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Finding the Optimum Regimen for Duchenne Muscular Dystrophy
study id #: NCT01603407
condition: Duchenne Muscular Dystrophy
status: active, not recruitingpurpose:
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.
intervention: Prednisone, Deflazacort
mechanism of action: Glucocorticoid to delay decline in muscle strength
start date: January 2013
estimated completion: October 2019
phase of development: Phase 3
size / enrollment: 196
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.
- Three-dimensional (multivariate) outcome [Time Frame: See description below]
Three-dimensional outcome consisting of the following three components (each averaged over the Month 3, Month 6, Month 12, Month 18, Month 24, Month 30, and Month 36 visits):
• time to stand from lying (log-transformed)
• forced vital capacity
• subject/parent global satisfaction with treatment, as measured by the Treatment Satisfaction Questionnaire for Medication
- The North Star Ambulatory Assessment (NSAA) [Time Frame: Once during screening period (1 to 3 months prior to baseline visit), then again at Baseline visit (Month 0), Month 3, 6, 12, 18, 24, 30, 36, 42, 48, 54 and 60]
17 Item timed function tests to evaluate the motor ability in ambulant children with DMD. Timed Function Test Grading to differentiate those subjects with similarly fast times who may achieve a ceiling time Total score = Sum of all graded items. Of primary interest will be the average value of these outcomes over all post-baseline visits over the three year follow-up period
- 6 minute walk test [Time Frame: Once during screening period (1 to 3 months prior to baseline visit), then again at Baseline visit (Month 0), Month 3, 6, 12, 18, 24, 30 and 36]
Measures the total distance walked in 6 minutes and the number of falls averaged over all post-baseline follow-up visits through Month 36
- Range of motion (goniometry) [Time Frame: Once during screening period (1 to 3 months prior to baseline visit), then again at Baseline visit (Month 0), Month 3, 6, 12, 18, 24, 30 and 36]
Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade
- Regimen tolerance [Time Frame: Month 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60]
Assess the ability to tolerate the starting regimen of corticosteroids, defined as completing 3-5 years of follow-up on study medication with no deviation from the initially prescribed dosage level (increases in dosage band to accommodate growth and weight gain are allowed)
- Adverse event profile [Time Frame: Month 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60]
The occurrence and severity of the following predictable adverse events (i.e., known side effects of corticosteroids) will be recorded. Behavior problems, bone fractures, cataracts, cushingoid features, GI symptoms, hypertension, immune/adrenal suppression, slow growth (height restriction), skin changes, weight gain, diabetes
- Quality of life [Time Frame: Baseline visit (Month 0), Month 3, 6, 12, 18, 24, 30 and 36]
Measured by child self-report (only in children aged 5 years and over) and by proxy (parent(s)/guardian(s)) report for all children. Utilizing Generic Peds QoL (23 questions ) NMD Disease-specific module will be used (25 questions).
The average values of these outcomes over all post-baseline assessments during the three-year follow-up period will be of primary interest.
- Cardiac function Cardiac function [Time Frame: One to three months prior to the baseline visit, then every two years to the age of 10 years, and annually thereafter or at the onset of cardiac signs and symptoms and the year 3 visit]
Monitored by trans-thoracic echocardiogram and 12-lead ECG. The findings will be categorized as: normal; abnormal but not clinically significant;abnormal and clinically significant. The earliest definite, echo detectable impairment of left ventricular function is defined as ejection fraction < 55% and/or fractional shortening < 28%. Monitorred 12-lead ECG. If echocardiogram shows any impaired left ventricular function or evidence of regional motion abnormalities (posterior wall), the interval between evaluations will be reduced and treatment will be initiated.
• 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.
• 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.
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