Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients

University of Florida

Status: Enrolling

Conditions: DMD; Duchenne Disease; Muscular Dystrophy in Children; Muscular Dystrophy; Duchenne Muscular Dystrophy

Treatments: Dietary Supplement: VM100 Nutritional Supplement

Study type: Interventional

Funder types: Other

Identifiers

NCT07766980

IRB202401626

Details and patient eligibility

About

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).

Full 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.

Enrollment: 20 estimated patients

Sex: Male

Ages: 6+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

VM100 Nutritional Supplement

Experimental group

Description:

Participants will receive the VM100 nutritional supplement orally twice daily for 10 weeks, consisting of 6 weeks of supplementation, followed by a 2-week washout period without supplementation, and a final 2 weeks of supplementation.

Treatment:

Dietary Supplement: VM100 Nutritional Supplement