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

Description

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).

Conditions

Muscular Atrophy, Spinal

Study Overview

Study Details

Study overview

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).

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

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

Condition
Muscular Atrophy, Spinal
Intervention / Treatment

-

Contacts and Locations

Cambridge

Research Site, Cambridge, Massachusetts, United States, 02142

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Participant who is currently pregnant (or was pregnant during the relevant exposure window) and enrolled in the United Kingdom (UK), United States (US), or Italy network of the ISMAR registry 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
  • * 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

Ages Eligible for Study

18 Years to 54 Years

Sexes Eligible for Study

FEMALE

Accepts Healthy Volunteers

No

Collaborators and Investigators

Biogen,

Medical Director, STUDY_DIRECTOR, Biogen

Study Record Dates

2033-10-31