A Phase 2 interventional study of Bumetanide and Placebo in Hypokalemic Periodic Paralysis, sponsored by University College, London. Terminated at 1 site in United Kingdom. Open to participants aged 18 Years to 64 Years. Per ClinicalTrials.gov, last updated 2018-02-07.
Sponsored by University College, London · Phase 2, Interventional, and Treatment
This is a randomised, double-blind, placebo-controlled phase II clinical trial with a cross-over design to investigate the efficacy of bumetanide in patients with hypokalemic periodic paralysis (HypoPP).
The aim is to assess the efficacy of bumetanide in reducing severity and duration of a focal attack of weakness in a hand muscle.
Twelve participants will be recruited.
Interested patients who provisionally meet inclusion/exclusion criteria will attend NHNN for a screening visit to check study eligibility and to have any questions relating to study participation answered. Each patient will undertake two assessment visits at approximately four weeks apart. Study participants will withhold carbonic anhydrase inhibitor medications for 72 hours prior to assessment visits as is standard for McManis testing and restart their routine treatment immediately after each visit. Participants will be admitted as an NHNN day case. Following baseline assessments a localised attack of weakness will be induced by isometric exercise of the abductor digit minimi (ADM) in the hand as per McManis protocol below. Participants will be randomly assigned to either bumetanide or placebo for the first visit. Identical appearing capsules will be prepared to blind both researcher and participant to treatment allocation. The assigned treatment will be taken by mouth at the onset of a focal attack defined as 40% decrement in ADM CMAP amplitude compared to the maximum CMAP amplitude recorded during or after the exercise. During the admission each patient will be monitored according to the research protocol. At the end of the assessment protocol the participant will be discharged home. The duration of each admission will be approximately 6 hours The second assessment will follow an identical protocol to the first, but with the other treatment administered.
750 studies on the registry are indexed under Paralysis; 133 are open to participants now.
This study's planned enrollment of 12 is below the median of 30 across 537 interventional studies indexed under Paralysis.
Browse Paralysis studies →University College, London is the lead sponsor of 632 studies on the registry; 145 are open to participants now.
Of its 6 completed or terminated interventional studies of FDA-regulated products, 2 (33%) have results posted.
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Exclusion Criteria:
Participants will be randomly assigned to either bumetanide or placebo for the first visit. The assigned treatment will be taken by mouth at the onset of a focal attack defined as 40% decrement in ADM CMAP amplitude compared to the maximum CMAP amplitude recorded during or after the exercise. The second assessment will follow an identical protocol to the first, but with the other treatment administered.
Participants will be randomly assigned to either bumetanide or placebo for the first visit. The assigned treatment will be taken by mouth at the onset of a focal attack defined as 40% decrement in ADM CMAP amplitude compared to the maximum CMAP amplitude recorded during or after the exercise. The second assessment will follow an identical protocol to the first, but with the other treatment administered.
Focal attack severity one hour after treatment
This will be measured as CMAP amplitude expressed as a percent of peak CMAP during or after the McManis exercise.
Time frame: The effect of treatment on focal attack severity one hour after treatment
Focal attack duration
This will be measured as the time between treatment administration until CMAP returns to 65% of peak CMAP within 4 hours following the treatment intake.
Time frame: 4 hours
The initial effect of treatment on severity of a focal attack
The effect of treatment on severity of a focal attack within the first two hours (0-2). This will be measured as CMAP amplitude (in percent compared to peak) area under the curve (AUC) from treatment administration until two hours post-treatment.
Time frame: The initial effect of treatment on severity of a focal attack within the first two hours post treatment
The late effect of treatment on severity of a focal attack
The effect of treatment on severity of a focal attack within the last 2 hours (3-4). This will be measured as CMAP amplitude (in percent) AUC from treatment administration during the third and the fourth hours post-treatment.
Time frame: The late effect of treatment on severity of a focal attack two to four hours post treatment
Safety of Bumetanide assessed by vital signs, physical exam, potassium levels and self-reported adverse events
Baseline instantaneous potassium measurements as well as laboratory measurements, vital signs (blood pressure/heart rate) and a physical exam including MRC score are done prior to exercise and IMP intake. During the first 4 hours following IMP intake vital signs (blood pressure/heart rate) and instantaneous serum potassium levels are measured frequently as per protocol. Any reported symptoms or adverse events are recorded. In addition intermittent electrophysiological recordings are taken from the non-exercised hand in order to identify the development of a major attack of paralysis early. At the end of the observation period (4 hours after IMP intake) serum potassium levels are measured by the local hospital laboratory and a physical exam is performed including an MRC score. Safety is also assessed by phone call evaluating adverse events reported by the participants and recorded in a diary occurring within 1 week following each visit.
Time frame: Safety of Bumetanide in HypoPP within 7 days of each study visit
This study is terminated, as verified in Feb 2018. You cannot join it, but the record below documents what was studied.
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Hypokalemic Periodic Paralysis
University College, London