Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Changes in DMD Quality of Life Physical Symptoms Domain Score
研究概览
简要总结
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).
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
VM100 Nutritional Supplement
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.
干预措施: VM100 Nutritional Supplement (Dietary Supplement)
结局指标
主要结局
Changes in DMD Quality of Life Physical Symptoms Domain Score
时间窗: 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
时间窗: 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
时间窗: 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.
次要结局
- Changes in Cognitive Function assessed by Trail Making Test.(Baseline and Week 6; Week 10 if the assessment is conducted)
- Changes in Cognitive Function assessed by Controlled Oral Word Association (COWA) Test(Baseline and Week 6; Week 10 if the assessment is conducted)
- Blood Concentration of Comprehensive Metabolic Panel(Baseline and Week 6)
- Blood Concentration of Creatine Kinase(Baseline and Week 6)
- Blood Concentration of Complete Blood Count(Baseline and Week 6)
- Total Cycling Time and Distance During Moderate-Intensity Cycle Exercise(Baseline and Week 6; Week 10 if the assessment is conducted)
- Percentage of Prescribed VM100 Doses Taken(Throughout the 10-week study)