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.
2+ Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - variant criteria varies by cohort
Industry/Sponsor:
Sarepta Therapeutics, Inc.
Ambulation:
Non-ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
6+ Years
Improving Muscle Growth & Protection
Actively Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
ITF Therapeutics LLC
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
All
7 Years to <12 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Excluding deletions including exons 1-11, 42-45, or 57-69
Industry/Sponsor:
Solid Biosciences Inc.
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
0 Years to <18 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Any deletion in exons 1 to 11 or 42 to 45 are excluded
Industry/Sponsor:
Solid Biosciences Inc.
Ambulation:
Varies by cohort
Steroid Use:
Cohorts 1,2,4,&5 - Stable course of steroids; Cohort 3 - N/A
Eligible Sexes:
Male
10+ Years
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Red Nucleus Enterprise Solutions, LLC
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
All ages
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
No specific requirement
Industry/Sponsor:
The Duchenne Registry
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
All
Next Steps:
4 Years to <19 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - amenable to exon 51 skipping
Industry/Sponsor:
Dyne Therapeutics
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
5+ Years
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Columbia University
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
All
Next Steps:
6+ Years
Improving Muscle Growth & Protection
Actively Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Italfarmaco
Ambulation:
Ambulatory or non-ambulatory
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Location:
United States, Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Serbia, Spain, United Kingdom
5 Years to <63 Years
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
No specific requirement
Industry/Sponsor:
University of Florida
Ambulation:
Varies by cohort
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
All ages
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
No specific requirement
Industry/Sponsor:
Vanderbilt University Medical Center
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
All
Next Steps:
18+ Years
Regulating Calcium Balance
Actively Recruiting
Therapeutic Approach:
Regulating Calcium Balance
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Sardocor Corp.
Ambulation:
No specific requirement
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
All ages
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Diagnosis of a neuromuscular disorder for self or family member
Industry/Sponsor:
Boston Children's Hospital
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
All
Next Steps:
1 Month to 30 Months
Reducing Inflammation
Actively Recruiting
Therapeutic Approach:
Reducing Inflammation
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Nationwide Children's Hospital
Ambulation:
No specific requirement
Steroid Use:
Steroid-naive
Eligible Sexes:
Male
4 Years to <19 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Amenable to exon 53 skipping
Industry/Sponsor:
Wave Life Sciences USA, Inc.
Ambulation:
Part A/B - Either; Part C - Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
2 Years to <8 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - variant fully contained between exons 45-55
Industry/Sponsor:
Precision BioSciences, Inc.
Ambulation:
Ambulatory
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
2+ Years
Reducing Inflammation
Actively Recruiting
Therapeutic Approach:
Reducing Inflammation
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Catalyst Pharmaceuticals, Inc.
Ambulation:
No specific requirement
Steroid Use:
Currently on treatment with Vamorolone (AGAMREE)
Eligible Sexes:
Male
8 to <19 Years
Improving Heart Function
Actively Recruiting
Therapeutic Approach:
Improving Heart Function
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Vanderbilt University Medical Center
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
4+ Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Excluding any deletion involving exon 8 and/or 9
Industry/Sponsor:
Sarepta Therapeutics, Inc.
Ambulation:
Ambulatory
Steroid Use:
No specific requirement
Eligible Sexes:
Male
0 Days to <3 Years
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
University of Rochester
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
All ages
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
No specific requirement
Industry/Sponsor:
Parent Project Muscular Dystrophy
Ambulation:
Either
Steroid Use:
No specific requirement
Eligible Sexes:
All
18+ Years
Improving Muscle Growth & Protection
Actively Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
University of Minnesota
Ambulation:
Non-ambulatory
Steroid Use:
No specific requirement
Eligible Sexes:
All
2 Years to 10 Years
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
No specific requirement
Industry/Sponsor:
Children's Hospital of Philadelphia
Ambulation:
Ambulatory
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
6+ Years
Improving Muscle Growth & Protection
Actively Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
University of Florida
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
10+ Years
Observational
Actively Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Vanderbilt University Medical Center
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
8 Years to 18+ Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Excluding any deletion of exon 8 and/or 9
Industry/Sponsor:
Sarepta Therapeutics, Inc.
Ambulation:
Cohort 1 - Non-ambulatory; Cohort 2 - Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
Location:
United States, Australia, Belgium, Canada, Germany, Hong Kong, Israel, Italy, Japan, South Korea, Spain, Sweden, Taiwan, United Kingdom
10+ Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Capricor Inc.
Ambulation:
No specific requirement
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
4 Years to <10 Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Edgewise Therapeutics, Inc.
Ambulation:
Ambulatory
Steroid Use:
Cohorts 1,2,3,4,5 - Stable course of steroids; Cohort 2NS - No steroids within 6 months
Eligible Sexes:
Male
8 Years to <18 Years
Enhancing Bone Health
Active, Not Recruiting
Therapeutic Approach:
Enhancing Bone Health
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Hoffmann-La Roche
Ambulation:
Group 1 - No specific requirement, Group 2 - Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
4 Years to <14 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - amenable to exon 51 skipping
Industry/Sponsor:
Sarepta Therapeutics, Inc.
