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Abstract Details

Long-Term Efficacy of Dichlorphenamide for the Treatment of Primary Periodic Paralysis (PPP)
Neuromuscular and Clinical Neurophysiology (EMG)
P5 - Poster Session 5 (5:30 PM-6:30 PM)
12-008
To evaluate the long-term (up to 61 weeks) efficacy of dichlorphenamide (DCP) for PPP.
Administration of DCP for 9 weeks is efficacious for reducing weekly attack rates in PPP. A pooled analysis of patients with hyperkalemic PPP (HYP) or hypokalemic PPP (HOP) was conducted to evaluate long-term DCP efficacy.
Patients in the randomized, double-blind, placebo-controlled HYP/HOP trial were randomly assigned to receive DCP (current DCP dose if taking before study start or 50 mg twice daily) or placebo (PBO) for 9 weeks during a double-blind phase. Patients then entered a 52-week open-label phase: patients in DCP group continued DCP double-blind dose (DCP/DCP population), and patients in PBO group switched to DCP 50 mg twice daily (PBO/DCP population). Changes from baseline to week 61 and from weeks 9-61 in attack rates and severity-weighted attack rates were analyzed for within- and between-group median differences.
63 patients were treated (36 in DCP/DCP group and 27 in PBO/DCP group); 47 (74.6%) completed 61 weeks. Median weekly attack rate significantly improved from baseline to week 61 in the DCP/DCP group (1.75 vs 0.00 at week 61; P<0.0001 [93.8% reduction]) and in the PBO/DCP group (2.25 vs 0.25 at week 61; P=0.01 [75.0% reduction]). Median severity-weighted attack rate also significantly improved from baseline to week 61 in both groups: DCP/DCP (3.25 vs 0.00; P<0.0001 [97.1% reduction]) and PBO/DCP (5.88 vs 0.75; P=0.01 [80.8% reduction]). There were no significant between-group differences for change from baseline to week 61 between PBO/DCP and DCP/DCP groups. The significant reduction in median weekly attack rate from week 9 (start of DCP treatment) to week 61 (1.78 vs 0.19; P=0.049) in PBO/DCP population further supports the efficacy of DCP previously demonstrated during weeks 2-9.
Long-term DCP treatment was efficacious and provided durable reduction in attack frequency and severity in patients with PPP.
Authors/Disclosures
Nicholas E. Johnson, MD, FAAN (Virginia Commonwealth University)
PRESENTER 		Dr. Johnson has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sarepta Therapuetics. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne Therapeutics. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vertex Pharma. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avidity. Dr. Johnson has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Arthex. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Juvena. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Rgenta. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AskBio. Dr. Johnson has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Johnson has received personal compensation in the range of $10,000-$49,999 for serving as an officer or member of the Board of Directors for Evolyra Therapeutics . Dr. Johnson has stock in Angle Therapeutics. Dr. Johnson has stock in Juvena Therapeutics. Dr. Johnson has stock in Evolyra Therapeutics. The institution of Dr. Johnson has received research support from Novartis. The institution of Dr. Johnson has received research support from Takeda . The institution of Dr. Johnson has received research support from AMO Pharma. The institution of Dr. Johnson has received research support from Sarepta. The institution of Dr. Johnson has received research support from Dyne Therapeutics. The institution of Dr. Johnson has received research support from AskBio. The institution of Dr. Johnson has received research support from Pepgen. The institution of Dr. Johnson has received research support from ML Bio. The institution of Dr. Johnson has received research support from Vertex. The institution of Dr. Johnson has received research support from Arthex. The institution of Dr. Johnson has received research support from Avidity Biosciences. The institution of Dr. Johnson has received research support from Edgewise. Dr. Johnson has received intellectual property interests from a discovery or technology relating to health care.
Jeffrey Statland, MD (University of Kansas Medical Center) Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arrowhead. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ML Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Epic Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Armatus . Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kate Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vita Therapeutics. Dr. Statland has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dyne Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avidity . Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum Therapeutics. Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vertex . The institution of Dr. Statland has received research support from NIH. The institution of Dr. Statland has received research support from FSHD Society. The institution of Dr. Statland has received research support from Friends of FSH Research. The institution of Dr. Statland has received research support from FSHD Canada. The institution of Dr. Statland has received research support from MDA.
Fredric J. Cohen, MD (Strongbridge Biopharma) No disclosure on file
Robert C. Griggs, MD, FAAN (University of Rochester Department of Neurology) Dr. Griggs has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Strongbridge Pharmaceuticals. Dr. Griggs has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Idera Pharmaceuticals. Dr. Griggs has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Solid Biopharma. Dr. Griggs has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Santhera Pharmaceuticals. Dr. Griggs has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elsevier. Dr. Griggs has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Lippincott. The institution of Dr. Griggs has received research support from PTC Pharmaceuticals. The institution of Dr. Griggs has received research support from Sarepta Pharmaceuticals. The institution of Dr. Griggs has received research support from National Institutes of Health. The institution of Dr. Griggs has received research support from Santhere Pharmaceuticals. Dr. Griggs has received personal compensation in the range of $500-$4,999 for serving as a Study section member with National Institutes of Health. Dr. Griggs has a non-compensated relationship as a Board of Directors;Chair Research Advisory Committee with American Brain Foundation that is relevant to AAN interests or activities.