Upper Limb Assessment in Duchenne Muscular Dystrophy

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Brief Title

Upper Limb Assessment in Duchenne Muscular Dystrophy

Official Title

Performance of Upper Limb (PUL) in Duchenne Muscular Dystrophy

Brief 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 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:

        1. 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).

        2. 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.

        3. 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.

        4. 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 Type

Observational


Primary Outcome

assessment of upper limb (PUL)


Condition

Duchenne Muscular Dystrophy



Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

300

Start Date

January 2013

Completion Date

May 2015

Primary Completion Date

March 2013

Eligibility Criteria

        Inclusion Criteria:

          -  proven DMD

        Exclusion Criteria:

          -  severe mental retardation
      

Gender

Male

Ages

4 Years - 35 Years

Accepts Healthy Volunteers

No

Contacts

Eugenio Mercuri, MD, PhD, , 

Location Countries

Italy

Location Countries

Italy

Administrative Informations


NCT ID

NCT02436720

Organization ID

UCSCPUL


Responsible Party

Principal Investigator

Study Sponsor

Catholic University of the Sacred Heart

Collaborators

 Bambino Gesù Hospital

Study Sponsor

Eugenio Mercuri, MD, PhD, Principal Investigator, Catholic University


Verification Date

May 2015