Efficacy of Kushmand Rasayana and Panchkarma therapies in managing ambulatory boys with Duchenne’s Muscular Dystrophy (DMD)- A Pilot Study
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- To evaluate the efficacy of Kushmand Rasayana and Panchkarma therapies in managing ambulatory boys with DMD aged 5 to 12 years over a period of 2 months as measured by NSAA scale
研究概览
简要总结
DMD is an inherited disorder caused by mutations in the dystrophin gene leading to progressive muscle weakness and wasting
Symptoms begin at 2 to 3 years of age with difficulty climbing stairs and frequent falls
In Ayurveda DMD is classified under inherited and genetic disorders affecting muscle tissue where vitiated Vata and impaired metabolic processes cause muscle degeneration and weakness
The prevalence of DMD is about 1 in 5000 male births. Most mutations are deletions, followed by duplications and point mutations often occurring in hotspot regions of the dystrophin gene
Currently there is no cure
Glucocorticoids can improve muscle strength temporarily but have significant side effects
With multidisciplinary management patients can survive into their forties
Ayurveda offers scope for supportive management through Panchakarma therapies and herbal medicines
The study aims to evaluate the effect of Kushmand Rasayana and Panchakarma in boys aged 5 to 12 years with DMD.
Objectives
Primary: Evaluate efficacy using North Star Ambulatory Assessment NSAA score
Secondary: Assess improvement in quality of life using PedsQL score, changes in inflammatory markers and muscle enzymes, and respiratory functions using spirometry
Study design
Type: Prospective pilot trial
Duration 3 months 2 months treatment plus 1 month follow u
Sample size: 30 boys
Location: AIIA New Delhi
Analysis: Paired t test
Interventions
Chitrakadi Vati for digestion and metabolism for 3 days
Kushmand Rasayana Avaleha 5 g for ages 5 to 8 years and 7 g for ages 9 to 12 years twice daily with lukewarm milk for 60 days
Panchakarma therapies including Udwartana powder massage for 3 days Sarvang Abhyanga oil massage and Swedana steam for 7 days and Matra Basti medicated enema with Chhagalyadi Ghrita for 7 days repeated in two courses with a 20 day gap
Inclusion criteria Male children aged 5 to 12 years diagnosed with DMD weighing at least 20 kg ambulant and able to walk at least 50 meters with stable cardiac and respiratory functions and parental consent
Exclusion criteria Allergy to treatment components concurrent participation in other trials planned surgery prior gene or cell therapy or any condition compromising safety
Ethical approval drug authentication and safety monitoring are ensured
Rescue medications will be allowed if required
The study seeks to validate traditional Ayurvedic therapies in improving quality of life delaying progression and providing integrative care for children with DMD
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 盲法
- None
入排标准
- 年龄范围
- 5.00 Year(s) 至 12.00 Year(s)（—）
- 性别
- Male
入选标准
- •Male patients aged 5 to 12 years already diagnosed with Duchenne muscular dystrophy weight atleast 20 kg at screening Ambulant patients able to walk at least 50 meters without assistance North Star Ambulatory Assessment NSAA total score atleast 40 percent of maximum Stable cardiac and respiratory functions Ability to comply with study procedures as determined by the investigator Patients taking steroids would be taken after a washout period of 30 days Written informed consent from parents OR legal guardians and assent from the child when appropriate.
排除标准
- •Allergy or hypersensitivity to any components used in Panchakarma therapy including medicated oils and herbal preparations Concurrent participation in any other interventional clinical trial or participation within 3 months prior to screening
- •Planned or expected spinal fusion surgery during the study period or previous spinal fusion surgery within the last 6 months Prior gene therapy or cell based therapy for DMD Any medical condition that in the investigators opinion might compromise patient safety or study results.
结局指标
主要结局
To evaluate the efficacy of Kushmand Rasayana and Panchkarma therapies in managing ambulatory boys with DMD aged 5 to 12 years over a period of 2 months as measured by NSAA scale
时间窗: 0,30,60,90 day
次要结局
- To assess improvement in Peds3 Quality of Life Scale
- To assess changes in inflammatory markers and muscle enzyme levels
- To assess changes in respiratory functions using spirometry
研究者
Dr Harshit Jain
ALL INDIA INSTITUTE OF AYURVEDA