Effects of Lumbopelvic Stabilization-based Physiotherapy and Rehabilitation Training in Duchenne Muscular Dystrophy

NCT06643923 | Not Yet Recruiting

• 2 other identifiers: 2024232Lokman hekim university
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 1

Brief Summary

This study aims to examine the effectiveness of supervised lumbopelvic stabilization in relation to factors associated with lower urinary tract symptoms in children diagnosed with Duchenne Muscular Dystrophy (DMD) who have lower urinary tract dysfunction. Children aged between 8 and 12 years, at stages 1-4 according to the Vignos scale, and who have a score of 8.5 or higher on the Dysfunctional Voiding Symptom Score (DVSS), will be included in the study. Demographic information will be collected through a general assessment form, while lower urinary tract symptoms will be assessed using the DVSS, a researcher-developed evaluation form, and a three-day bladder diary. Bowel symptoms will be evaluated using the Rome IV criteria and a seven-day bowel diary. Physical performance will be assessed via the Timed Up and Go Test and Gower's Test, muscle strength using the microFET2 hand dynamometer, lumbar lordosis angle with a Bubble inclinometer, participation in daily living activities via the Barthel Index, and perceived well-being of both the child and the parent will be assessed using the Faces Rating Scale. Participants will be randomly assigned to two groups (Treatment and Active Control) using a block randomization method. In the Active Control group, children will receive only urotherapy education. In the Treatment group, children will receive supervised, online, lumbopelvic exercise-based physiotherapy and rehabilitation sessions, in addition to urotherapy, conducted by a physiotherapist. The sessions will last eight weeks, with a total of 24 sessions. At the end of the eight-week period, both groups will be re-evaluated using the same assessment methods. Intra-group and inter-group comparisons will be completed using appropriate analytical methods.

Conditions

Duchenne Muscular Dystrophy (DMD); Lower Urinary Track Symptoms

Keywords

Duchenne muscular dystrophy; lower urinary tract symptoms; urotherapy; lumbopelvic exercise; physiotherapy

Outcome Measures

Primary Outcomes (1)

• Lower urinary tract symptoms assessment

  Lower urinary tract symptoms will be assessed with the UIRS questionnaire. UIRS questions the frequency of lower urinary tract symptoms in the last month and measures the severity of lower urinary tract dysfunction. Akbal et al. stated that a score of 8.5 is the optimum threshold value and that this value can be used to determine whether there are functional voiding symptoms. This cut-off value will be used as the inclusion criterion in our study. In addition to the UIRS, a three-day bladder diary (voiding diary, frequency-volume chart) will be used to assess lower urinary tract symptoms. This diary allows recording bladder functions and facilitates follow-up by documenting them. In addition, it is a practical, repeatable, valid and reliable assessment method. Before the diaries are given, individuals will definitely be given training on how to fill out the diary and what to look for when filling out the diary.

  8 weeks

Secondary Outcomes (7)

• Bowel symptom assessment

  8 weeks

• Physical performance assessment

  8 weeks

• Muscle strength assessment

  8 weeks

• Lumbar lordosis angle assessment

  8 weeks

• Evaluation of participation in daily living activities

  8 weeks

Study Arms (2)

Treatment

EXPERIMENTAL

Both groups of participants will receive the same urotherapy education from the same therapist (Demet Öztürk). Following the education, both groups will be instructed to implement the lifestyle changes taught during the urotherapy education for a period of eight weeks. In addition to the education, the treatment group will receive online, supervised exercise sessions based on lumbopelvic stabilization exercises, administered by the physiotherapist. The exercise program has been designed according to the FITTEA principle, which is one of the key principles in exercise prescription.

Other: Exercise; Behavioral: Urotherapy

Active Control

ACTIVE COMPARATOR

Approximately 1.5 hours of urotherapy training will be given and the patient will be asked to apply the lifestyle changes learned in this training for 8 weeks.

Behavioral: Urotherapy

Interventions

Exercise OTHER

F (Frequency): 3 days per week, with a total of 24 sessions. I (Intensity): Low to moderate intensity exercises with progression, ensuring no fatigue is induced. T (Time): Each session will last 30 minutes, over a total period of 8 weeks. T (Type): Warm-up exercises Core stabilization exercises: Aimed at improving core stability. Cool-down exercises E (Enjoyment): Gamified exercises, with sessions conducted to music. A (Adherence): A follow-up system will be implemented, including reminder notes and regular sending of session links to ensure compliance.

Treatment

Urotherapy BEHAVIORAL

The urotherapy education program was developed by an expert physiotherapist and was piloted with the families of children with DMD (16 parents) prior to the study. Pre-test and post-test assessments demonstrated that the knowledge level of DMD families increased after the training. Additionally, results from the satisfaction survey indicated a high level of satisfaction among the families regarding the urotherapy education (Mean satisfaction score: 33.875/35.000, Minimum=28, Maximum=35). Therefore, it was deemed appropriate to deliver the same urotherapy presentation to the DMD families within the scope of this study. The training lasts approximately 1 hour and will be delivered to families via an online method. Each family will receive the education individually, and no group sessions will be conducted.

Active Control; Treatment

Eligibility Criteria

• Age: 8 Years - 12 Years
• Gender Eligibility Details: Duchenne muscular dystrophy is an X-linked recessive hereditary disease. For these reasons, women can only be carriers. Diagnosed individuals are only men.
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Having been diagnosed with DMD by a specialist physician,
• Having received a score of 8.5 or higher on the Voiding Disorders Symptom Score (VMSS),
• Being between the ages of 8-12,
• Continuing ambulation (Vignos Scale Stage 1-4),
• Volunteering to participate in the study by parents and reading and signing the informed consent form.

You may not qualify if:

• Having another neuromuscular disease diagnosed other than DMD and/or accompanying DMD,
• Having family and/or child having cooperation problems in completing the evaluations for any reason,
• Lack of internet infrastructure or technical infrastructure that may cause difficulty in continuing distance education/follow-up,
• Having difficulty in understanding and speaking the Turkish language.

Sponsors & Collaborators

• Lokman Hekim University: lead

Study Sites (1)

Lokman Hekim University

Ankara, Çankaya, 06530, Turkey (Türkiye)

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Lower Urinary Tract Symptoms

Interventions

Exercise

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; Urological Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Motor Activity; Movement; Musculoskeletal Physiological Phenomena; Musculoskeletal and Neural Physiological Phenomena

Central Study Contacts

Demet Öztürk, M.Sc.

CONTACT

+90-5068394288; demett.ozturkk@gmail.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Research Assistant

Model Details: Children and their families who have scored 8.5 or higher on the DVSS will be invited for further evaluation. Participants will be divided into two groups (Treatment and Active Control) using a 6-way block randomization method among the volunteer families.

Study Record Dates

• First Submitted: October 7, 2024
• First Posted: October 16, 2024
• Study Start: December 1, 2024
• Primary Completion: December 1, 2025
• Study Completion (Estimated): December 1, 2026
• Last Updated: October 16, 2024

Record last verified: 2024-10

Locations

TU (1)