Pulmonary and Upper Limb Functions in Duchenne Muscular Dystrophy

• ClinicalTrials.gov Identifier: NCT03552874
• Recruitment Status: Completed
• First Posted: June 12, 2018
• Last Update Posted: June 12, 2018
• Sponsor: Hacettepe University
• Information provided by (Responsible Party): Numan Bulut, Hacettepe University

Study Description

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.

Condition or disease	Intervention/treatment
Duchenne Muscular Dystrophy; Upper Limb Function; Respiratory Function Test	Other: The Performance of Upper Limb Questionnaire (PUL)

Study Design

• Study Type: Observational
• Actual Enrollment: 47 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: The Comparison of Pulmonary and Upper Extremity Functions Between Children With Duchenne Muscular Dystrophy and Healthy Peers
• Actual Study Start Date: January 1, 2017
• Actual Primary Completion Date: July 31, 2017
• Actual Study Completion Date: March 31, 2018

Groups and Cohorts

Group/Cohort	Intervention/treatment
Patient Group; Children whose ages between 5-10 years with Duchenne Muscular Dystrophy.	Other: The Performance of Upper Limb Questionnaire (PUL); Other Name: Pulmonary Function Test (PFT)
Healthy Group; Healthy peers	Other: The Performance of Upper Limb Questionnaire (PUL); Other Name: Pulmonary Function Test (PFT)

Outcome Measures

Primary Outcome Measures :

1. Pulmonary Function Test (PFT) [ Time Frame: 5 minutes ]
   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.
2. The Performance Upper Limb (PUL) [ Time Frame: 15 minutes ]
   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.

Eligibility Criteria

• Ages Eligible for Study: 5 Years to 10 Years (Child)
• Sexes Eligible for Study: Male
• Gender Based Eligibility: Yes
• Gender Eligibility Description: This study is gender based because Duchenne Muscular Dystrophy is only seen in males.
• Accepts Healthy Volunteers: Yes
• Sampling Method: Probability Sample

Study Population

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

Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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

Contacts and Locations

No Contacts or Locations Provided

More Information

• Responsible Party: Numan Bulut, Department of Physiotherapy and Rehabilitation, Principal Investigator, Research Assistant, Hacettepe University
• ClinicalTrials.gov Identifier: NCT03552874
• Other Study ID Numbers: GO 16/773
• First Posted: June 12, 2018
• Last Update Posted: June 12, 2018
• Last Verified: June 2018

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked