Duchenne Muscular DystrophyNCT04012671
A Registered Cohort Study on Duchenne Muscular Dystrophy
An observational study of Duchenne Muscular Dystrophy, run by Ning Wang, MD., PhD. at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 2,000 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2019to 31 Dec 2049
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients who are diagnosed as Duchenne Muscular Dystrophy in the hosipital
Who may be able to join
- 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.
Who cannot join
- Presence of other clinically significant illness
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Ming Jin, MD contact
China1 site, 1 recruiting
- Recruiting
First Affiliated Hospital of Fujian Medical University
Fuzhou
Ming Jin, MD, Safariday@live.com
Ning Wang, MD,PhD
About this study
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.
- Conditions
- Duchenne Muscular Dystrophy
- Medical subject headings
- Muscular Dystrophy, Duchenne
What is being measured
The primary outcome is the main question the study is designed to answer.
Age at death
the time when patient die
Measured: 20 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2019
- Study start1 Jul 2019
- Primary completion31 Dec 2039 (estimated)
- Study completion31 Dec 2049 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 2,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ning Wang, MD., PhD.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Ning Wang, MD., PhD., First Affiliated Hospital of Fujian Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04012671
- Sponsor's protocol ID
- MRCTA,ECFAH of FMU [2019]193
- Status last verified
- Feb 2021
- First submitted
- 7 Jul 2019
- Last updated
- 22 Mar 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Registered Cohort Study on Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT04012671
- Acronym / sponsor ID
- MRCTA,ECFAH of FMU [2019]193
- Conditions
- Duchenne Muscular Dystrophy
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 2,000 participants (estimated)
- Lead sponsor
- Ning Wang, MD., PhD.University, hospital or other
- Study start
- 1 Jul 2019 (actual)
- Primary completion
- 31 Dec 2039 (estimated)
- Study completion
- 31 Dec 2049 (estimated)
- First posted
- 9 Jul 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients who are diagnosed as Duchenne Muscular Dystrophy in the hosipital
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (1 listed)
- Presence of other clinically significant illness
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Age at death
Time frame: 20 years
the time when patient die
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Mar 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Ming Jin, MD contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy, run by Ning Wang, MD., PhD. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ning Wang, MD, PhD (13805015340 ext. 13805015340, ningwang@fjmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Age at death (time frame: 20 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Mar 2021; AzTrials last refreshed its copy on 31 Oct 2026.