welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!
enrolling by invitation

Weekly Steroids in Muscular Dystrophy

key information

study id #: NCT04054375

condition: Limb-girdle Muscular Dystrophy, Becker Muscular Dystrophy

status: enrolling by invitation


The purpose of this study is to evaluate the safety and efficacy of oral weekly glucocorticoid steroids in patients with Becker Muscular Dystrophy (BMD), an inherited disorder in which patients experience weakness of the legs and pelvis, and Limb Girdle Muscular Dystrophy (LGMD), an inherited disorder in which patients experience progressive muscular weakness predominately in their hip and shoulders. The primary objective is safety which we the investigators will measure using laboratory testing and forced vital capacity (FVC), a breathing test that measures the strength of your lungs. The secondary objective is efficacy which will be measured by a change in MRI muscle mass, improved muscle performance, and quality of life.

The investigators hypothesize that patients who receive oral weekly glucocorticoid steroids will have improviements in strength and quality of life compared to their baseline. Furthermore, the investigators anticipate that oral weekly glucocorticoid steroids will not have significant adverse impact on patients.

intervention: Prednisone

mechanism of action: Glucocorticoid to delay decline in muscle strength; Antioxidant to reduce inflammation

results: https://clinicaltrials.gov/ct2/show/results/NCT04054375

last updated: December 04, 2019