Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection

Muscular Atrophy, Spinal Clinical Trial

— PANDA  

Official title:

A Multicenter, Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection When Used in Routine Medical Practice in China

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04419233
• Other study ID #: 232SM402
• Status: Completed
• Start date: November 18, 2020
• Est. completion date: November 21, 2023

Study information

• Verified date: December 2023
• Source: Biogen
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 50
• Est. completion date: November 21, 2023
• Est. primary completion date: November 21, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Muscular Atrophy
• Muscular Atrophy, Spinal

Intervention

Drug:
• Nusinersen Sodium Injection
Administered as specified in the treatment arm.

Locations

Country	Name	City	State
China	Research Site	Beijing	Xicheng
China	Research Site	Changchun	Jilin
China	Research Site	Chengdu	Sichuan
China	Research Site	Fuzhou	Fujian
China	Research Site	Guangzhou	Guangdong
China	Research Site	Hangzhou	Zhejiang
China	Research Site	Minhang	Shanghai
China	Research Site	Suzhou	Jiangsu
China	Research Site	Xicheng	Beijing Shi

Sponsors (1)

Lead Sponsor	Collaborator
Biogen

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Percentage of Participants who Achieved World Health Organization (WHO) Motor Milestone	WHO Multicentre Growth Reference Study (MGRS), a component to assess gross motor development and is usually assessed in terms of age of achievement of motor milestones. Six distinct gross motor milestones criteria are as follows: sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone.	Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
Secondary	Hammersmith Infant Neurological Examination (HINE) Section 2 Scores	Section 2 of the HINE is used to assess motor milestones of the participants. It is composed of 8 motor milestone categories: voluntary grasp, ability to kick in supine position, head control, rolling, sitting, crawling, standing, and walking.	Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
Secondary	Number of Participants with Ventilatory Support		Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)
Secondary	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)
Secondary	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)