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

Spinraza (Nusinersen) SMA Pregnancy Exposure Study Within Existing SMA Registries

Observational Biogen · NCT05789758

This study is trying to see how pregnancy goes for women with spinal muscular atrophy who have used Spinraza, compared to women without the condition who haven't used the drug.

Quick facts

Study typeObservational
Enrollment20 (estimated)
Ages18 Years to 54 Years
SexFemale
SponsorBiogen Industry-sponsored
Locations14 sites (Palo Alto, California and 13 other locations)
Trial IDNCT05789758 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates include pregnant women with genetically documented spinal muscular atrophy linked to chromosome 5q who have been exposed to nusinersen within the specified timeframes.

Not a fit: Patients who are not pregnant or those who have not been exposed to nusinersen during the relevant periods will not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide critical information on the safety of nusinersen exposure during pregnancy, guiding treatment decisions for women with SMA.

How similar studies have performed: While there may be studies on SMA treatments, this specific focus on nusinersen exposure during pregnancy is novel and has not been extensively tested.

Eligibility criteria

Show full inclusion / exclusion criteria 
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 this trial is running

Palo Alto, California and 13 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Muscular Atrophy, Spinal
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.