Clinical trial NCT05618379: Adult Spinal Muscular Atrophy (SMA) China Registry
Official title: Adult Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Muscular Atrophy, Spinal
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2022-11-16
Enrollment: 200
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Adult 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) among adult Chinese participants with SMA linked to chromosome 5q (5q-SMA).


Eligibility criteria: Key Inclusion Criteria:

-Genetically confirmed 5q-SMA.

Key Exclusion Criteria:

-Other types of SMA (non 5q-SMA).

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Primary outcomes:
measure: Number of Participants Characterized by Natural History of 5q-SMA Observed Among Adult Chinese Participants
timeFrame: Up to 60 Months
measure: Number of Participants Characterized by Utilization of Disease Modifying Therapies (DMTs) of 5q-SMA Observed Among Adult Chinese Participants
timeFrame: Up to 60 Months
Secondary outcomes:
Other outcomes:
measure: Time to Mortality
timeFrame: Up to 60 Months
measure: Number of Participants With Clinical Characteristics
timeFrame: Up to 60 Months
measure: Number of Participants with Hospitalization
timeFrame: Up to 60 Months
measure: Number of Participants With Comorbidities
timeFrame: Up to 60 Months
measure: Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAEs)
timeFrame: Up to 60 Months
Locations:
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
city: Fuzhou
country: China
facility: The First Affiliated Hospital of Fujian Medical University
state: Fujian
city: Guangzhou
country: China
facility: The First Affiliated Hospital, Sun Yat-sen University
state: Guangdong
city: Shenzhen
country: China
facility: Peking University Shenzhen Hospital
state: Guangdong
city: Shijiazhuang
country: China
facility: The Second Hospital of Hebei Medical University
state: Hebei
city: Changsha
country: China
facility: Xiangya Hospital of Central South University
state: Hunan
city: Suzhou
country: China
facility: The First Affiliated Hospital of Soochow University
state: Jiangsu
city: Nanchang
country: China
facility: The First Affiliated Hospital of Nanchang University
state: Jiangxi
city: Jinan
country: China
facility: Qilu Hospital of Shandong University
state: Shandong
city: Shanghai
country: China
facility: Huashan Hospital, Fudan University
state: Shanghai
city: Chengdu
country: China
facility: West China Hospital, Sichuan University
state: Sichuan
city: Kunming
country: China
facility: The First People's Hospital of Yunnan Province
state: Yunnan
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: