Clinical trial NCT04419233: Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection
Official title: A Multicenter, Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection When Used in Routine Medical Practice in China
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Muscular Atrophy, Spinal
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2020-06-05
Enrollment: 50
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants who are prescribed nusinersen sodium injection in China according to the local marketing authorization (including documentation of 5q SMA diagnosis), agree to the collection and use of data as specified in the protocol, and meet the eligibility criteria.

Brief summary: 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.


Eligibility 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:

* 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/Exclusion criteria may apply.

Primary outcomes:
measure: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Day 0 up to End of Treatment (2 Years)
Secondary outcomes:
measure: Percentage of Participants who Achieved World Health Organization (WHO) Motor Milestone
timeFrame: Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
measure: Hammersmith Infant Neurological Examination (HINE) Section 2 Scores
timeFrame: Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
measure: Number of Participants with Ventilatory Support
timeFrame: Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
measure: Plasma Concentrations of Nusinersen Sodium Injection
timeFrame: 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)
measure: Cerebrospinal Fluid (CSF) Concentrations of Nusinersen Sodium Injection
timeFrame: pre-dose on Day 0, Day 63, and Every 4 Months Up to End of Treatment (2 years)
Other outcomes:
Locations:
city: Xicheng
country: China
facility: Research Site
state: Beijing Shi
city: Fuzhou
country: China
facility: Research Site
state: Fujian
city: Guangzhou
country: China
facility: Research Site
state: Guangdong
city: Suzhou
country: China
facility: Research Site
state: Jiangsu
city: Changchun
country: China
facility: Research Site
state: Jilin
city: Minhang
country: China
facility: Research Site
state: Shanghai
city: Chengdu
country: China
facility: Research Site
state: Sichuan
city: Beijing
country: China
facility: Research Site
state: Xicheng
city: Hangzhou
country: China
facility: Research Site
state: Zhejiang
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: