A Registered Cohort Study on SMA

Recruiting now

Conditions studied: Spinal Muscular Atrophy

In brief

Spinal muscular atrophy (SMA) is an autosomal recessive disease that causes progressive muscle wasting and weakness due to loss of motor neurons in the spinal cord. This is a registered cohort of spinal muscular atrophy (SMA) type I，II and III in China. This study will provide further insights into the clinical course of SMA including overall survival, demographic characteristics, motor function, respiratory support, feeding and nutritional support, growth and development. The correlation of genotype and phenotype will be conducted.

Key facts

Study ID

NCT04010604

Run by

Wan-Jin Chen

People needed

2000

Starts

2019-07-01

Expected to finish

2049-12-31

Last updated by the study team

2019-09-23

Who can join

Age: 0 and older, up to 70. Sex: any. Healthy volunteers: accepted.

You may qualify if…

• Patients with SMA types I, II and III
• Asymptomatic SMA carriers
• Relatives of SMA patients or carriers
• Unrelated healthy controls
• Participants or Parent(s)/legal guardian(s) willing and able to complete the informed consent process

You may not qualify if…

• Participants are unable to comply with trial procedures and visit schedule

Where it is running

• Department of Neurology, First Affiliated Hospital Fujian Medical University — Fuzhou, Fujian, China (enrolling)

Full record on ClinicalTrials.gov

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