Upper Limb Assessment in Duchenne Muscular Dystrophy

NCT02436720 | Unknown

• 1 other identifier: UCSCPUL
• Study Type: observational
• Target: 300
• Locations: 1 country
• Sites: 1

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.

Conditions

Duchenne Muscular Dystrophy

Outcome Measures

Primary Outcomes (1)

• 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

  2 years

Eligibility Criteria

• Age: 4 Years - 35 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

Study Population

Boys and adults affected by Duchenne muscular dystrophy

You may qualify if:

• proven DMD

You may not qualify if:

• severe mental retardation

Sponsors & Collaborators

• Catholic University of the Sacred Heart: lead
• Bambino Gesù Hospital: collaborator
• IRCCS National Neurological Institute "C. Mondino" Foundation: collaborator
• Azienda Ospedaliera San Giovanni Battista: collaborator
• University of Messina: collaborator
• Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta: collaborator
• University of Naples: collaborator
• IRCCS Fondazione Stella Maris: collaborator
• Istituto Giannina Gaslini: collaborator
• University of Padova: collaborator
• Maggiore Bellaria Hospital, Bologna: collaborator
• IRCCS Eugenio Medea: collaborator
• Fondazione Serena Onlus - Centro Clinico NeMO Milano: collaborator

Study Sites (1)

Catholic University

Rome, 00168, Italy

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Eugenio Mercuri, MD, PhD

  Catholic University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Pediatric neurology

Study Record Dates

• First Submitted: April 30, 2015
• First Posted: May 7, 2015
• Study Start: January 1, 2013
• Primary Completion: March 1, 2013
• Study Completion: May 1, 2015
• Last Updated: May 7, 2015

Record last verified: 2015-05

Locations

IT (1)