Upper Limb Assessment in Duchenne Muscular Dystrophy

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.

Conditions

• Duchenne Muscular Dystrophy

Sponsors & Collaborators

• Bambino Gesù Hospital

  collaborator UNKNOWN

• IRCCS National Neurological Institute "C. Mondino" Foundation

  collaborator OTHER

• Azienda Ospedaliera San Giovanni Battista

  collaborator OTHER

• University of Messina

  collaborator OTHER

• Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

  collaborator OTHER

• University of Naples

  collaborator OTHER

• IRCCS Fondazione Stella Maris

  collaborator OTHER

• Istituto Giannina Gaslini

  collaborator OTHER

• University of Padova

  collaborator OTHER

• Maggiore Bellaria Hospital, Bologna

  collaborator OTHER

• IRCCS Eugenio Medea

  collaborator OTHER

• Fondazione Serena Onlus - Centro Clinico NeMO Milano

  collaborator OTHER

• Catholic University of the Sacred Heart

  lead OTHER

Principal Investigators

• Eugenio Mercuri, MD, PhD · Catholic University

Eligibility

Min Age

4 Years

Max Age

35 Years

Sex

MALE

Healthy Volunteers

No

Timeline & Regulatory

Start

2013-01-31

Primary Completion

2013-03-31

Completion

2015-05-31

Countries

• Italy

Study Locations