Weekly Steroids in Muscular Dystrophy
Condition: Limb-girdle Muscular Dystrophy; Becker Muscular Dystrophy
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Dystrophies, Limb-Girdle
Intervention: Prednisone
Intervention description: Subjects will be asked to take weekly GC oral prednisone dosed based on weight (1mg/kg for patients who weigh less than or equal to 70 kg and 0.75 mg/kg for patients who weigh more than 70 kg). Subjects will also be instructed to take their weekly prednisone on Mondays after their last meal between 7 and 9 PM
Intervention MeSH terms: Prednisone; Prednisolone
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 20.0
Sex: All
Minimum age: 18 Years
Maximum age: 65 Years
Healthy volunteers: No
Keywords: Steroids, Prednisone

Summary:

      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.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Patients with Becker muscular dystrophy or LGMD2A (CAPN3), LGMD 2B (DYSF), LGMD 2C
             (SGCG), LGMD2E (SGCB), LGMD2F (SGCD), LGMD 2I (FKRP), LGMD (ANO5). Genetic mutation or
             muscle biopsy staining required to confirm genetic subtype

          2. Ages 18-65 years

          3. EKG without evidence of prior infarct or atrial fibrillation done within 2 months of
             study initiation.

          4. Echocardiogram with LVEF >25% done within 6 months of study initiation.

          5. Stable medications (same medication and dose) for the previous 3 months

          6. Stable pulmonary status for the previous 6 months (No change in FVC by more than 20%
             in the past 6-months)

        Exclusion Criteria:

          1. Diabetes

          2. BMI>35 kg/m2

          3. Cardiac transplantation

          4. Myocardia Infarct in the past 2-years from screening

          5. Any history of tuberculosis

          6. Untreated or uncontrolled (medication and/or dose change in previous month from
             screening) hypertension

          7. A diagnosis of congestive heart failure

          8. A diagnosis of chronic kidney disease

          9. A diagnosis of untreated hypothyroidism

         10. The patient is believed to be at high risk of osteoporosis by the primary investigator

         11. Inability to provide consent

         12. Full time ventilator dependency

         13. Heart failure symptoms or LVEF <25%

         14. Orthopedic surgery within the prior year or upcoming elective orthopedic surgery
             within the 6-months from Day 0.

         15. Inability to complete MRI (claustrophobia, metal implants)

         16. Pregnant women at screening, women seeking to become pregnant, or men seeking to
             father a child within 6-months from Day 0 should not participate in this study.