Nutritional Status and Therapy in DMD Patients

November 20, 2025 updated by: Betül ULU, Ankara University

Determination of Nutritional Status of Duchenne Muscular Dystrophy Patients and Evaluation of the Effectiveness of Disease-Specific Nutrition Therapy

Brief Summary

The goal of this clinical trial is to evaluate the impact of a disease-specific, individualized diet on the nutritional status and functional abilities of Duchenne muscular dystrophy (DMD) patients. The study will focus on children aged 4-8 years residing in Ankara, Turkey.

Key questions the investigators aim to answer:

Can a tailored dietary intervention improve the nutritional status of DMD patients? Does a specialized diet positively impact the functional abilities of DMD patients, as measured by the North Star Ambulation Assessment (NSAA)? Participants will undergo a comprehensive nutritional assessment, including anthropometric measurements, and will receive individualized dietary counseling. The intervention will focus on optimizing energy, protein, calcium, and fluid intake, as well as addressing the potential side effects of corticosteroid therapy.

The primary outcome measure will be changes in nutritional status, as assessed by anthropometric measurements. Secondary outcome measures will include changes in functional abilities as measured by the NSAA and quality of life assessments.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Study Objective This study aims to investigate the efficacy of a disease-specific, individualized dietary intervention in improving the nutritional status and functional abilities of Duchenne Muscular Dystrophy (DMD) patients aged 4-8 years residing in Ankara, Turkey.

Study Design This is a prospective, interventional study. Intervention

A 12-week individualized dietary intervention will be implemented for each participant. The intervention will focus on:

  • Energy Intake: Caloric needs will be calculated based on the patient's age, weight, and activity level, taking into account the potential impact of corticosteroid therapy on energy expenditure.
  • Protein Intake: Protein requirements will be met through a balanced diet, aiming to optimize muscle protein synthesis.
  • Calcium and Vitamin D Intake: Adequate intake of these nutrients will be emphasized to support bone health and prevent osteoporosis.
  • Fluid Intake: Fluid intake will be monitored to prevent dehydration and constipation.
  • Carbohydrate Intake: A low-glycemic index diet will be recommended to manage blood glucose levels and insulin resistance, especially in patients on corticosteroid therapy.
  • Sodium Intake: Sodium intake will be restricted to minimize fluid retention and hypertension.

Outcome Measures Changes in anthropometric measurements (body weight, height, BMI, skinfold measurements) Changes in functional abilities as assessed by the North Star Ambulation Assessment (NSAA) Changes in functional abilities as assessed by handgrip strenght Changes in micro and micro nutrient intake as assessed by three-day food consumption record Data Collection Data will be collected through face-to-face interviews with parents or primary caregivers and patients with DMD. After the first interview, a second interview will be conducted 12 weeks after the personalized diet has been planned.

Baseline Assessment:

  • Demographic information
  • Medical history
  • Nutritional assessment (anthropometric measurements, dietary intake assessment)
  • Functional assessment (NSAA)

Follow-up Assessments:

  • Anthropometric measurements
  • Functional assessment (NSAA)
  • Dietary intake assessment Data Analysis Statistical analysis will be performed using appropriate statistical software. Descriptive statistics will be used to summarize the baseline characteristics of the participants. Parametric or non-parametric tests will be used to compare pre- and post-intervention outcomes, as appropriate.

Ethical Considerations The study will be conducted in accordance with the Declaration of Helsinki and approved by the local ethics committee. Informed consent will be obtained from the parents or legal guardians of the participants.

By addressing the specific nutritional needs of DMD patients, this study aims to improve their overall health and quality of life.

