Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)

Muscular Atrophy, Spinal Clinical Trial

— STANDARD  

Official title:

A Multicenter, Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium) When Used in Routine Medical Practice in Korea

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04317794
• Other study ID #: 232SM403
• Status: Recruiting
• Start date: July 2, 2019
• Est. completion date: July 1, 2027

Study information

• Verified date: May 2024
• Source: Biogen
• Contact: US Biogen Clinical Trial Center
• Phone: 866-633-4636
• Email: clinicaltrials[@]biogen.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The primary objective is to evaluate the safety of nusinersen sodium injection in the postmarketing setting in Korea. The secondary objective is to evaluate the effectiveness of nusinersen sodium injection in the postmarketing setting in Korea.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 145
• Est. completion date: July 1, 2027
• Est. primary completion date: July 1, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Key Inclusion Criteria:

• Currently receiving or about to initiate treatment with commercial Spinraza in the postmarketing setting
• Genetic documentation of 5q-linked SMA

Key Exclusion Criteria:

• Hypersensitivity to the active substance or any of the excipients of Spinraza
• Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrollment in other interventional clinical trials for the treatment of SMA
• Inability to comply with study requirements 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
Korea, Republic of	Pusan Natioanl University Hospital	Busan
Korea, Republic of	Samsung Changwon Hospital	ChangWon
Korea, Republic of	Chungbuk National University Hospital	Cheongju-si
Korea, Republic of	Kyungpook National University Hospital	Daegu
Korea, Republic of	Yeungnam University Hospital	Daegu
Korea, Republic of	Chungnam National University Hospital	Daejeon
Korea, Republic of	Chonnam National University Hospital	Gwangju
Korea, Republic of	Chonbuk National University Hospital	Jeonju-si
Korea, Republic of	Asan Medical Center	Seoul
Korea, Republic of	Korea university Anam Hospital	Seoul
Korea, Republic of	Korea University Guro Hospital	Seoul
Korea, Republic of	Samsung Medical Center	Seoul
Korea, Republic of	Seoul Natioanl University Hospital	Seoul
Korea, Republic of	Severance Hospital, Yonsei University Health System	Seoul
Korea, Republic of	Pusan National University Yangsan Hospital	Yangsan

Sponsors (1)

Lead Sponsor	Collaborator
Biogen

Country where clinical trial is conducted

Korea, Republic of, 

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.	Up to End of Treatment (2 Years)
Secondary	Change from Baseline in 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 Approximately Every 4 Months Up to End of Treatment (2 years)
Secondary	Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE)	The HFMSE is a tool used to assess motor function in children with SMA. The original 20 item Hammersmith Functional Motor Scale (HFMS) was expanded to include 13 additional items to improve sensitivity for the higher functioning ambulant population. Participants are asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability where 0=unable, 1=performed with some assistance, and 2=unaided. The overall score is the sum of the scores for all activities.	Baseline, Day 63 and Approximately Every 4 Months Up to End of Treatment (2 years)
Secondary	Time to Death		Up to End of Treatment (2 years)
Secondary	Number of Participants with Ventilatory Support		Up to End of Treatment (2 years)