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

study id #: NCT04419233

condition: Muscular Atrophy, Spinal

status: Recruiting

purpose:

The primary objective of this study is to evaluate the safety of nusinersen sodium injection in the postmarketing setting in China.The secondary objectives are to collect data on the efficacy and the pharmacokinetics of nusinersen sodium injection in the post-marketing setting in China.

intervention: Nusinersen Sodium Injection

results: https://clinicaltrials.gov/ct2/show/results/NCT04419233

last updated: March 03, 2022

start date: November 18, 2020

estimated completion: May 11, 2023

last updated: May 21, 2021

size / enrollment: 50

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.
• Day 0 up to End of Treatment (2 Years)
  

secondary outcomes:

• Percentage of Participants who Achieved World Health Organization (WHO) Motor Milestone
  Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
• Hammersmith Infant Neurological Examination (HINE) Section 2 Scores
  Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
• Number of Participants with Ventilatory Support
  Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
• Plasma Concentrations of Nusinersen Sodium Injection
  Pre-dose and multiple time points post-dose on Day 0, post-dose on Day 28 and pre-dose every 4 months up to end of treatment (2 years)
• Cerebrospinal Fluid (CSF) Concentrations of Nusinersen Sodium Injection
  pre-dose on Day 0, Day 63, and Every 4 Months Up to End of Treatment (2 years)

inclusion criteria:

Key Inclusion Criteria:

Ability of the participant or his/her legally authorized representative (e.g., parent or legal guardian), as appropriate and applicable, to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local subject privacy regulations.
The decision to newly prescribe nusinersen sodium injection according to the local marketing authorization, including documentation of 5q SMA diagnosis, has been made by the Investigator and agreed to by the participant (or legally authorized representative)

Key

exclusion criteria: Criteria:

Hypersensitivity to the active substance or any of the excipients of nusinersen sodium injection.
Current or past administration of nusinersen sodium injection, in either a commercial or a clinical study setting.
Other unspecified reasons that, in the opinion of the Investigator or the Sponsor, make the participant unsuitable for enrollment.

NOTE: Other protocol defined Inclusion/

sponsor: Biogen

contacts: US Biogen Clinical Trial Center, 866-633-4636, clinicaltrials@biogen.com

investigators: Medical Director,Biogen

trial center locations: China

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