A clinical trial aims to evaluate nusinersen therapy among pregnant women with spinal muscular atrophy (SMA).
The upcoming clinical trial sponsored by Biogen Inc began on April 15, 2023. The trial aims to prospectively assess pregnancy complications and outcomes in patients with SMA. The investigators will also look at birth outcomes and adverse effects in infants born to patients with SMA who were exposed to nusinersen up to 14 months before the first day of their last menstrual period (LMP) before conception, at any point in the pregnancy, or 14.5 months before the conception date.
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The study also aims to assess pregnancy outcomes in patients with SMA exposed to nusinersen as compared to patients without SMA and not exposed to nusinersen.
According to the study, a total of 20 participants will be evaluated for a follow-up period of 2 years. Study participants need to be pregnant females 18 to 54 years of age and enrolled in the United Kingdom, United States, or Italy network of the ISMAR registry or the Germany, Austria, or Switzerland network of the SMArtCARE registry.
For data analysis, the investigators will prospectively follow SMArtCARE and International Spinal Muscular Atrophy Registry (ISMAR) up to 3 months postdelivery. Then, the infants will be evaluated for up to 2 years postdelivery, and the available information will be retrospectively collected from these sources.
The primary outcomes of the study are reported to be the number of spontaneous abortions, number of pregnancy terminations, number of fetal deaths, number of neonatal, perinatal, and infant deaths, number of ectopic and molar pregnancies, number of major congenital malformations, number of live births, number of infants small for gestational age birth, number of maternal deaths, and number of infants with abnormal postnatal growth and development and neurobehavioral impairment.
The trial is expected to be completed by October 31, 2033.
Reference
A study of Spinraza (Nusinersen) exposure in pregnant women with spinal muscular atrophy (SMA) within existing SMA registries. ClinicalTrials.gov. March 29, 2023. Accessed April 18, 2023.
