Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 196
- 试验地点
- 32
- 主要终点
- Forced Vital Capacity
研究概览
简要总结
The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. Using the results of this study, the investigators aim to provide patients and families with clearer information about the best way to take these drugs.
详细描述
Boys with Duchenne muscular dystrophy experience progressive muscle weakness as they grow up. Corticosteroids are currently the only medicine that has been shown to increase muscle strength in boys with DMD. Benefits include an increase in the length of time that boys could continue to walk, reduction in the development of curvature of the spine, a longer time of adequate breathing, and possible protection against the development of heart problems.
Doctors have tried different ways of prescribing corticosteroids in order to decrease undesirable side effects of the drug. No controlled, long-term study has ever looked at the effects of different corticosteroids to see which one improves strength the most and which one causes the fewest side effects, over a period of time. Different doctors in different countries prescribe the drugs in different ways, and some do not prescribe corticosteroids at all.
The FOR DMD study will enroll boys with DMD ages 4-7. The study will look at three ways of taking the following corticosteroids by the mouth to determine which increases muscle strength the most, and which causes the fewest side effects:
- Prednisone 0.75mg/kg/day
- Prednisone 0.75mg/kg/day switching between 10 days on and 10 days off treatment
- Deflazacort 0.9mg/kg/day.
The study will take place at 40 academic medical centers in the United States, Canada, United Kingdom, Germany and Italy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 4 Years 至 7 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Evidence of signed and dated informed consent form.
- •Confirmed diagnosis of Duchenne muscular dystrophy
- •Age greater than or equal to 4 years and less than 8 years old
- •Ability to rise independently from floor, from supine to standing
- •Willingness and ability to comply with scheduled visits, drug administration plan and study procedures
- •Ability to maintain reproducible FVC measurements.
排除标准
- •History of major renal or hepatic impairment, immunosuppression or other contraindications to corticosteroid therapy.
- •History of chronic systemic fungal or viral infections. Acute bacterial infection(including TB) would exclude from enrolment until the infection had been appropriately treated and resolved.
- •Diabetes mellitus.
- •Idiopathic hypercalcuria.
- •Lack of chicken pox immunity and refusal to undergo immunization.
- •Evidence of symptomatic cardiomyopathy at screening assessment (one to three months prior to the baseline visit). Asymptomatic cardiac abnormality on investigation would not be an exclusion.
- •Current or previous treatment (greater than four consecutive weeks of oral therapy) with corticosteroids or other immunosuppressive treatments for DMD or other recurrent indications (e.g., asthma), unless approved by FOR-DMD Team (i.e., concurrent participation in another allowed DMD trial).
- •Inability to take tablets, as assessed by the site investigator by the end of the screening period (the screening period ranges from one to three months prior to the baseline visit).
- •Allergy/sensitivity to study drugs or their formulations including lactose and/or sucrose intolerance.
- •Severe behavioral problems, including severe autism.
- •Previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow up will be correctly completed or impair the assessment of study results, in the judgment of the site investigator.
- •Weight of less than 13 kilograms.
- •Exposure to any investigational drug currently or within 3 months prior to start of study treatment.
研究组 & 干预措施
Daily prednisone
daily prednisone (0.75 mg/kg/day)
干预措施: Prednisone (Drug)
Intermittent prednisone
intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off)
干预措施: Prednisone (Drug)
Daily deflazacort
daily deflazacort (0.9 mg/kg/day
干预措施: Deflazacort (Drug)
结局指标
主要结局
Forced Vital Capacity
时间窗: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Forced vital capacity was measured during a spirometry test. Forced expiratory volume (FEV) measures how much air a person can exhale during a forced breath. Forced vital capacity (FVC) is the total amount of air exhaled during the FEV test.
Rise From the Floor Velocity
时间窗: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
Reciprocal of time to rise from the floor
Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score
时间窗: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits
The TSQM Global Satisfaction with Treatment is a 14-item questionnaire that ranges from 0 - 100 with higher scores indicating better outcomes.
次要结局
- Heart Rate(36 months)
- PR Interval(36 months)
- Participant Height(36 months)
- Number of Participants Who Tolerated the Regimen(3 years)
- Range of Motion (Goniometry) of Left Ankle(Average of Months 3, 6, 12, 18, 24, 30 and 36 visits)
- Range of Motion (Goniometry) of Right Ankle(Average of Months 3, 6, 12, 18, 24, 30 and 36 visits)
- Quality of Life - Parent(Average of Months 12, 24, and 36 visits)
- Quality of Life- Child(Average of Months 12, 24, and 36 visits)
- North Star Ambulatory Assessment (NSAA) Score(Average of Months 3, 6, 12, 18, 24, 30 and 36 visits)
- 6 Minute Walk Test(Average of Months 3, 6, 12, 18, 24, 30 and 36 visits)
- Left Ventricular Ejection Fraction Percent(36 months)
- Fractional Shortening Percent(36 months)
- Participant Weight(36 months)
- Participant Body Mass Index(36 months)
研究者
Robert Griggs, MD
Professor of Neurology
University of Rochester