Clinical trial NCT04317794: Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)
Official title: A Multicenter, Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium) When Used in Routine Medical Practice in Korea
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Muscular Atrophy, Spinal
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2020-03-23
Enrollment: 145
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants who are receiving or about to initiate treatment with commercial Spinraza in Korea according to the local marketing authorization (including documentation of SMA diagnosis), agree to the collection and use of data as specified in the protocol, and meet the eligibility criteria.

Brief 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Up to End of Treatment (2 Years)
Secondary outcomes:
measure: Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Scores
timeFrame: Baseline, Day 63 and Approximately Every 4 Months Up to End of Treatment (2 years)
measure: Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE)
timeFrame: Baseline, Day 63 and Approximately Every 4 Months Up to End of Treatment (2 years)
measure: Time to Death
timeFrame: Up to End of Treatment (2 years)
measure: Number of Participants with Ventilatory Support
timeFrame: Up to End of Treatment (2 years)
Other outcomes:
Locations:
city: Busan
country: Korea, Republic of
facility: Pusan Natioanl University Hospital
city: ChangWon
country: Korea, Republic of
facility: Samsung Changwon Hospital
city: Cheongju-si
country: Korea, Republic of
facility: Chungbuk National University Hospital
city: Daegu
country: Korea, Republic of
facility: Kyungpook National University Hospital
city: Daegu
country: Korea, Republic of
facility: Yeungnam University Hospital
city: Daejeon
country: Korea, Republic of
facility: Chungnam National University Hospital
city: Gwangju
country: Korea, Republic of
facility: Chonnam National University Hospital
city: Jeonju-si
country: Korea, Republic of
facility: Chonbuk National University Hospital
city: Seoul
country: Korea, Republic of
facility: Severance Hospital
city: Seoul
country: Korea, Republic of
facility: Asan Medical Center
city: Seoul
country: Korea, Republic of
facility: Korea university Anam Hospital
city: Seoul
country: Korea, Republic of
facility: Korea University Guro Hospital
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: Seoul Natioanl University Hospital
city: Seoul
country: Korea, Republic of
facility: Severance Hospital, Yonsei University Health System
city: Yangsan
country: Korea, Republic of
facility: Pusan National University Yangsan Hospital
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: