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Completed

NCT Number: NCT04419233

Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection

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.

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Key information

Conditions

Muscular Atrophy, Spinal Central Nervous System Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Research Site, Xicheng, Beijing Municipality, China

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Nusinersen Sodium Injection

Drug

Administered as specified in the treatment arm.

Other names: BIIB058, ISIS 396443, Spinraza

Primary outcomes

  1. Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: Day 0 up to End of Treatment (2 Years)

    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.

Secondary outcomes

  1. Percentage of Participants who Achieved World Health Organization (WHO) Motor Milestone

    Time frame: Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)

    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.

  2. Hammersmith Infant Neurological Examination (HINE) Section 2 Scores

    Time frame: Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)

    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.

  3. Number of Participants with Ventilatory Support

    Time frame: Baseline, Day 63 and Every 4 Months Up to End of Treatment (2 years)

  4. Plasma Concentrations of Nusinersen Sodium Injection

    Time frame: 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)

  5. Cerebrospinal Fluid (CSF) Concentrations of Nusinersen Sodium Injection

    Time frame: pre-dose on Day 0, Day 63, and Every 4 Months Up to End of Treatment (2 years)

Sponsors and collaborators

Lead sponsor

Biogen

Industry

Registry information

Official study title

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

Acronym: PANDA

Important dates

Study start
2020
Primary completion
2023
Study completion
2023
First posted
Jun 5, 2020
Registry last updated
Dec 7, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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