A Registered Cohort Study on SMA | NCT04010604

Studies
A Registered Cohort Study on SMA

NCT04010604

Sponsor

Wan-Jin Chen

Status

Recruiting

Last Update Posted

Sep 23, 2019Actual

Enrollment

2,000Estimated

Phase

Not provided

Conditions

Spinal Muscular Atrophy

Interventions

Not provided

Countries

China

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Sep 2019

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 1, 2019Actual

Primary Completion Date

Dec 31, 2039Estimated

Completion Date

Dec 31, 2049Estimated

First Submitted Date

Jul 1, 2019

First Submission Date that Met QC Criteria

Jul 3, 2019

First Posted Date

Jul 8, 2019Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Sep 19, 2019

Last Update Posted Date

Sep 23, 2019Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Wan-Jin Chen, The Vice-Director for the Department of Neurology, First Affiliated Hospital of Fujian Medical UniversitySponsor-Investigator

Lead Sponsor

Wan-Jin ChenOTHER

Collaborators

Not provided

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

2,000Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
SMA type I
SMA type II
SMA type III
Asymptomatic carriers of SMA
Relatives of SMA patients and carriers
Unrelated healthy controls

Interventions

Not provided

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
The time to death		From date of enrollment until the date of death from any cause, assessed up to 20years
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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

1 Week

Maximum Age

70 Years

Standard Ages

ChildAdultOlder Adult

Study Population

SMA patients are diagnosed with SMN1 gene deletion or mutation.

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Yi Lin, PhD	Contact	86-0591-87982772		linyi7811@163.com
Wan-Jian Chen, PhD	Contact	86-0591-87982772		wanjinchen75@fjmu.edu.cn

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Department of Neurology, First Affiliated Hospital Fujian Medical University	Recruiting	Fuzhou	Fujian	350005	China

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D009134	Muscular Atrophy, Spinal

Ancestor Terms

ID	Term
D013118	Spinal Cord Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D016472	Motor Neuron Disease

Browse Leaves

Not provided

Browse Branches

Not provided