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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04010604
Other study ID # MRCTA,ECFAHOFFMU[2019]196
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date July 1, 2019
Est. completion date December 31, 2049

Study information

Verified date September 2019
Source First Affiliated Hospital of Fujian Medical University
Contact Yi Lin, PhD
Phone 86-0591-87982772
Email linyi7811@163.com
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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.


Recruitment information / eligibility

Status Recruiting
Enrollment 2000
Est. completion date December 31, 2049
Est. primary completion date December 31, 2039
Accepts healthy volunteers Accepts Healthy Volunteers
Gender All
Age group N/A to 70 Years
Eligibility Inclusion Criteria:

- 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

Exclusion Criteria:

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

Study Design


Locations

Country Name City State
China Department of Neurology, First Affiliated Hospital Fujian Medical University Fuzhou Fujian

Sponsors (1)

Lead Sponsor Collaborator
Wan-Jin Chen

Country where clinical trial is conducted

China, 

Outcome

Type Measure Description Time frame Safety issue
Primary The time to death From date of enrollment until the date of death from any cause, assessed up to 20years
Primary The correlation of genotype and phenotype Genotype is defined by survival motor neuron (SMN) 2 copy number(s) and phenotype is defined by clinical types and characteristics. From date of enrollment until the date of death from any cause, assessed up to 20years
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