Open Label Safety and Efficacy of Once Weekly Steroid in Patients With LGMD and Becker Muscular Dystrophy

STU00208443

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.

2019-07-01

2020-06-01

2022-03-01

Completed

Early phase I

20

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.

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2023-09-21