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
Location:
United States, Colombia, Czechia, Denmark, France, Germany, Greece, Hungary, India, Ireland, Italy, Jordan, Mexico, Netherlands, New Zealand, Norway, Poland, Romania, Serbia, Slovenia, South Korea, Spain, Switzerland, Taiwan, Turkey (Türkiye), United Kingdom
2 Years to <5 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - variants in exons 18 to 58
Industry/Sponsor:
Insmed Gene Therapy LLC
Ambulation:
Ambulatory
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
0 Years to <25 Years
Observational
Active, Not Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
REGENXBIO Inc.
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
1 to <12 years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Part 1: Excluding variants in exons 1-17, Part 2 and 3: Excluding deletions or point mutations in exons 8, 9, and/or 10
Industry/Sponsor:
REGENXBIO Inc.
Ambulation:
Ambulatory
Steroid Use:
Varies by cohort and age
Eligible Sexes:
Male
18+ Years
Observational
Active, Not Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Known female carrier and/or mother of a child with Duchenne or Becker
Industry/Sponsor:
Nationwide Children's Hospital
Ambulation:
No specific requirement
Steroid Use:
No current steroid treatment
Eligible Sexes:
Female
Next Steps:
6+ Years
Observational
Active, Not Recruiting
Therapeutic Approach:
Observational
Variant Requirement:
Becker - Variant requirement varies by cohort
Industry/Sponsor:
Virginia Commonwealth University
Ambulation:
Varies by cohort
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
4+ Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Amenable to exon 53 skipping
Industry/Sponsor:
NS Pharma, Inc.
Ambulation:
No specific requirement
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
4 Years to <18 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Solid Biosciences Inc.
Ambulation:
Ambulatory for children, non-ambulatory for adolescents
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
Next Steps:
4 years to <16 years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - amenable to exon 50 skipping
Industry/Sponsor:
NS Pharma, Inc.
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
4 Years to <15 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Amenable to exon 44 skipping
Industry/Sponsor:
NS Pharma, Inc.
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
10+ years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - excludes exon 44 skip-amenable or deletion of exons 3-7
Industry/Sponsor:
Capricor Inc.
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
All
7 Years to <28 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Amenable to exon 44 skipping
Industry/Sponsor:
Avidity Biosciences, Inc.
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
Male
6 Years to <18 Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Edgewise Therapeutics, Inc.
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
7 Years to <10 Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Satellos Bioscience, Inc.
Ambulation:
Ambulatory
Steroid Use:
No specific requirement
Eligible Sexes:
Male
4 Years to <17 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - amenable to exon 51 skipping
Industry/Sponsor:
Dyne Therapeutics
Ambulation:
Ambulatory or non-ambulatory <2 years
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
5 to <11 Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
MED Institute Inc.
Ambulation:
Ambulatory
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
Next Steps:
7 Years to <13 Years
Improving Muscle Growth & Protection
Active, Not Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
University of Florida
Ambulation:
Ambulatory
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
18+ Years
Improving Muscle Growth & Protection
Not Yet Recruiting
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Duchenne - No specific requirement
Industry/Sponsor:
Vanderbilt University Medical Center
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
2+ Years
Restoring or Replacing Dystrophin
Enrolling by Invitation
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Excluding any deletion of exon 8 and/or 9
Industry/Sponsor:
Sarepta Therapeutics, Inc.
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
Male
All ages
Restoring or Replacing Dystrophin
Enrolling by Invitation
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Amenable to exon 45, 51 or 53 skipping
Industry/Sponsor:
Sarepta Therapeutics, Inc.
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
Male
Next Steps:
4+ Years
Restoring or Replacing Dystrophin
Enrolling by Invitation
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Excluding any deletion of exon 8 and/or 9
Industry/Sponsor:
Sarepta Therapeutics, Inc.
Ambulation:
No specific requirement
Steroid Use:
Stable course of steroids
Eligible Sexes:
Male
12+ Years
Improving Muscle Growth & Protection
Enrolling by Invitation
Therapeutic Approach:
Improving Muscle Growth & Protection
Variant Requirement:
Becker - no specific requirement
Industry/Sponsor:
Edgewise Therapeutics, Inc.
Ambulation:
No specific requirement
Steroid Use:
No current use
Eligible Sexes:
Male
Location:
United States, Australia, Belgium, Denmark, France, Germany, Israel, Italy, Netherlands, New Zealand, Spain, United Kingdom
2+ Years
Restoring or Replacing Dystrophin
Available
Therapeutic Approach:
Restoring or Replacing Dystrophin
Variant Requirement:
Duchenne - Amenable to exon 44 skipping
Industry/Sponsor:
Avidity Biosciences, Inc.
Ambulation:
No specific requirement
Steroid Use:
No specific requirement
Eligible Sexes:
Male