Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection

NCT04419233 | Completed

• 1 other identifier: 232SM402
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 9

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.

Conditions

Muscular Atrophy, Spinal

Outcome Measures

Primary Outcomes (1)

• 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 (5)

• 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)

Study Arms (1)

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

Interventions

Nusinersen Sodium Injection DRUG

Administered as specified in the treatment arm.

Also known as: BIIB058, ISIS 396443, Spinraza

All Participants

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• Biogen: lead

Study Sites (9)

Research Site

Xicheng, Beijing Municipality, 100045, China

Location

Research Site

Fuzhou, Fujian, UNK, China

Location

Research Site

Guangzhou, Guangdong, UNK, China

Location

Research Site

Suzhou, Jiangsu, UNK, China

Location

Research Site

Changchun, Jilin, 130021, China

Location

Research Site

Minhang, Shanghai Municipality, 201103, China

Location

Research Site

Chengdu, Sichuan, 610041, China

Location

Research Site

Beijing, Xicheng, 100045, China

Location

Research Site

Hangzhou, Zhejiang, 310052, China

Location

Related Publications (1)

• Jiang Y, Wang Y, Xiong H, Li W, Luo R, Chen W, Yin F, Lu J, Liang J, Chen WJ, Lu X, Wang H, Tang J, Monine M, Makepeace C, Jin X, Foster R, Chin R, Berger Z. A Post-Marketing Surveillance Study of Nusinersen for Spinal Muscular Atrophy in Routine Medical Practice in China: Interim Results. Adv Ther. 2024 Jul;41(7):2743-2756. doi: 10.1007/s12325-024-02852-7. Epub 2024 May 9.

  PMID: 38722537 DERIVED

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Interventions

nusinersen

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Study Officials

• Medical Director

  Biogen

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 3, 2020
• First Posted: June 5, 2020
• Study Start: November 18, 2020
• Primary Completion: November 21, 2023
• Study Completion: November 21, 2023
• Last Updated: December 7, 2023

Record last verified: 2023-12

Data Sharing

• IPD Sharing: Will share

Locations

CN (9)