Status:
COMPLETED
Weekly Steroids in Muscular Dystrophy
Lead Sponsor:
Northwestern University
Conditions:
Limb-girdle Muscular Dystrophy
Becker Muscular Dystrophy
Eligibility:
All Genders
18-65 years
Phase:
PHASE2
Brief 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 experi...
Detailed Description
Glucocorticoid (GC) steroids are a mainstay of therapy for Duchenne Muscular Dystrophy, where they have been shown to prolong ambulation in for DMD in random clinical trials (Gloss et al., 2016). Dosi...
Eligibility Criteria
Inclusion
- 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
- Ages 18-65 years
- EKG without evidence of prior infarct or atrial fibrillation done within 2 months of study initiation.
- Echocardiogram with LVEF \>25% done within 6 months of study initiation.
- Stable medications (same medication and dose) for the previous 3 months
- Stable pulmonary status for the previous 6 months (No change in FVC by more than 20% in the past 6-months)
Exclusion
- Diabetes
- BMI\>35 kg/m2
- Cardiac transplantation
- Myocardia Infarct in the past 2-years from screening
- Any history of tuberculosis
- Untreated or uncontrolled (medication and/or dose change in previous month from screening) hypertension
- A diagnosis of congestive heart failure
- A diagnosis of chronic kidney disease
- A diagnosis of untreated hypothyroidism
- The patient is believed to be at high risk of osteoporosis by the primary investigator
- Inability to provide consent
- Full time ventilator dependency
- Heart failure symptoms or LVEF \<25%
- Orthopedic surgery within the prior year or upcoming elective orthopedic surgery within the 6-months from Day 0.
- Inability to complete MRI (claustrophobia, metal implants)
- 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.
Key Trial Info
Start Date :
July 1 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2022
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT04054375
Start Date
July 1 2019
End Date
March 1 2022
Last Update
September 21 2023
Active Locations (1)
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1
Northwestern University
Chicago, Illinois, United States, 60611