A Study of Spinraza (Nusinersen) Exposure in Pregnant Women With Spinal Muscular Atrophy (SMA) Within Existing SMA Registries

Biogen

Status: Enrolling

Conditions: Muscular Atrophy, Spinal

Treatments: Other: No Intervention

Study type: Observational

Funder types: Industry

Identifiers

NCT05789758

232SM405 (Other Identifier)

CH-SPN-12180

Details and patient eligibility

About

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).

Enrollment: 20 estimated patients

Sex: Female

Ages: 18 to 54 years old

Volunteers: No Healthy Volunteers

Inclusion and 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

Trial design

20 participants in 1 patient group

Nusinersen Treated Participants

Description:

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.

Treatment:

Other: No Intervention