Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection (PANDA)

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

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.

Study Overview

• Status: Completed
• Conditions: Muscular Atrophy, Spinal
• Intervention / Treatment: Drug: Nusinersen Sodium Injection

• Study Type: Observational
• Enrollment (Actual): 50

Contacts and Locations

Study Locations

• China
  • Beijing Shi
    • Xicheng, Beijing Shi, China, 100045
      • Research Site
  • Fujian
    • Fuzhou, Fujian, China, UNK
      • Research Site
  • Guangdong
    • Guangzhou, Guangdong, China, UNK
      • Research Site
  • Jiangsu
    • Suzhou, Jiangsu, China, UNK
      • Research Site
  • Jilin
    • Changchun, Jilin, China, 130021
      • Research Site
  • Shanghai
    • Minhang, Shanghai, China, 201103
      • Research Site
  • Sichuan
    • Chengdu, Sichuan, China, 610041
      • Research Site
  • Xicheng
    • Beijing, Xicheng, China, 100045
      • Research Site
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310052
      • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
All Participants; Participants with 5q SMA and who were prescribed with nusinersen sodium injection in China according to the local marketing authorization.	Drug: Nusinersen Sodium Injection; Administered as specified in the treatment arm.; Other Names: ISIS 396443; Spinraza; BIIB058

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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)
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)
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)

Collaborators and Investigators

• Sponsor: Biogen
• Investigators: Study Director: Medical Director, Biogen

Study record dates

Study Major Dates

• Study Start (Actual): November 18, 2020
• Primary Completion (Actual): November 21, 2023
• Study Completion (Actual): November 21, 2023

Study Registration Dates

• First Submitted: June 3, 2020
• First Submitted That Met QC Criteria: June 3, 2020
• First Posted (Actual): June 5, 2020

Study Record Updates

• Last Update Posted (Estimated): December 7, 2023
• Last Update Submitted That Met QC Criteria: December 6, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Pathological Conditions, Anatomical; Spinal Cord Diseases; Atrophy; Motor Neuron Disease; Muscular Atrophy; Muscular Atrophy, Spinal
• Other Study ID Numbers: 232SM402

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No