A Registered Cohort Study on Duchenne Muscular Dystrophy | NCT04012671

Studies
A Registered Cohort Study on Duchenne Muscular Dystrophy

NCT04012671

Sponsor

Ning Wang, MD., PhD.

Status

Recruiting

Last Update Posted

Mar 22, 2021Actual

Enrollment

2,000Estimated

Phase

Not provided

Conditions

Duchenne Muscular Dystrophy

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

Feb 2021

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 7, 2019

First Submission Date that Met QC Criteria

Jul 7, 2019

First Posted Date

Jul 9, 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

Mar 18, 2021

Last Update Posted Date

Mar 22, 2021Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Ning Wang, MD., PhD., Professor, First Affiliated Hospital of Fujian Medical UniversitySponsor-Investigator

Lead Sponsor

Ning Wang, MD., PhD.OTHER

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

Dystrophinopathy is a term of X-linked recessive genetic disease, including Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and the X-linked dilated cardiomyopathy. The aim of this study is to determine the clinical spectrum and natural progression of dystrophinopathy in a prospective multicenter natural history study, to assess the clinical, genetic of patients with dystrophinopathy to optimize clinical management.

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

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Age at death	the time when patient die	20 years

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Beyond 2 years old
Diagnosis with Duchenne Muscular Dystrophy, and female carriers, genotypically confirmed
Diagnosis should be supported by muscle biopsy, if no genetic confirmation.

Exclusion Criteria:

Presence of other clinically significant illness

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

2 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Patients who are diagnosed as Duchenne Muscular Dystrophy in the hosipital

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Ning Wang, MD, PhD	Contact	13805015340	13805015340	ningwang@fjmu.edu.cn
Ming Jin, MD	Contact			Safariday@live.com

Overall Officials

Not provided

Locations

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

Condition Browse Module

MeSH Terms

ID	Term
D020388	Muscular Dystrophy, Duchenne

Ancestor Terms

ID	Term
D009136	Muscular Dystrophies
D020966	Muscular Disorders, Atrophic
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases

Browse Leaves

Not provided

Browse Branches

Not provided