A randomised, double-blind, placebo-controlled, phase II clinical trial with a cross-over design assessing efficacy of a single dose of bumetanide in reducing focal attack severity in hypokalaemic periodic paralysis (HypoPP) assessed using the McManis protocol
|Dr Doreen Fialho
|More information on the trial
HypoPP is an autosomal dominant muscle channelopathy with onset in the first or second decade of life. It is characterized by attacks of reversible flaccid paralysis lasting from several hours to days.
These patients may have frequent attacks of weakness interfering with daily activities and work. They are often hospitalized for intravenous potassium treatment causing a significant economic burden. They may also progress to chronic myopathy because there are no treatments available.
Studies of bumetanide in a mouse model of HypoPP have shown that it can stop paralytic attacks.
This is a phase II, double-blind, randomised, placebo-controlled cross-over, single-site study.
To assess the efficacy of bumetanide in reducing severity and duration of acute attacks of weakness in HypoPP patients.
We expect bumetanide to stop acute attacks of weakness faster and reduce their severity. This will reduce the likelihood of patients' hospitalization during severe attacks. Bumetanide will be added as an additional treatment to potassium intake during an attack.