DMD, Duchenne Disease, Duchenne Muscular Dystrophy, Muscular Dystrophy, Muscular Dystrophy in Children
Conditions
Keywords
DMD, Nutritional supplement, Quality of life, Cognitive function, Fatigue
Brief summary
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).
Detailed description
Ongoing efforts at therapeutic development for Duchenne muscular dystrophy (DMD) are aimed at genetic and pharmacologic strategies to mitigate muscle damage and loss of strength. However there is also an important and largely overlooked need to improve general mental and physical quality of life of DMD patients. It is estimated that between 20% to 80% of DMD patients use vitamins or nutritional supplements, but there is very little research on their efficacy. This may relate in part to poor bioavailability (the extent and rate at which active ingredients are absorbed and utilized by the body). This exploratory trial will investigate the preliminary efficacy of VM100, a nutritional supplement in boys with DMD. VM100 contains a combination of 10 ingredients specially formulated into a drink to increase absorption and bioavailability, and developed to support pathophysiology of DMD (inflammation, mitochondrial impairment, oxidative stress) in both skeletal muscle and brain. This study is predominantly home-based, where patients will take VM100 in the form of a drink, twice a day. Patients and their parent or caregiver will be asked to fill out a daily survey of supplement compliance, as well as questionnaires and an interview 5 times over the course of the study. These questionnaires will assess the impact of VM100 on quality of life for boys with DMD, with a focus on mental, cognitive, mood and social behavior-related measures, as well as physical symptoms such as endurance and general fatigue. Two blood draws will be obtained (before and 6 weeks after taking VM100) to assess impact on proinflammatory cytokines and markers of muscle damage. Pilot data generated from this study will be used to support a more robust, placebo-controlled trial including objective outcome measures and assessing potential of VM100 to synergize with other therapeutics.
Interventions
DIETARY_SUPPLEMENTVM100 Nutritional Supplement
VM100 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.
Sponsors
University of Florida
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Changes in DMD Quality of Life Physical Symptoms Domain Score | Baseline, Weeks 2, 6, 8, and 10 | 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 | Baseline, Weeks 2, 6, 8, and 10 | 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 | Baseline, Weeks 2, 6, 8, and 10 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Changes in Cognitive Function assessed by Trail Making Test. | Baseline and Week 6; Week 10 if the assessment is conducted | Time required to complete the Trail Making Test, measured in seconds. |
| Changes in Cognitive Function assessed by Controlled Oral Word Association (COWA) Test | Baseline and Week 6; Week 10 if the assessment is conducted | 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 | Baseline and Week 6 | Determined laboratory values from the comprehensive metabolic panel, including measures of metabolic and organ function, obtained from blood samples. |
| Blood Concentration of Creatine Kinase | Baseline and Week 6 | Determine blood concentration of creatine kinase (CK), a biomarker of muscle damage, measured using analysis of blood samples. |
| Blood Concentration of Complete Blood Count | Baseline and Week 6 | Determine laboratory values from the complete blood count with differential obtained from blood samples. |
| Total Cycling Time and Distance During Moderate-Intensity Cycle Exercise | Baseline and Week 6; Week 10 if the assessment is conducted | Total cycling time and distance achieved during a moderate-intensity cycle exercise test using a recumbent cycle ergometer or Rifton adaptive tricycle. |
| Percentage of Prescribed VM100 Doses Taken | Throughout the 10-week study | Percentage of prescribed VM100 doses taken during the study, calculated from daily compliance logs recorded by participants' parents or caregivers using the study REDCap database. |
Countries
United States
Contacts
CONTACTTanja Taivassalo
PRINCIPAL_INVESTIGATORTanja Taivassalo, MD
University of Florida
Outcome results
None listed