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出境医 / 临床实验 / A Registered Cohort Study on Duchenne Muscular Dystrophy
A Registered Cohort Study on Duchenne Muscular Dystrophy
Study Description
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.
| Condition or disease |
|---|
| Duchenne Muscular Dystrophy |
Study Design
| Study Type : | Observational |
| Estimated Enrollment : | 2000 participants |
| Observational Model: | Cohort |
| Time Perspective: | Prospective |
| Official Title: | A Registered Cohort Study on Duchenne Muscular Dystrophy |
| Actual Study Start Date : | July 1, 2019 |
| Estimated Primary Completion Date : | December 31, 2039 |
| Estimated Study Completion Date : | December 31, 2049 |
Arms and Interventions
Outcome Measures
Primary Outcome Measures :
- Age at death [ Time Frame: 20 years ]the time when patient die
Eligibility Criteria
| Ages Eligible for Study: | 2 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
Study Population
Patients who are diagnosed as Duchenne Muscular Dystrophy in the hosipital
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
Contacts and Locations
Contacts
| Contact: Ning Wang, MD, PhD | 13805015340 ext 13805015340 | ningwang@fjmu.edu.cn | |
| Contact: Ming Jin, MD | Safariday@live.com |
Locations
| China | |
| First Affiliated Hospital of Fujian Medical University | Recruiting |
| Fuzhou, China | |
| Contact: Ming Jin, MD Safariday@live.com | |
| Principal Investigator: Ning Wang, MD,PhD | |
| Sub-Investigator: Wan-Jin Chen, MD,PhD | |
Sponsors and Collaborators
Ning Wang, MD., PhD.
| Tracking Information | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| First Submitted Date | July 7, 2019 | ||||||||
| First Posted Date | July 9, 2019 | ||||||||
| Last Update Posted Date | March 22, 2021 | ||||||||
| 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 |
Age at death [ Time Frame: 20 years ] the time when patient die
|
||||||||
| 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 Duchenne Muscular Dystrophy | ||||||||
| Official Title | A Registered Cohort Study on Duchenne Muscular Dystrophy | ||||||||
| 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 | ||||||||
| 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 | Patients who are diagnosed as Duchenne Muscular Dystrophy in the hosipital | ||||||||
| Condition | Duchenne Muscular Dystrophy | ||||||||
| Intervention | Not Provided | ||||||||
| Study Groups/Cohorts | Not Provided | ||||||||
| 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 |
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:
Exclusion Criteria:
|
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| Sex/Gender |
|
||||||||
| Ages | 2 Years and older (Child, Adult, Older Adult) | ||||||||
| Accepts Healthy Volunteers | No | ||||||||
| Contacts |
|
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| Listed Location Countries | China | ||||||||
| Removed Location Countries | |||||||||
| Administrative Information | |||||||||
| NCT Number | NCT04012671 | ||||||||
| Other Study ID Numbers | MRCTA,ECFAH of FMU [2019]193 | ||||||||
| Has Data Monitoring Committee | Yes | ||||||||
| U.S. FDA-regulated Product |
|
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| IPD Sharing Statement | Not Provided | ||||||||
| Responsible Party | Ning Wang, MD., PhD., First Affiliated Hospital of Fujian Medical University | ||||||||
| Study Sponsor | Ning Wang, MD., PhD. | ||||||||
| Collaborators | Not Provided | ||||||||
| Investigators | Not Provided | ||||||||
| PRS Account | First Affiliated Hospital of Fujian Medical University | ||||||||
| Verification Date | February 2021 | ||||||||
