Prevention of Scoliosis in Patients With Duchenne Muscular Dystrophy Using Portable Seat Device

Prevention of Scoliosis in Patients With Duchenne Muscular Dystrophy Using Portable Seat Device Devised to Maintain Lumbar Lordosis : 5 Year Follow up Study

This study will be conducted without blind method. The portable seat device devised to maintain lumbar lordosis will be made within 1 year after the loss of ambulation in the participants with Duchenne muscular dystrophy with prospective design.

In the control group, the presence of scoliosis will be calculated 5 years after the loss of ambulation in participants with Duchenne muscular dystrophy through analysis of retrospective medical records who had not been applied the portable seat device.

Study Overview

• Status: Recruiting
• Conditions: Duchenne Muscular Dystrophy; Scoliosis Neuromuscular; Lordosis Lumbar
• Intervention / Treatment: Device: Portable seat device devised to maintain lumbar lordosis

Detailed Description

In the retrospective data of Seoul National University Children's Hospital, the incidence of 5-year scoliosis in the control group in the past using steroids alone was 70% in patients with confirmed Duchenne muscular dystrophy. When applying the portable seat device (experimental group), the incidence of scoliosis is expected to decrease to 50% (ie, 35% because of 50% of 70%) compared with using steroid alone.

In this study, investigators will enroll 70 control subjects who did not use the portable seat device through retrospective data. The number of subjects required to show the difference between the significance level of 5% and the power of 80% was 19, and 28 participants with DMD are required considering 30% dropout rate.

• Study Type: Interventional
• Enrollment (Anticipated): 98
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: You Gyoung Yi, M.D., MSc; Phone Number: 82-10-9683-7582; Email: lyk861124@gmail.com

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of
    • Recruiting
    • Seoul National University Hospital
    • Contact: You Gyoung Yi, M.D.; Phone Number: 82-10-9683-7582; Email: lyk861124@gmail.com
  • Seoul, Korea, Republic of, 03080
    • Recruiting
    • Seoul National University
    • Contact: Hyung-Ik Shin, PhD; Phone Number: +82-02-2072-3218; Email: hyungik1@snu.ac.kr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 7 years to 15 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

Inclusion Criteria:

• Patients with the diagnosis of Duchenne muscular dystrophy diagnosed by genetic study were included.

  1. Within 1 year after loss of ambulation (Vignos scale 7 points or more)
  2. Condition without scoliosis
  3. Conditions that do not have physical (eg, cerebral palsy) and mental (eg, moderate or higher intellectual disability) comorbid conditions that will affect the use of postural seat device.

Exclusion Criteria:

1. Patients who do not agree to participate in this study
2. Patients not taking steroids
3. Patient with scoliosis

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental group; When loss of ambulation was observed in patients with Duchenne muscular dystrophy, portable seat device devised to maintain lumbar lordosis were applied within 1 year, and then compliance with the the device were evaluated at 6-month intervals for 5 years.	Device: Portable seat device devised to maintain lumbar lordosis; The posterior lumbar spine pads are used to maintain lumbar lordosis and fix it with the shoulder strap so that the hip does not slip forward (so that it does not become a sacral sitting posture). The seat is equipped with a 5 cm air filled villus cushion to relieve pressure on the ischial tuberosity when sitting for a long time. The portable seat device can be moved so that the DMD can sit on the device even when sitting in a wheelchair or a chair for home, school, or outdoors, so that the lumbar lordosis can be constantly maintained.
No Intervention: Control group; Analysis of retrospective medical records who had not been applied portable seat device devised to maintain lumbar lordosis

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of scoliosis	Frequency of scoliosis more than 10 degrees on spine x-ray on supine position	5 years after loss of ambulation

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of scoliosis	Comparison of cobb's angle on spine x-ray on supine position	"Day 0", "Month 6" "Month 12" "Month 18" "Month 24" "Month 30" "Month 36" "Month 42" "Month 48" "Month 54" "Month 60"

Collaborators and Investigators

• Sponsor: Seoul National University Hospital
• Investigators: Principal Investigator: You Gyoung Yi, M.D., MSc, Seoul National University Hospital

Study record dates

Study Major Dates

• Study Start (Actual): August 7, 2018
• Primary Completion (Anticipated): December 1, 2024
• Study Completion (Anticipated): December 1, 2024

Study Registration Dates

• First Submitted: July 25, 2018
• First Submitted That Met QC Criteria: August 1, 2018
• First Posted (Actual): August 2, 2018

Study Record Updates

• Last Update Posted (Actual): September 18, 2018
• Last Update Submitted That Met QC Criteria: September 15, 2018
• Last Verified: September 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Nutrition Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Deficiency Diseases; Malnutrition; Muscular Disorders, Atrophic; Spinal Diseases; Bone Diseases; Spinal Curvatures; Muscular Dystrophies; Scoliosis; Muscular Dystrophy, Duchenne; Lordosis; Swayback; Physiological Effects of Drugs; Central Nervous System Depressants; Peripheral Nervous System Agents; Sensory System Agents; Anesthetics; Anesthetics, Local; Benzocaine
• Other Study ID Numbers: 1806-171-955 SNUH

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No