Duchenne Muscular DystrophyPhase 2NCT07766980
Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients
A Phase 2 study testing VM100 Nutritional Supplement in people with Duchenne Muscular Dystrophy and Duchenne Disease, run by University of Florida at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy in Children
- What is being tested?
- VM100 Nutritional Supplement
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 27 Jul 2026to Dec 2028
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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 locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Florida
Gainesville, Florida, 32610
Tanja Taivassalo, 352-294-8748, ttaivassalo@ufl.edu
Tanja Taivassalo
About this study
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).
Read the 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.
- Conditions
- Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy in Children, DMD, Muscular Dystrophy
- Being tested
- VM100 Nutritional Supplement
- Keywords
- DMD, Nutritional supplement, Quality of life, Cognitive function, Fatigue
- Medical subject headings
- Muscular Dystrophy, Duchenne, Tabes Dorsalis, Muscular Dystrophies, Fatigue
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VM100 Nutritional SupplementDrug
Also called VM100
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.
| Study group | Role | What they receive |
|---|---|---|
| VM100 Nutritional Supplement | Experimental | 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. Receives: Drug: VM100 Nutritional Supplement |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline, Weeks 2, 6, 8, and 10
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.
Measured: Baseline, Weeks 2, 6, 8, and 10
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.
Measured: Baseline, Weeks 2, 6, 8, and 10
Secondary outcomes (7)
Changes in Cognitive Function assessed by Trail Making Test.
Time required to complete the Trail Making Test, measured in seconds.
Measured: Baseline and Week 6; Week 10 if the assessment is conducted
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.
Measured: Baseline and Week 6; Week 10 if the assessment is conducted
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.
Measured: Baseline and Week 6
Blood Concentration of Creatine Kinase
Determine blood concentration of creatine kinase (CK), a biomarker of muscle damage, measured using analysis of blood samples.
Measured: Baseline and Week 6
Blood Concentration of Complete Blood Count
Determine laboratory values from the complete blood count with differential obtained from blood samples.
Measured: Baseline and Week 6
Total Cycling Time and Distance During Moderate-Intensity Cycle Exercise
Total cycling time and distance achieved during a moderate-intensity cycle exercise test using a recumbent cycle ergometer or Rifton adaptive tricycle.
Measured: Baseline and Week 6; Week 10 if the assessment is conducted
Percentage of Prescribed VM100 Doses Taken
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.
Measured: Throughout the 10-week study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2026
- Study start27 Jul 2026
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by University of FloridaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Tanja Taivassalo, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07766980
- Sponsor's protocol ID
- IRB202401626
- Status last verified
- Oct 2025
- First submitted
- 4 Aug 2026
- Last updated
- 8 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No