Duchenne Muscular Dystrophy (DMD)IBISDNCT01380964
Research of Biomarkers in Duchenne Muscular Dystrophy Patients
An observational study of Duchenne Muscular Dystrophy (DMD), run by Genethon at 1 site in France.
Trial summary
The purpose of this study is to identify potential biomarkers for the diagnosis, disease progression assessment and response to treatment in patients with Duchenne Muscular Dystrophy. Read more about this study
- Who is it for?
- Ages 3 to 20 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- How many people
- 220 participants
- Where
- 1 siteFrance
- Study dates
- Jun 2011to Dec 2015
- Phase
- Observational
- Are there results?
- None posted in the registry
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 3 to 20 years
- Men only
- Healthy volunteers accepted
- Child, Adult
Study population: patients with Duchenne Muscular Dystrophy controls (healthy or patients with a non-related disease)
Who may be able to join
- FOR PATIENTS:
- Diagnosis of DMD confirmed by genetic testing
- Age over 3 years
- Weight over 15 kg
- Informed consent signed
- FOR CONTROLS:
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- Age over 3 years
- Male gender
- Weight over 15 kg
- Subjects with national health insurance coverage
- Informed consent signed
- Nonacute or chronic muscular, allergic, infectious, endocrine or inflammatory disorder in the 3 weeks preceding inclusion
Who cannot join
- FOR PATIENTS:
- Concomitant chronic or acute muscular, endocrine, infectious, allergic or inflammatory disorder in the three weeks preceding the blood test
- Intake of medicines other than angiotensin-converting enzyme inhibitors, beta blockers, dietary supplements, vitamins, alendronate and methylphenidate. Steroids (and medicines prescribed with them such as calcium supplements and proton pump inhibitors) will be discussed
- Mental retardation or autism
- Vaccination or treatment with immunoglobulins within the three months preceding inclusion
- FOR CONTROLS:
- Concomitant chronic or acute muscular, neurological (including mental retardation and autism), infectious or inflammatory disorder in the three weeks preceding the blood test
- Vaccination or treatment with immunoglobulins within the three months preceding inclusion
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Research of Biomarkers for Disease Diagnosis, Disease Monitoring and Therapeutic Treatment Response in Duchenne Muscular Dystrophy Patients
The purpose of this study is to identify potential biomarkers for the diagnosis, disease progression assessment and response to treatment in patients with Duchenne Muscular Dystrophy.
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- Duchenne Muscular Dystrophy, biomarkers, transcriptomics, mironics, proteomics
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| DMD patients | DMD Patients | |
| Control patients | Control patients |
What is being measured
The primary outcome is the main question the study is designed to answer.
IBiSD aims to identify and validate new and disease-specific biomarkers.
This study will establish the relevance of urinary and blood biomarkers for the diagnosis, follow-up and assessment of treatment response in patients with DMD (IBiSD1, 2 and 4). IBiSD will also attempt to establish the seroprevalence to the different strains of AAV in patients with DMD (IBiSD3).
Measured: End of study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2011 (estimated)
- Study startJun 2011
- Primary completionDec 2015
- Study completionDec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies 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
- 220 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genethon
- Sponsor type
- University, hospital or other
- Collaborators
- Institute of Myology
- Responsible party
- Sponsor
- Investigators
- Laurent SERVAIS, MD, Principal investigator, Myology Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01380964
- Sponsor's protocol ID
- GEE006.10
- Acronym
- IBISD
- Status last verified
- Aug 2016
- First submitted
- 23 Jun 2011
- Last updated
- 17 Aug 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Research of Biomarkers for Disease Diagnosis, Disease Monitoring and Therapeutic Treatment Response in Duchenne Muscular Dystrophy Patients
- ClinicalTrials.gov ID
- NCT01380964
- Acronym / sponsor ID
- IBISD / GEE006.10
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- Duchenne Muscular Dystrophy, biomarkers, transcriptomics, mironics, proteomics
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 220 participants (actual)
- Lead sponsor
- GenethonUniversity, hospital or other
- Collaborators
- Institute of Myology
- Study start
- Jun 2011
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 27 Jun 2011 (estimated)
- 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.
The purpose of this study is to identify potential biomarkers for the diagnosis, disease progression assessment and response to treatment in patients with Duchenne Muscular Dystrophy.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| DMD patients | – | –DMD Patients |
| Control patients | – | –Control patients |
Who may qualify
- Ages
- 3 to 20 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- patients with Duchenne Muscular Dystrophy controls (healthy or patients with a non-related disease)
Key inclusion criteria (12 listed)
- FOR PATIENTS:
- Diagnosis of DMD confirmed by genetic testing
- Age over 3 years
- Weight over 15 kg
- Informed consent signed
Key exclusion criteria (8 listed)
- FOR PATIENTS:
- Concomitant chronic or acute muscular, endocrine, infectious, allergic or inflammatory disorder in the three weeks preceding the blood test
- Intake of medicines other than angiotensin-converting enzyme inhibitors, beta blockers, dietary supplements, vitamins, alendronate and methylphenidate. Steroids (and medicines prescribed with them such as calcium supplements and proton pump inhibitors) will be discussed
- Mental retardation or autism
- Vaccination or treatment with immunoglobulins within the three months preceding inclusion
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
IBiSD aims to identify and validate new and disease-specific biomarkers.
Time frame: End of study
This study will establish the relevance of urinary and blood biomarkers for the diagnosis, follow-up and assessment of treatment response in patients with DMD (IBiSD1, 2 and 4). IBiSD will also attempt to establish the seroprevalence to the different strains of AAV in patients with DMD (IBiSD3).
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Aug 2016.
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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. This study is not recruiting at the moment.
Laurent SERVAIS, MD principal investigator
Myology Institute
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 (DMD), run by Genethon at 1 site in France.
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 12 inclusion and 8 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: finished, as of the sponsor's last update on 17 Aug 2016. 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 France. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
IBiSD aims to identify and validate new and disease-specific biomarkers. (time frame: End of study). 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 17 Aug 2016.