Duchenne Muscular DystrophyPUL in DMDNCT02436720
Upper Limb Assessment in Duchenne Muscular Dystrophy
An observational study of Duchenne Muscular Dystrophy, run by Catholic University of the Sacred Heart at 1 site in Italy.
Trial summary
The literature on outcome measures assessing upper limbs in Duchenne muscular dystrophy (DMD) is quite scanty. While there have been considerable advances for ambulant DMD boys, no prospective study has so far been devoted to outcome measures in non ambulant patients, with increasing complaints from families and… Read more about this study
- Who is it for?
- Ages 4 to 35 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 300 participants
- Where
- 1 siteItaly
- Study dates
- Jan 2013to May 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 4 to 35 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Boys and adults affected by Duchenne muscular dystrophy
Who may be able to join
- proven DMD
Who cannot join
- severe mental retardation
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Performance of Upper Limb (PUL) in Duchenne Muscular Dystrophy
The literature on outcome measures assessing upper limbs in Duchenne muscular dystrophy (DMD) is quite scanty. While there have been considerable advances for ambulant DMD boys, no prospective study has so far been devoted to outcome measures in non ambulant patients, with increasing complaints from families and patients. This information appears to be highly important not only for a better understanding of the progression of the disease but also for possible enrollment of patients in future trials.
The aim of this project is to identify outcome measures for non ambulant patients in an Italian population of DMD patients. At least 200 non ambulant DMD boys and adults will be included in the study. All patients will be assessed using the newly developed Performance of Upper limb (PUL) test. This measure will be used at baseline and 6 and 12 months after baseline. This will allow to monitor possible changes over time and the rate of changes in patients with different level of ability and age. As part of this study the investigators will also correlate possible changes in upper limb function with other measures of care and function such as the EK scale.
The investigators aim to assess the suitability of the individual measures in a large number of patients, trying to establish whether whole scales or individual items appear to be relevant across ages and level of abilities. The investigators also aim to assess the suitability of the selected measures in a multicentric setting and the quantity of training required The data collected will also be analysed using Rasch analysis in order to improve the statistical properties of the measures used.
Read the detailed description
This is a multicentric study involving a large number of patients that could not be collected in a single Unit.
The aim of the project is to collect a large number of patients that will allow to assess the suitability of different measures assessing upper limb function. As DMD is a relatively rare condition, such goal can only be reached in a multicentric setting. All the participating centres (Rome Gemelli, Messina, Rome Bambin Gesù, Pavia, Genoa, Naples, Turin, Bologna, Padua, Milan, Bosisio Parini, Pisa) have a good record of cooperation in similar projects having already been involved in the validation of the North Star in DMD and of the Hammersmith Functional motor Scale for SMA.
The projects will be developed through different steps, related to different specific aims:
- Interobserver reliability/quality and quantity of training One of the aims of the study is to assess reliability of the selected measures in a multicentric setting and the quantity of training required to be sure of the reliability among observers after the training sessions. Following our experience in the training of the North Star Ambulatory Assessment (NSAA) in the same network, the investigators have become aware that a single training session may not be sufficient to be sure that interobserver reliability will be maintained after the training sessions (Mazzone et al, 2009).
- Natural history data collection The PUL will be applied to the whole cohort with repeated assessments at 6, 12, 18 and 24 months . This will allow to monitor possible changes over time and the rate of changes in patients with different level of ability and age. This will also help to evaluate the responsiveness to change and the level of sensitivity of the measure over an interval that would be useful for a study of a putative agent that could improve muscle power.
- Correlation with other measures Another aim of the study is to relate possible changes in upper limb function with other measures of care and function such as the EK scale, that provides information on aspects of function, such as transfer or self feeding that are important in everyday life. Correlation with respiratory function will also be performed.
- improving statistical properties of the scales. There has been increasing evidence in the last few years that Rasch analysis may help to improvethe statistical properties. The data collected will offer the opportunity to perform these new psychometric techniques.
- 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.
