NCT04012671

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
2,000

participants targeted

Target at P75+ for all trials

Timeline
288mo left

Started Jul 2019

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress22%
Jul 2019Dec 2049

Study Start

First participant enrolled

July 1, 2019

Completed
6 days until next milestone

First Submitted

Initial submission to the registry

July 7, 2019

Completed
2 days until next milestone

First Posted

Study publicly available on registry

July 9, 2019

Completed
20.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2039

Expected
10 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2049

Last Updated

March 22, 2021

Status Verified

February 1, 2021

Enrollment Period

20.5 years

First QC Date

July 7, 2019

Last Update Submit

March 18, 2021

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

  • Age at death

    the time when patient die

    20 years

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients who are diagnosed as Duchenne Muscular Dystrophy in the hosipital

You may qualify if:

  • 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.

You may not qualify if:

  • Presence of other clinically significant illness

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

First Affiliated Hospital of Fujian Medical University

Fuzhou, China

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Ning Wang, MD, PhD

CONTACT

13805015340ningwang@fjmu.edu.cn

Ming Jin, MD

CONTACT

Safariday@live.com

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

July 7, 2019

First Posted

July 9, 2019

Study Start

July 1, 2019

Primary Completion (Estimated)

December 31, 2039

Study Completion (Estimated)

December 31, 2049

Last Updated

March 22, 2021

Record last verified: 2021-02

Locations

CN(1)