This pilot study will investigate the potential efficacy of VM100, a nutritional supplement specifically formulated for patients with DMD, on quality of life and physical symptoms. Twenty patients (aged 8 an over) will be enrolled to undergo a 10-week placebo-controlled intervention with VM100. Outcomes will include validated questionnaires and qualitative interview to assess impact on mental, cognitive and mood related measures, as well as endurance and fatigue).
Diagnosis of DMD confirmed by genetic report
Age 8 years or older.
Stable glucocorticoid and/or other medication regimen for at least 3 months before enrollment and throughout study participation.
Unstable medical conditions or significant concomitant illness.
Secondary condition affecting muscle function or metabolism (e.g., myasthenia gravis, endocrine disorders, mitochondrial disease).
Participation in another investigational clinical trial within the previous 3 months.
Single group
VM100 Nutritional Supplement
DrugVM100 is an investigational dietary supplement formulated specifically for individuals with Duchenne muscular dystrophy. It contains ten naturally derived ingredients formulated using a proprietary technology to enhance bioavailability. The supplement is intended to support mitochondrial function, reduce inflammation and oxidative stress, improve cognitive function and mood, and promote overall quality of life. Participants will self-administer the supplement orally twice daily during the intervention period.
Changes in DMD Quality of Life Physical Symptoms Domain Score
Duchenne Muscular Dystrophy Quality of Life (DMD-QoL) Physical Symptoms Domain Score, as measured by the validated DMD-QoL questionnaire. Scores will be reported according to the scoring system of the DMD-QoL instrument.
Changes in Mental Wellbeing assessed Warwick-Edinburgh Mental Well-being Scale Score
Total score on the Warwick-Edinburgh Mental Well-being Scale, a 14- or 7-item caregiver-reported measure of positive mental well-being, including optimism, energy, clear thinking, and social connectedness. Items are scored on a 1-5 scale.
Changes in Quality of Life assessed by PedsQL 4.0 Short Form (SF-15) Total Score
Total score on the PedsQL 4.0 Short Form (SF-15), Acute Version, completed by child self-report and parent proxy report. The questionnaire includes Physical, Emotional, Social, and School Functioning domains and assesses functioning over the previous 7 days.
Changes in Cognitive Function assessed by Trail Making Test.
Time required to complete the Trail Making Test, measured in seconds.
Changes in Cognitive Function assessed by Controlled Oral Word Association (COWA) Test
Total number of acceptable words generated during the Controlled Oral Word Association (COWA) Test within the specified time period.
Blood Concentration of Comprehensive Metabolic Panel
Determined laboratory values from the comprehensive metabolic panel, including measures of metabolic and organ function, obtained from blood samples.
Blood Concentration of Creatine Kinase
Determine blood concentration of creatine kinase (CK), a biomarker of muscle damage, measured using analysis of blood samples.
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