Pediatric Spinal Muscular Atrophy (SMA) China Registry

Muscular Atrophy, Spinal Clinical Trial

Official title:

Pediatric Patients With Spinal Muscular Atrophy in China: A Nationwide Registry

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05042921
• Other study ID #: CN-SMG-11774
• Status: Recruiting
• Start date: November 18, 2021
• Est. completion date: September 2, 2027

Study information

• Verified date: July 2023
• Source: Biogen
• Contact: US Biogen Clinical Trial Center
• Phone: 866-633-4636
• Email: clinicaltrials[@]biogen.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 600
• Est. completion date: September 2, 2027
• Est. primary completion date: September 2, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal

Locations

Country	Name	City	State
China	Beijing Children's Hospital, Capital Medical University	Beijing	Beijing
China	The First Hospital of Jilin University	Changchun	Jilin
China	Hunan Children's Hospital	Changsha	Hunan
China	Xiangya Hospital, Central South University	Changsha	Hunan
China	West China Second University Hospital, Sichuan University	Chengdu	Sichuan
China	Children's Hospital of Chongqing Medical University	Chongqing	Chongqing
China	Dalian Women and Children's Medical Group	Dalian	Liaoning
China	Fujian Medical University Union Hospital	Fuzhou	Fujian
China	The First Affiliated Hospital, Sun Yat-sen University	Guangzhou	Guangdong
China	The Children's Hospital, Zhejiang University School of Medicine	Hangzhou	Zhejiang
China	Anhui Children's Hospital	Hefei	Anhui
China	The Affiliated Hospital of Inner Mongolia Medical University	Hohhot	Inner Mongolia
China	Children's Hospital Affiliated to Shandong University	Jinan	Jinan Shandong
China	Jiangxi Provincial Children's Hospital	Nanchang	Jiangxi
China	Maternity and Child Health Care of Guangxi Zhuang Autonomous Region	Nanning	Guangxi
China	Children's Hospital of Fudan University	Shanghai	Shanghai
China	Shengjing Hospital of China Medical University	Shenyang	Liaoning
China	Shenzhen Children's Hospital	Shenzhen	Guangdong
China	The Second Hospital of Hebei Medical University	Shijiazhuang	Hebei
China	Wuhan Children's Hospital, Tongji Medical College Huazhong University of Science & Technology	Wuhan	Hubei
China	The Second Affiliated Hospital of Xi'An Jiaotong University	Xi'an	Shanxi
China	Xiamen Maternal and Child Health Hospital	Xiamen	Fujian
China	Xuzhou Central Hospital	Xuzhou	Jiangsu
China	People's Hospital of Ningxia Hui Autonomous Region	YInchuan	Ningxia
China	The Third Affiliated Hospital of Zhengzhou University	Zhengzhou	Henan

Sponsors (1)

Lead Sponsor	Collaborator
Biogen

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Effectiveness of DMT Treatments	Effectiveness will be assessed using following measures: time to mortality, motor function, motor measures, pulmonary function, scoliosis, hospitalizations and comorbidities, clinical observations, patient reported outcomes, wheelchair use, nutrition and electrophysiology and biomarkers.	Up to 60 months
Other	Safety of DMT Treatments	Safety will be assessed by adverse events (AEs) and serious AEs.	Up to 60 months
Primary	Natural History and Utilization of Disease Modifying Therapy (DMT) Treatments Among Pediatric Chinese Participants With Spinal Muscular Atrophy Linked to Chromosome 5q (5q-SMA)		Up to 60 months