolgensma in spinal muscular atrophy patients in Japan

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.