Phase 2 N=20 Treatment

Weekly Steroids in Muscular Dystrophy

Limb-girdle Muscular Dystrophy · Becker Muscular Dystrophy

Bottom Line

View on ClinicalTrials.gov: NCT04054375 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Jul 2021

Primary outcome: Primary: Fasting Glucose — 93; 102 mg/dL

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Prednisone (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Northwestern University
Primary completion: Jun 2020

Outcome Measures

Outcome	Result	p-value
PRIMARY Fasting Glucose	93; 102	—
PRIMARY HbgA1c	5.2; 5.3	—
PRIMARY Fasting Lipid Profile	182; 185	—
PRIMARY Creatine Kinase	1574; 1047	—
PRIMARY Respiratory Changes	80; 79	—
SECONDARY Functional Assessments - NSAD Change	18.4; 18.6	—
SECONDARY 6 Minute Walk Test	386; 410	—
SECONDARY 10 Meter Run Timed	7.32; 6.67	—
SECONDARY Brooke Scale Score	3; 3	—
SECONDARY Vignos Scale Score	5; 5	—
SECONDARY Muscle Imaging	-14	—
SECONDARY Bone Density	-1.64; -1.65	—
SECONDARY Lean Mass %	37.5; 38.1	—
SECONDARY Functional Assessments - Upper Limb Strength	39; 41	—
SECONDARY Muscle Strength Test	3; 3	—

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

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 Criteria

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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04054375). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.