Duchenne Muscular DystrophyNCT01753804
A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
An observational study of Duchenne Muscular Dystrophy, run by BioMarin Pharmaceutical at 16 sites in 10 countries.
Trial summary
To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible. Read more about this study
- Who is it for?
- Ages 3 to 18 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Observational study
- How many people
- 269 participants
- Where
- 16 sitesin 10 countries
- Study dates
- 1 Sep 2012to 1 Oct 2016
- 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 18 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Subjects diagnosed with DMD resulting from a mutation in the DMD gene which is confirmed by a state of the art DNA diagnostic technique covering all DMD gene exons
Who may be able to join
- Diagnosis of DMD resulting from a mutation in the DMD gene confirmed by a state of the art DNA diagnostic technique covering all DMD gene exons.
- Age 3 - 18 years
- Willing and able to comply with protocol requirements
- Life expectancy of at least 3 years
- Able to give informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations)
Who cannot join
- Current participation in a clinical study with an Investigational Medicinal Product (IMP)
- Participation within the previous 1 month in a clinical study with an IMP
Trial locations and contacts
16 sites in 10 countries.
16 sites in 10 countries
United States3 sites
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Nationwide Children's Hospital
Columbus, Ohio, 43205
UC Davis Health System
Sacramento, California, 95817
Belgium2 sites
Universitair Ziekenhuis
Ghent
Universitair Ziekenhuis Leuven
Leuven
Germany2 sites
Universitaetsklinikum Essen
Essen
Universitaetsklinikum Freiburg
Freiburg im Breisgau
Italy2 sites
Azienda Ospedaliera Universitaria Policlinico G. Martino
Messina
Policlinico Univsersitario Agostino Gemelli
Rome
Netherlands2 sites
Leids Universitair Medisch Centrum
Leiden
UMC St. Radboud
Nijmegen
About this study
Official title: A Prospective Natural History Study of Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy.
To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible.
Read the detailed description
This is a prospective study. All DMD patients that fulfil the inclusion/exclusion criteria are eligible although the study is weighted towards ambulant subjects aged 3 years or older. There will be 7 study visits and subjects will be in the study for a maximum of 3 years. Visits will occur every 6 months (+/- 1 month).
Up to 250 DMD subjects planned in the following categories :
- 75 % ambulant subjects aged between 3 and 18 years at study entry
- 25% non-ambulant subjects with a maximum age of 18 years at study entry
Subjects will be asked to perform muscle testing assessment with a clinical evaluator, such as walking for 6 minutes, climb stairs, breathe in a tube, see how they can move their arms and legs. They will be asked questions about how they feel overall and perform daily activities. These measurements will be assessed every 6 months.
Urine and blood samples will be collected once a year to measure biomarkers that will allow to have a better overview of DMD.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Observational study
- Keywords
- DMD, Myopathy, Natural History, Muscular dystrophy, Biomarkers, Muscle testing
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Diseases, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Observational studyOther
There is no medication or device tested in this study. This is an obversational study on the progression of the disease.
| Study group | Role | What they receive |
|---|---|---|
| Study participants | All participants will follow the same protocol, including muscle strength and function testing, and blood and urine collection, for a maximum of 7 visits over 3 years. Receives: Other: Observational study |
What is being measured
The primary outcome is the main question the study is designed to answer.
6 minute walk distance
Participants are asked to walk at their own preferred speed on a fixed distance for 6 minutes. Subjects are warned of the time and that they may stop earlier if they feel unable to continue. Total distance walked within 6 minutes (or until stopping) is recorded.
Measured: Change from visit 1 walking distance
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Dec 2012 (estimated)
- Study start1 Sep 2012
- Primary completion1 Oct 2016
- Study completion1 Oct 2016
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.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 269 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Nathalie Goemans, MD, Principal investigator, UZ Leuven, Belgium
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01753804
- Sponsor's protocol ID
- PRO-DMD-01
- Status last verified
- Dec 2017
- First submitted
- 13 Dec 2012
- Last updated
- 8 Dec 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Natural History Study of Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy.
- ClinicalTrials.gov ID
- NCT01753804
- Acronym / sponsor ID
- PRO-DMD-01
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- DMD, Myopathy, Natural History, Muscular dystrophy, Biomarkers, Muscle testing
- Study type
- Observational
- Recruitment status
- Terminated
- Enrolment
- 269 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 1 Sep 2012 (actual)
- Primary completion
- 1 Oct 2016 (actual)
- Study completion
- 1 Oct 2016 (actual)
- First posted
- 20 Dec 2012 (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.
To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible.
Detailed description
This is a prospective study. All DMD patients that fulfil the inclusion/exclusion criteria are eligible although the study is weighted towards ambulant subjects aged 3 years or older. There will be 7 study visits and subjects will be in the study for a maximum of 3 years. Visits will occur every 6 months (+/- 1 month).
Up to 250 DMD subjects planned in the following categories :
- 75 % ambulant subjects aged between 3 and 18 years at study entry
- 25% non-ambulant subjects with a maximum age of 18 years at study entry
Subjects will be asked to perform muscle testing assessment with a clinical evaluator, such as walking for 6 minutes, climb stairs, breathe in a tube, see how they can move their arms and legs. They will be asked questions about how they feel overall and perform daily activities. These measurements will be assessed every 6 months.
Urine and blood samples will be collected once a year to measure biomarkers that will allow to have a better overview of DMD.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Study participants | – | Other: Observational studyAll participants will follow the same protocol, including muscle strength and function testing, and blood and urine collection, for a maximum of 7 visits over 3 years. |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Subjects diagnosed with DMD resulting from a mutation in the DMD gene which is confirmed by a state of the art DNA diagnostic technique covering all DMD gene exons
Key inclusion criteria (5 listed)
- Diagnosis of DMD resulting from a mutation in the DMD gene confirmed by a state of the art DNA diagnostic technique covering all DMD gene exons.
- Age 3 - 18 years
- Willing and able to comply with protocol requirements
- Life expectancy of at least 3 years
- Able to give informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations)
Key exclusion criteria (2 listed)
- Current participation in a clinical study with an Investigational Medicinal Product (IMP)
- Participation within the previous 1 month in a clinical study with an IMP
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Observational studyThere is no medication or device tested in this study. This is an obversational study on the progression of the disease. | Other | Study participants |
Primary outcome
6 minute walk distance
Time frame: Change from visit 1 walking distance
Participants are asked to walk at their own preferred speed on a fixed distance for 6 minutes. Subjects are warned of the time and that they may stop earlier if they feel unable to continue. Total distance walked within 6 minutes (or until stopping) is recorded.
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2017.
- BioMarin websiteLink provided by the sponsor.
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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.
Nathalie Goemans, MD principal investigator
UZ Leuven, Belgium
16 sites in 10 countries: 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 BioMarin Pharmaceutical at 16 sites in 10 countries.
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 5 inclusion and 2 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: stopped early, as of the sponsor's last update on 8 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, Belgium, Germany, Italy, Netherlands and 5 more countries. 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 was stopped early. Results are not available in the source record.
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?
6 minute walk distance (time frame: Change from visit 1 walking distance). 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 8 Dec 2017.