Pulmonary and Upper Limb Functions in Duchenne Muscular Dystrophy

NCT03552874 | Completed

• 1 other identifier: GO 16/773
• Study Type: observational
• Target: 47
• Locations: 0 countries
• Sites: N/A

Brief Summary

Although it is known that the functions of pulmonary and upper limb is affected in late stage of Duchenne Muscular Dystrophy (DMD) negatively, the investigators do not have clear information about its early stage. The aim of this study was to investigate the differences in pulmonary and upper limb functions between children with DMD in early stage and healthy peers.

Conditions

Duchenne Muscular Dystrophy; Upper Limb Function; Respiratory Function Test

Outcome Measures

Primary Outcomes (2)

• Pulmonary Function Test (PFT)

  The pulmonary function tests of the participants were evaluated with a spirometry. The child was asked to perform the spirometric tests for three times with maximum effort. The child was motivated before each attempt for stronger and longer expiration.The result of Pulmonary function Test were recorded.

  5 minutes

• The Performance Upper Limb (PUL)

  Upper limb functions of participants were evaluated with The Performance of Upper Limb (PUL) scale. This scale, which was shown to be reliable in DMD, has 22 items in total. It consists of 3 different levels of function: high, mid, and distal. PUL also has four items determining timed performance.

  15 minutes

Study Arms (2)

Patient Group

Children whose ages between 5-10 years with Duchenne Muscular Dystrophy.

Other: The Performance of Upper Limb Questionnaire (PUL)

Healthy Group

Healthy peers

Other: The Performance of Upper Limb Questionnaire (PUL)

Interventions

The Performance of Upper Limb Questionnaire (PUL) OTHER

Also known as: Pulmonary Function Test (PFT)

Healthy Group; Patient Group

Eligibility Criteria

• Age: 5 Years - 10 Years
• Gender Eligibility Details: This study is gender based because Duchenne Muscular Dystrophy is only seen in males.
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)
• Sampling Method: Probability Sample

Study Population

Fourty seven participants including 31 children with DMD and 16 healthy peers were included

You may qualify if:

• children with DMD was;
• age between 5-10 years
• being on treatment with corticosteroids for more than 6 months
• being at Level I according to Brooke Upper and Lower Extremity Classification Systems
• Healthy peers;
• age between 5-10 years
• not having any diagnosed disease

You may not qualify if:

• Children has undergo surgery and disease affect upper extremity or pulmonary function in last six months.

Sponsors & Collaborators

• Hacettepe University: lead

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

Respiratory Physiological Phenomena

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

Intervention Hierarchy (Ancestors)

Circulatory and Respiratory Physiological Phenomena

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Department of Physiotherapy and Rehabilitation, Principal Investigator, Research Assistant

Study Record Dates

• First Submitted: May 25, 2018
• First Posted: June 12, 2018
• Study Start: January 1, 2017
• Primary Completion: July 31, 2017
• Study Completion: March 31, 2018
• Last Updated: June 12, 2018

Record last verified: 2018-06

Data Sharing

• IPD Sharing: Will not share