Study Type

Interventional

Enrollment (Actual)

9

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Turkey (Türkiye)
    • Çankaya
      • Ankara, Çankaya, Turkey (Türkiye), 06530
        • DMD Aileleri Derneği

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

Clinical diagnosis of Duchenne Muscular Dystrophy (DMD) Must be between 4 and 8 years old Able to ambulate independently Must be residing in Ankara province, Turkey Must be willing to participate and adhere to the dietary intervention, as confirmed by both the patient and their legal guardian(s) Must be currently receiving corticosteroid therapy

Exclusion Criteria:

Inability to read and write in Turkish Wheelchair-bound or unable to ambulate independently Significant liver or kidney dysfunction Scheduled for major surgery within 6 months of study enrollment Symptoms of dysphagia and swallowing difficulties Significant respiratory distress or respiratory insufficiency requiring mechanical ventilation.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Supportive Care
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Dietary Intervention
The participants of the arm will follow diet program specially prepared for them for 12 weeks.
Other: Individualised dietary intervention
  • Energy Intake: Caloric needs will be calculated based on the patient's age, weight, and activity level, taking into account the potential impact of corticosteroid therapy on energy expenditure.
  • Protein Intake: Protein requirements will be met through a balanced diet, aiming to optimize muscle protein synthesis.
  • Calcium and Vitamin D Intake: Adequate intake of these nutrients will be emphasized to support bone health and prevent osteoporosis.
  • Fluid Intake: Fluid intake will be monitored to prevent dehydration and constipation.
  • Carbohydrate Intake: A low-glycemic index diet will be recommended to manage blood glucose levels and insulin resistance, especially in patients on corticosteroid therapy.
  • Sodium Intake: Sodium intake will be restricted to minimize fluid retention and hypertension.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Anthropometric measurements
Time Frame: From enrollment to the end of diet at 12 weeks
Changes in body weight (kg). Body weight will be measured with an electronic scale sensitive to 0.1 kilograms.
From enrollment to the end of diet at 12 weeks
Anthropometric measurements
Time Frame: From enrollment to the end of diet at 12 weeks
Changes in skinfold thickness (triceps, biceps, subscapular, and suprailiac) (in millimeters) Skinfold thickness will be compared to the World Health Organization's percentile and Z score charts to determine adequacy, deficiency, or excess.
From enrollment to the end of diet at 12 weeks
Anthropometric measurements
Time Frame: From enrollment to the end of diet at 12 weeks
Changes in height (cm). Height is measured with the individual standing upright and the head in the Frankfurt plane (the orbitale point and tragion point are set to be on the horizontal plane), while the vertical distance from the ground to the vertex, which is the highest point of the head, will be measured with a tape measure on a fixed plane.
From enrollment to the end of diet at 12 weeks
Anthropometric measurements
Time Frame: From enrollment to the end of diet at 12 weeks

Changes in Body Mass Index (BMI)(kg/m²). BMI will be obtained by dividing the particioant's body weight in kg by the square of the height in meters.

Body mass index will be evaluated with the World Health Organization's percentile and Z score charts.
From enrollment to the end of diet at 12 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Functional Ability
Time Frame: From enrollment to the end of diet at 12 weeks
Changes in the North Star Ambulation Assessment (NSAA) score meausered by a specialist physiotherapist. The total score varies between 0-34. A higher score indicates better ambulation and motor function.
From enrollment to the end of diet at 12 weeks
Functional Ability
Time Frame: From enrollment to the end of diet at 12 weeks
Changes in hand grip strength measured by hand dynamometry (in kilograms)
From enrollment to the end of diet at 12 weeks

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Dietary Adherence
Time Frame: From enrollment to the end of diet at 12 weeks
After the dietary intervention, 3-day food consumption records will be taken again. The 3-day food consumption records before and after the dietary intervention will be examined. The compatibility of the latest consumption records with the macro and micronutrients of the recommended diet and the differences between the first 3-day food consumption record will be examined. The computer program called BeBIS will be used for the analysis of macro and micronutrients.
From enrollment to the end of diet at 12 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ankara University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 18, 2024

Primary Completion (Actual)

February 20, 2025

Study Completion (Actual)

February 21, 2025

Study Registration Dates

First Submitted

November 5, 2024

First Submitted That Met QC Criteria

November 8, 2024

First Posted (Actual)

November 12, 2024

Study Record Updates

Last Update Posted (Actual)

November 21, 2025

Last Update Submitted That Met QC Criteria

November 20, 2025

Last Verified

November 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 111-659-22

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

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Duchenne Muscular Dystrophy (DMD)

Clinical Trials on Individualised dietary intervention

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Italy

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe