Bumetanide in Hypokalaemic Periodic Paralysis
Research type
Research Study
Full title
A randomised, double-blind, placebo-controlled, phase II clinical trial with a cross-over design assessing efficacy of a single dose of bumetamide in reducing focal attack severity in hypokalaemic periodic paralysis assessed using the McManis protocol.
IRAS ID
136368
Contact name
Doreen Fialho
Contact email
Sponsor organisation
University College of London
Eudract number
2013-004195-36
Research summary
Hypokalemic periodic paralysis (HypoPP) is a rare muscle disorder characterised by attacks of reversible limb weakness lasting from several hours to days. Patients usually have normal muscle strength in between attacks but some go on to develop more permanent weakness later in life. Even with optimal medical preventative treatment as well as lifestyle alteration many patients still experience significant number of attacks. This has an impact on quality of life as well as causing economic burden due to effects on employment and need for hospitalisation during severe attacks. Potassium supplement can be administered as treatment during an acute attack but response is often incomplete and there are no alternative agents for the acute stage. Recent experimental evidence using bumetanide in a mouse model of HypoPP has provided compelling evidence that it can abort paralytic attacks. Bumetanide is a diuretic (water tablet) currently in use for fluid overload and heart failure. During this study we will assess the efficacy of bumetanide on reducing the severity of a focal attack of weakness in a small hand muscle, which will be provoked by exercising this muscle for 5 minutes followed by complete rest of the hand. The response of the muscle will be monitored using electrical stimulation of the corresponding nerve and recording of the electrical muscle response (neurophysiological assessment). The protocol follows closely a well established test used frequently in the diagnosis of patients with periodic paralysis (McManis test). Patients with genetically confirmed HypoPP and a history of ongoing attacks are eligible to enter the study. Following screening procedures they will be asked to attend on two separate occasions to receive bumetanide or placebo in a random order. Each visit will last approximately six hours. The assessing researcher and patient will be blinded to which tablet the patient receives at each visit.
REC name
London - Westminster Research Ethics Committee
REC reference
14/LO/0774
Date of REC Opinion
14 Jul 2014
REC opinion
Further Information Favourable Opinion