Clinical trial NCT05042921: Pediatric Spinal Muscular Atrophy (SMA) China Registry
Official title: Pediatric Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Muscular Atrophy, Spinal
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2021-09-13
Enrollment: 600
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Pediatric participants with 5q-SMA will be enrolled to obtain information on both the natural history of the disease and effectiveness and safety of DMTs.

Brief summary: The primary objective of the study is to describe the natural history and utilization of disease modifying therapy (DMT) treatments among pediatric Chinese participants with spinal muscular atrophy linked to chromosome 5q (5q-SMA). The study will examine SMA natural history and DMT outcomes in a real-world setting both prospectively and retrospectively.


Eligibility criteria: Key Inclusion Criteria:

* Ability of the participant and/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, to provide informed consent, and to authorize the use of confidential health information in accordance with national and local privacy regulations.
* Genetically confirmed 5q-SMA.
* Age \< 18 years at enrollment.

Key Exclusion Criteria:

* Unable or unwilling to provide informed consent.
* Other types of SMA (non 5q-SMA).

NOTE: Other protocol defined inclusion/ exclusion criteria may apply.

Primary outcomes:
measure: Natural History and Utilization of Disease Modifying Therapy (DMT) Treatments Among Pediatric Chinese Participants With Spinal Muscular Atrophy Linked to Chromosome 5q (5q-SMA)
timeFrame: Up to 60 months
Secondary outcomes:
Other outcomes:
measure: Effectiveness of DMT Treatments
timeFrame: Up to 60 months
measure: Safety of DMT Treatments
timeFrame: Up to 60 months
Locations:
city: Hefei
country: China
facility: Anhui Children's Hospital
state: Anhui
city: Beijing
country: China
facility: Beijing Children's Hospital, Capital Medical University
state: Beijing
city: Chongqing
country: China
facility: Children's Hospital of Chongqing Medical University
state: Chongqing
city: Fuzhou
country: China
facility: Fujian Medical University Union Hospital
state: Fujian
city: Xiamen
country: China
facility: Xiamen Maternal and Child Health Hospital
state: Fujian
city: Guangzhou
country: China
facility: The First Affiliated Hospital, Sun Yat-sen University
state: Guangdong
city: Shenzhen
country: China
facility: Shenzhen Children's Hospital
state: Guangdong
city: Nanning
country: China
facility: Maternity and Child Health Care of Guangxi Zhuang Autonomous Region
state: Guangxi
city: Shijiazhuang
country: China
facility: The Second Hospital of Hebei Medical University
state: Hebei
city: Zhengzhou
country: China
facility: The Third Affiliated Hospital of Zhengzhou University
state: Henan
city: Wuhan
country: China
facility: Wuhan Children's Hospital, Tongji Medical College Huazhong University of Science & Technology
state: Hubei
city: Changsha
country: China
facility: Hunan Children's Hospital
state: Hunan
city: Changsha
country: China
facility: Xiangya Hospital, Central South University
state: Hunan
city: Hohhot
country: China
facility: The Affiliated Hospital of Inner Mongolia Medical University
state: Inner Mongolia
city: Xuzhou
country: China
facility: Xuzhou Central Hospital
state: Jiangsu
city: Nanchang
country: China
facility: Jiangxi Provincial Children's Hospital
state: Jiangxi
city: Changchun
country: China
facility: The First Hospital of Jilin University
state: Jilin
city: Jinan
country: China
facility: Children's Hospital Affiliated to Shandong University
state: Jinan Shandong
city: Dalian
country: China
facility: Dalian Women and Children's Medical Group
state: Liaoning
city: Shenyang
country: China
facility: Shengjing Hospital of China Medical University
state: Liaoning
city: YInchuan
country: China
facility: People's Hospital of Ningxia Hui Autonomous Region
state: Ningxia
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
state: Shanghai
city: Xi'an
country: China
facility: The Second Affiliated Hospital of Xi'An Jiaotong University
state: Shanxi
city: Chengdu
country: China
facility: West China Second University Hospital, Sichuan University
state: Sichuan
city: Hangzhou
country: China
facility: The Children's Hospital, Zhejiang University School of Medicine
state: Zhejiang
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: