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

Part of paid clinical trials in Palo Alto, California.

Sponsor
Biogen
Study ID
NCT05789758
Status
Recruiting
Apply to this trial

Conditions

  • Muscular Atrophy, Spinal

Eligibility Criteria

Sex
FEMALE
Age
18 Years - 54 Years
Healthy Volunteers
Not accepted

Interventions

  • No Intervention — OTHER
    Administered as specified in the treatment arm.

Study Details

In this study, researchers will know more about the effects of nusinersen, also known as Spinraza®, in pregnant participants with spinal muscular atrophy, also known as SMA. This is a drug available for doctors to prescribe for people with SMA. Due to the current treatment options that exist, people with SMA may now reach the age where they can become pregnant. But, there is not enough information known yet about what the effects of nusinersen may be on pregnant people with SMA or on their babies. This is known as an "observational" study, which collects health information about study participants without changing their medical care. The pregnant participants for this study will be found using 3 different groups of SMA study research centers: * ISMAR-US (International SMA Registry in the United States) * UK Adult SMA-REACH (Adult SMA Research and Clinical Hub in the United Kingdom) * SMArtCARE (Austria, Germany, and Switzerland) The main goal of this study is to collect birth and health information from 3 groups of participants and their babies. These groups are: * Those who received nusinersen 14 months before the first day of their last period before getting pregnant * Those who received nusinersen 14.5 months before the day they got pregnant * Those who received nusinersen during any time in their pregnancy The main questions researchers want to learn about in this study are: * Loss of pregnancy overall * Loss of pregnancy before the baby was 20 weeks old * Loss of pregnancy after the baby becomes 20 weeks old * Live births * Loss of the baby after birth * Babies who have problems in their body that develop during pregnancy * Babies who are small for their age while in the participant's uterus * Pregnancy that happens outside of the uterus * How many participants die during pregnancy, while the baby is being born, and up to 12 weeks after delivering the baby * Babies who develop problems in their body after birth Researchers will also compare this information to people without SMA who have not received nusinersen. This study will be done as follows: * Information will start being collected when the participant decides to join the study. * Participants will be contacted at each trimester (about every 3 months) to learn about their health and pregnancy. * Participants' doctors will be contacted at each trimester, when the participants are about 6 or 7 months pregnant, and about 4 weeks after the delivery of the baby. * The babies' doctors will be contacted when the baby is 1, 2, 6, 12, 18, and 24 months old. * Each participant will be in the study until the end of their pregnancy and for up to 12 weeks after delivery. Each baby will be in the study for up to 2 years after birth. * The study overall will last at least 10 years from when the first participant joins the study.

Key Dates

Start date
Dec 15, 2023
Status verified
Mar 2026
Primary completion
Oct 31, 2032
Completion
Oct 31, 2033

Study Design

Enrollment
20 participants (estimated)

Arms

  • Arm: Nusinersen Treated Participants
    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.

Primary Outcome Measure

Number of Pregnancy Terminations [ Time Frame: Up to 10 years ]

Central Contacts

Locations (4)

FacilityCityStateZIPSite coordinators
Stanford Neuroscience Health CenterPalo AltoCalifornia94304
650-725-7622
John W. Day (PRINCIPAL_INVESTIGATOR)
Boston Children's HospitalBostonMassachusetts02115
617-355-8036
Basil T. Darras (PRINCIPAL_INVESTIGATOR)
Research SiteCambridgeMassachusetts02142-
Columbia University Irving Medical CenterNew YorkNew York10032
212-305-1319
Michio Hirano (PRINCIPAL_INVESTIGATOR)

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By research site
Stanford Neuroscience Health Center· Palo Alto, CABoston Children's Hospital· Boston, MAResearch Site· Cambridge, MAColumbia University Irving Medical Center· New York, NY

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