Ireland
--:--IST
Active, not recruiting Observational

Adult Spinal Muscular Atrophy (SMA) China Registry

NCT05618379 · tracked via the Priya Life Science China tracker
Sponsor
Phase
Observational
Started
2023-01-06
Last updated
2026-08-04

Condition(s) studied

Muscular Atrophy, Spinal

Study 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

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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 outcome measure(s)

  • Number of Participants Characterized by Natural History of 5q-SMA Observed Among Adult Chinese Participants — Up to 60 Months
    The natural history characteristics will include the categories of the evolution of the patient's condition over the time of data collection from the registry, to describe the natural history of the disease in a real-life situation.
  • Number of Participants Characterized by Utilization of Disease Modifying Therapies (DMTs) of 5q-SMA Observed Among Adult Chinese Participants — Up to 60 Months
    The DMT utilization characteristics will include the categories of the evolution of the DMT treated patient's condition over the time of data collection from the registry, to describe the utilization of DMTs of adult SMA patients in a real-life situation.

Trial sites (12)

FacilityCityRegionStatus
Peking Union Medical College Hospital Beijing Beijing Municipality
The First Affiliated Hospital of Fujian Medical University Fuzhou Fujian
The First Affiliated Hospital, Sun Yat-sen University Guangzhou Guangdong
Peking University Shenzhen Hospital Shenzhen Guangdong
The Second Hospital of Hebei Medical University Shijiazhuang Hebei
Xiangya Hospital of Central South University Changsha Hunan
The First Affiliated Hospital of Soochow University Suzhou Jiangsu
The First Affiliated Hospital of Nanchang University Nanchang Jiangxi
Qilu Hospital of Shandong University Jinan Shandong
Huashan Hospital, Fudan University Shanghai Shanghai Municipality
West China Hospital, Sichuan University Chengdu Sichuan
The First People's Hospital of Yunnan Province Kunming Yun'nan
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05618379 on ClinicalTrials.gov ↗ ← All trials in China