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A Registered Cohort Study on SMA

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ClinicalTrials.gov Identifier: NCT04010604
Recruitment Status : Recruiting
First Posted : July 8, 2019
Last Update Posted : September 23, 2019
Sponsor:
Information provided by (Responsible Party):
Wan-Jin Chen, First Affiliated Hospital of Fujian Medical University

Tracking Information
First Submitted Date July 1, 2019
First Posted Date July 8, 2019
Last Update Posted Date September 23, 2019
Actual Study Start Date July 1, 2019
Estimated Primary Completion Date December 31, 2039   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: July 3, 2019)
  • The time to death [ Time Frame: From date of enrollment until the date of death from any cause, assessed up to 20years ]
  • The correlation of genotype and phenotype [ Time Frame: From date of enrollment until the date of death from any cause, assessed up to 20years ]
    Genotype is defined by survival motor neuron (SMN) 2 copy number(s) and phenotype is defined by clinical types and characteristics.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Registered Cohort Study on SMA
Official Title A Registered Cohort Study on Spinal Muscular Atrophy
Brief 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.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population SMA patients are diagnosed with SMN1 gene deletion or mutation.
Condition Spinal Muscular Atrophy
Intervention Not Provided
Study Groups/Cohorts
  • SMA type I
  • SMA type II
  • SMA type III
  • Asymptomatic carriers of SMA
  • Relatives of SMA patients and carriers
  • Unrelated healthy controls
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: July 3, 2019)
2000
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 31, 2049
Estimated Primary Completion Date December 31, 2039   (Final data collection date for primary outcome measure)
Eligibility Criteria

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

Sex/Gender
Sexes Eligible for Study: All
Ages up to 70 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers Yes
Contacts
Contact: Yi Lin, PhD 86-0591-87982772 linyi7811@163.com
Contact: Wan-Jian Chen, PhD 86-0591-87982772 wanjinchen75@fjmu.edu.cn
Listed Location Countries China
Removed Location Countries  
 
Administrative Information
NCT Number NCT04010604
Other Study ID Numbers MRCTA,ECFAHOFFMU[2019]196
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Wan-Jin Chen, First Affiliated Hospital of Fujian Medical University
Study Sponsor Wan-Jin Chen
Collaborators Not Provided
Investigators Not Provided
PRS Account First Affiliated Hospital of Fujian Medical University
Verification Date September 2019