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

Launched by BIOGEN · Mar 16, 2023

Keywords

ClinConnect Summary

This clinical trial is studying the effects of a medication called Spinraza (nusinersen) in pregnant women who have a condition known as spinal muscular atrophy (SMA). The main goal is to understand any complications or outcomes related to pregnancy in women with SMA who have taken nusinersen before or during their pregnancy. Researchers want to compare these outcomes with those of women without SMA who did not take nusinersen, helping to gather important safety information for both mothers and their babies.

To be eligible for the study, participants must currently be pregnant or have been pregnant during a specific time frame and must be enrolled in certain SMA registries in the UK, US, or parts of Europe. They also need to have a confirmed diagnosis of SMA and a history of taking nusinersen. Participants can expect to be part of a supportive research environment where their experiences and health will be carefully monitored, contributing valuable knowledge to help others in the future. It’s important to note that women who have used a different SMA medication called risdiplam during their pregnancy will not be included in this study.

Gender

FEMALE

Eligibility 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