Abstract Details

Title 24-week open label clinical trial to test safety and efficacy of oral weekly prednisone in adults with Becker (BMD) and Limb-Girdle Muscular Dystrophy (LGMD).

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) Neuromuscular and Clinical Neurophysiology (EMG) Posters (7:00 AM-5:00 PM)

Poster/Presentation
Number 006

Objective To determine if once weekly prednisone is safe and can benefit patients with BMD and LGMD.

Background

Corticosteroids improve mortality and ambulation in patients with Duchenne Muscular Dystrophy (DMD). High-dose, weekend steroids in DMD provides similar benefits with fewer adverse effects as compared to daily steroids. Animal models of LGMD treated with once-weekly steroids have previously shown to promote strength and lean muscle mass. We now conducted an open label safety and tolerability trial of weekly steroids in BMD and LGMD (NCT04054375).

Design/Methods Participants received prednisone at 0.75-1g/kg orally on Mondays after dinner for twenty-four weeks. Participants completed functional assessments, serum chemistry and lipid profiles, whole body DEXA scans, and muscle MRI imaging at baseline and end of study.

Results Twenty patients completed 24-weeks of once-weekly prednisone (1 BMD, 19 LGMD; 13 males, 7 females; ages 18-63 years old). Both ambulatory and non-ambulatory patients were enrolled to represent the entire disease spectrum. There was no significant negative impact on body weight, blood pressure, forced vital capacity, bone density or serum chemistries/lipids. Serum CK significantly decreased and was accompanied by an increase in lean muscle mass measured by whole body DEXA scans. Ambulatory patients (n=6) experienced a trend toward improvement in 6-minute walk distance and time to run 10 meters. Once-weekly prednisone was overall well tolerated with the most frequent side effects being increased anxiety on Monday nights and facial acne. All but one participant chose to continue to take weekly steroids after completing the study.

Conclusions

Once weekly steroids, given as 0.75-1gm/Kg was well tolerated in BMD and LGMD. Additional multicenter randomized clinical trials are needed to better understand how weekly steroids may benefit patients with BMD and LGMD.

Authors/Disclosures
Aaron Zelikovich, MD PRESENTER	Dr. Zelikovich has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genentech.
	No disclosure on file
	No disclosure on file
Senda Ajroud-Driss, MD, F�鶹��ýӳ�� (Northwestern University)	Dr. Ajroud-Driss has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Ajroud-Driss has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Astrazeneca. Dr. Ajroud-Driss has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. The institution of Dr. Ajroud-Driss has received research support from NIH. The institution of Dr. Ajroud-Driss has received research support from University of Minesota/FDA. The institution of Dr. Ajroud-Driss has received research support from Edgewise Therapeutics. The institution of Dr. Ajroud-Driss has received research support from Biogen. The institution of Dr. Ajroud-Driss has received research support from Woolsey Pharmaceuticals. The institution of Dr. Ajroud-Driss has received research support from Uniqure Biopharma. The institution of Dr. Ajroud-Driss has received research support from Coya Therapeutics. The institution of Dr. Ajroud-Driss has received research support from Novartis. The institution of Dr. Ajroud-Driss has received research support from Alnylam pharmaceuticals. The institution of Dr. Ajroud-Driss has received research support from Healey and AMG center for ALS . The institution of Dr. Ajroud-Driss has received research support from les Turner ALS Foundation. The institution of Dr. Ajroud-Driss has received research support from Hopewell. The institution of Dr. Ajroud-Driss has received research support from Peripheral neuropathy foundation. Dr. Ajroud-Driss has received publishing royalties from a publication relating to health care.

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