assessment of upper limb (PUL)
the test evaluates performance of upper limbs by providing a score for shoulder, elbow and distal domain and total score
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 May 2015 (estimated)
- Study startJan 2013
- Primary completionMar 2013
- Study completionMay 2015 (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
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Catholic University of the Sacred Heart
- Sponsor type
- University, hospital or other
- Collaborators
- Bambino Gesù Hospital, IRCCS National Neurological Institute "C. Mondino" Foundation, Azienda Ospedaliera San Giovanni Battista, University of Messina, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, University of Naples, IRCCS Fondazione Stella Maris, Istituto Giannina Gaslini, University of Padova, Maggiore Bellaria Hospital, Bologna, IRCCS Eugenio Medea, Fondazione Serena Onlus - Centro Clinico NeMO Milano
- Responsible party
- Principal investigator, Eugenio Mercuri, Catholic University of the Sacred Heart
- Investigators
- Eugenio Mercuri, MD, PhD, Principal investigator, Catholic University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02436720
- Sponsor's protocol ID
- UCSCPUL
- Acronym
- PUL in DMD
- Status last verified
- May 2015
- First submitted
- 30 Apr 2015
- Last updated
- 7 May 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Performance of Upper Limb (PUL) in Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02436720
- Acronym / sponsor ID
- PUL in DMD / UCSCPUL
- Conditions
- Duchenne Muscular Dystrophy
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Catholic University of the Sacred HeartUniversity, hospital or other
- Collaborators
- Bambino Gesù Hospital, IRCCS National Neurological Institute "C. Mondino" Foundation, Azienda Ospedaliera San Giovanni Battista, University of Messina, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, University of Naples, IRCCS Fondazione Stella Maris, Istituto Giannina Gaslini, University of Padova, Maggiore Bellaria Hospital, Bologna, IRCCS Eugenio Medea, Fondazione Serena Onlus - Centro Clinico NeMO Milano
- Study start
- Jan 2013
- Primary completion
- Mar 2013 (actual)
- Study completion
- May 2015 (estimated)
- First posted
- 7 May 2015 (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 literature on outcome measures assessing upper limbs in Duchenne muscular dystrophy (DMD) is quite scanty. While there have been considerable advances for ambulant DMD boys, no prospective study has so far been devoted to outcome measures in non ambulant patients, with increasing complaints from families and patients. This information appears to be highly important not only for a better understanding of the progression of the disease but also for possible enrollment of patients in future trials.
The aim of this project is to identify outcome measures for non ambulant patients in an Italian population of DMD patients. At least 200 non ambulant DMD boys and adults will be included in the study. All patients will be assessed using the newly developed Performance of Upper limb (PUL) test. This measure will be used at baseline and 6 and 12 months after baseline. This will allow to monitor possible changes over time and the rate of changes in patients with different level of ability and age. As part of this study the investigators will also correlate possible changes in upper limb function with other measures of care and function such as the EK scale.
The investigators aim to assess the suitability of the individual measures in a large number of patients, trying to establish whether whole scales or individual items appear to be relevant across ages and level of abilities. The investigators also aim to assess the suitability of the selected measures in a multicentric setting and the quantity of training required The data collected will also be analysed using Rasch analysis in order to improve the statistical properties of the measures used.
Detailed description
This is a multicentric study involving a large number of patients that could not be collected in a single Unit.
The aim of the project is to collect a large number of patients that will allow to assess the suitability of different measures assessing upper limb function. As DMD is a relatively rare condition, such goal can only be reached in a multicentric setting. All the participating centres (Rome Gemelli, Messina, Rome Bambin Gesù, Pavia, Genoa, Naples, Turin, Bologna, Padua, Milan, Bosisio Parini, Pisa) have a good record of cooperation in similar projects having already been involved in the validation of the North Star in DMD and of the Hammersmith Functional motor Scale for SMA.
The projects will be developed through different steps, related to different specific aims:
- Interobserver reliability/quality and quantity of training One of the aims of the study is to assess reliability of the selected measures in a multicentric setting and the quantity of training required to be sure of the reliability among observers after the training sessions. Following our experience in the training of the North Star Ambulatory Assessment (NSAA) in the same network, the investigators have become aware that a single training session may not be sufficient to be sure that interobserver reliability will be maintained after the training sessions (Mazzone et al, 2009).
- Natural history data collection The PUL will be applied to the whole cohort with repeated assessments at 6, 12, 18 and 24 months . This will allow to monitor possible changes over time and the rate of changes in patients with different level of ability and age. This will also help to evaluate the responsiveness to change and the level of sensitivity of the measure over an interval that would be useful for a study of a putative agent that could improve muscle power.
- Correlation with other measures Another aim of the study is to relate possible changes in upper limb function with other measures of care and function such as the EK scale, that provides information on aspects of function, such as transfer or self feeding that are important in everyday life. Correlation with respiratory function will also be performed.
- improving statistical properties of the scales. There has been increasing evidence in the last few years that Rasch analysis may help to improvethe statistical properties. The data collected will offer the opportunity to perform these new psychometric techniques.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 4 to 35 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Boys and adults affected by Duchenne muscular dystrophy
Key inclusion criteria (1 listed)
- proven DMD
Key exclusion criteria (1 listed)
- severe mental retardation
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
assessment of upper limb (PUL)
Time frame: 2 years
the test evaluates performance of upper limbs by providing a score for shoulder, elbow and distal domain and total score
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2015.
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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.
Eugenio Mercuri, MD, PhD principal investigator
Catholic University
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 Catholic University of the Sacred Heart at 1 site in Italy.
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 1 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: status not confirmed, as of the sponsor's last update on 7 May 2015. 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 Italy. 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?
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?
assessment of upper limb (PUL) (time frame: 2 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 7 May 2015; AzTrials last refreshed its copy on 29 Oct 2026.