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

Study identification

• EU PAS number:
  EUPAS104368
• Study ID:
  104369
• Official title and acronym:
  Spinraza (nusinersen) SMA Pregnancy Exposure Study Within Existing SMA Registries
• DARWIN EU® study:
  No
• Study countries:
  Austria

  Germany

  Poland

  Switzerland

  United Kingdom

  United States
• Study description:
  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 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 up to 14 months before the first day of their last period before getting pregnant. This was either the standard dose of 12 milligrams (mg) of nusinersen or the higher dose (50/28 mg). • Those who received nusinersen 14.5 months before the day they got pregnant • Those who received 12 mg or 50/28 mg of 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.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Biogen

Study timelines

Date when funding contract was signed

Planned:30/11/2023

Actual:15/12/2023

Study start date

Planned:30/11/2023

Actual:15/12/2023

Data analysis start date

Planned:30/11/2023

Actual:15/12/2023

Date of final study report

Planned:31/10/2033

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Biogen

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  Non-EU RMP only

Other study registration identification numbers and links

NCT05789758

Link to Clinicaltrials.gov