Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)

study id #: NCT04317794

condition: Muscular Atrophy, Spinal

status: Recruiting

The primary objective is to evaluate the safety of nusinersen sodium injection in the postmarketing setting in Korea. The secondary objective is to evaluate the effectiveness of nusinersen sodium injection in the postmarketing setting in Korea.

intervention: Nusinersen Sodium Injection

results: https://clinicaltrials.gov/ct2/show/results/NCT04317794

last updated: March 03, 2022

start date: July 2, 2019

estimated completion: July 1, 2027

last updated: March 24, 2021

size / enrollment: 145

primary outcomes:

• Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
  An AE is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose results in death, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect, or is a medically important event.
• Up to End of Treatment (2 Years)
  

secondary outcomes:

• Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Scores
  Baseline, Day 63 and Approximately Every 4 Months Up to End of Treatment (2 years)
• Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE)
  Baseline, Day 63 and Approximately Every 4 Months Up to End of Treatment (2 years)
• Time to Death
  Up to End of Treatment (2 years)
• Number of Participants with Ventilatory Support
  Up to End of Treatment (2 years)

inclusion criteria:

exclusion criteria: Criteria:

Hypersensitivity to the active substance or any of the excipients of Spinraza
Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrollment in other interventional clinical trials for the treatment of SMA
Inability to comply with study requirements

NOTE: Other protocol defined Inclusion/

sponsor: Biogen

contacts: US Biogen Clinical Trial Center, 866-633-4636, [email protected]

investigators: Medical Director,Biogen

trial center locations: Korea, Republic of

• Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)