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

Muscular Atrophy, Spinal Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05789758
• Other study ID #: CH-SPN-12180
• Secondary ID: 232SM405
• Status: Recruiting
• Start date: December 15, 2023
• Est. completion date: October 31, 2033

Study information

• Verified date: January 2024
• Source: Biogen
• Contact: US Biogen Clinical Trial Center
• Phone: 866-633-4636
• Email: clinicaltrials[@]biogen.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: October 31, 2033
• Est. primary completion date: October 31, 2032
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 18 Years to 54 Years

Eligibility

Key Inclusion 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

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

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal

Intervention

Other:
• No Intervention
Administered as specified in the treatment arm.

Locations

Country	Name	City	State
United States	Research Site	Cambridge	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Biogen

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Pregnancy Terminations	Pregnancy termination is defined as any induced or voluntary fetal loss during pregnancy.	Up to 10 years
Primary	Number of Spontaneous Abortions	Spontaneous abortion is defined as any loss of a fetus due to natural causes at <20 weeks of gestation.	Up to 10 years
Primary	Number of Fetal Deaths	Fetal deaths include early fetal loss which is fetal death occurring at =20 weeks but <28 weeks of gestation and late fetal loss which is fetal death occurring at =28 weeks of gestation.	Up to 10 years
Primary	Number of Live Births	Live births include premature births which is defined as delivery at <37 weeks of gestation, full term birth which is defined as delivery at =37 weeks of gestation.	Up to 10 years
Primary	Number of Neonatal, Perinatal, and Infant Deaths	Neonatal death is defined as death occurring in a newborn prior to 28 days after birth. Perinatal death is defined as death occurring at or after 28 days and prior to 12 weeks after birth. Infant death is defined as death occurring between 12 and 52 weeks after birth, inclusive.	Up to 10 years
Primary	Number of Major Congenital Malformations (MCMs)	MCMs (interchangeably referred to as congenital malformations, congenital anomalies, and birth defects) are abnormalities in structural development that are medically or cosmetically significant, are present at birth, and persist in postnatal life unless or until repaired.	Up to 10 years
Primary	Number of Infants Small for Gestational Age Birth	Birth weight will be classified as small for gestational age (<10th percentile), appropriate (10th-90th percentile), or large (>90th percentile).	Up to 10 years
Primary	Number of Ectopic and Molar Pregnancies	Ectopic pregnancy is defined as a pregnancy that occurs outside of the uterine cavity. Molar pregnancy is defined as genetically abnormal conceptions characterized by abnormal chorionic villi, trophoblastic hyperplasia, poor fetal development, and an increased risk of malignant disease development.	Up to 10 years
Primary	Number of Maternal Deaths	Maternal deaths during pregnancy, labor, delivery, or up to 12 weeks after delivery will be reported.	Up to 10 years
Primary	Number of Infants With Abnormal Postnatal Growth and Development and Neurobehavioral Impairment	Abnormal postnatal growth and development and neurobehavioral impairment in infants up to 24 months of age will be reported.	Up to 10 years