A clinical trial to study the effects of Deflazacort at various dosages in boys with Duchenne muscular dystrophy.
- Conditions
- Health Condition 1: G710- Muscular dystrophy
- Registration Number
- CTRI/2019/02/017388
- Lead Sponsor
- Dr Renu Suthar
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 97
1.Boys with a diagnosis of DMD: ( two should present)
1With phenotypic characteristic symptoms or signs (waddling gait, Gowerâ??s sign, and calf hypertrophy)
2Elevated CK, and positive genetic analysis of dystrophin gene or muscle biopsy that demonstrated a clear alteration in dystrophin amount or distribution in the muscle
3.He should be ambulatory at the time of enrolment
4.Baseline 6MWD should be more than 150 meters
5.Steroid naive patients or who have received <6 months of any form of steroid therapy in immediate past
6.Willing for functional assessment and follow up in the clinic
1.Prior long-term use of oral GCs for >6 months
2.Active peptic ulcer disease or history of any gastrointestinal or perforation
3.Boys with DMD and autism/ intellectual disability/ fractures
4.Not willing for giving written informed consent
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To determine the mean change in 6MWD (in meters) from base lineTimepoint: at 24 ± 4 weeks
- Secondary Outcome Measures
Name Time Method To determine the mean change from base line to week 24 in time (in sec) for <br/ ><br>timed function tests and in <br/ ><br> muscle strength (lbs) for hand held myometery <br/ ><br> <br/ ><br>timed function tests <br/ ><br>10 meter walk test <br/ ><br>4 stair ascent time <br/ ><br>4 stair descent time <br/ ><br>gowers time <br/ ><br> <br/ ><br>Hand held myometery at biceps and quadriceps femorisTimepoint: at 24 ± 4 weeks