Bumetanide in Hypokalaemic Periodic Paralysis

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. - Bumetanide in Hypokalaemic Periodic Paralysis

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-004195-36-GB

Enrollment

Registered

2014-06-04

Start date

2014-07-21

Completion date

Unknown

Last updated

2019-06-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hypokalaemic Periodic Paralysis MedDRA version: 17.0 Level: PT Classification code 10016208 Term: Familial periodic paralysis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Trade Name: Bumetanide Product Name: 2mg Overencapsulated Bumetanide Pharmaceutical Form: Capsule INN or Proposed INN: Bumetanide CAS N

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1) At least 18 years of age; 2) Diagnosis of genetically confirmed HypoPP; 3) Clinical symptoms or signs of active symptomatic disease (at least 1 attack in last 12 months); 4) Practicing an acceptable method of birth control for the duration of the trial. Methods of birth control for women and men are addressed on Patient Information Sheet and on section section 11.4.5 of this protocol; Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 12 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0

Exclusion criteria

Exclusion criteria: 1) Inability or unwillingness to provide informed consent; 2) People older than 64 years old; 3) Other conditions causing non-dominant hand weakness which could interfere with study measurements (e.g. due to a stroke, trauma or arthritis); 4) Patients with a history of cardiac disease, renal failure or hepatic disease. Note: abnormalities in serum transaminases are common in people with HypoPP as they arise from skeletal muscle rather than any specific liver abnormality. Consequently, raised serum bilirubin >20% above the baseline value will be used to identify abnormal liver function; 5) Women who are pregnant or breast-feeding; 6) Patients with a previous history of diabetes, porphyria, symptomatic hypotension, prostatic hypertrophy or difficulty with micturition, or allergy to sulfonamides or thiazides; 7) Patients on lithium; 8) Patients known to be allergic to bumetanide or its excipients; 9) Patients with a history of inadequately treated Addison's disease; 10) Patients participating in another interventional trial in the previous 1 month.

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the efficacy of bumetanide in reducing the severity of a focal attack of muscle weakness affecting a small hand muscle one hour after attack onset in hypokalaemic periodic paralysis.; Secondary Objective: 1) To assess the efficacy of bumetanide in reducing the duration of a focal attack; 2) To assess the efficacy of bumetanide in reducing the severity of a focal attack early (0-2hr) after treatment administration; 3) To assess the efficacy of bumetanide in reducing the severity of a focal attack late (2-4hr) after treatment administration; 4) To assess the safety of bumetanide in hypokalaemic periodic paralysis; ;Primary end point(s): The primary outcome measure is focal attack severity one hour after treatment administration. Treatment will be administered when the CMAP amplitude reaches 60% of its peak value.;Timepoint(s) of evaluation of this end point: This will be measured as CMAP amplitude expressed as a percent of peak CMAP during or after the McManis exercise 1 hour after treatment administration.	—

Measure

Time frame

Main Objective: To assess the efficacy of bumetanide in reducing the severity of a focal attack of muscle weakness affecting a small hand muscle one hour after attack onset in hypokalaemic periodic paralysis.; Secondary Objective: 1) To assess the efficacy of bumetanide in reducing the duration of a focal attack; 2) To assess the efficacy of bumetanide in reducing the severity of a focal attack early (0-2hr) after treatment administration; 3) To assess the efficacy of bumetanide in reducing the severity of a focal attack late (2-4hr) after treatment administration; 4) To assess the safety of bumetanide in hypokalaemic periodic paralysis; ;Primary end point(s): The primary outcome measure is focal attack severity one hour after treatment administration. Treatment will be administered when the CMAP amplitude reaches 60% of its peak value.;Timepoint(s) of evaluation of this end point: This will be measured as CMAP amplitude expressed as a percent of peak CMAP during or after the McManis exercise 1 hour after treatment administration.

—

Secondary

Measure	Time frame
Timepoint(s) of evaluation of this end point: o The effect of treatment on focal attack duration. This will be measured as the time between treatment administration until CMAP returns to 65% peak CMAP. o The initial effect of treatment on severity of a focal attack within the first two hours. This will be measured as CMAP amplitude (in percent of peak) area under the curve (AUC) from treatment administration until two hours post-treatment. o The late effect of treatment on severity of a focal attack two to four hours post treatment. This will be measured as CMAP amplitude (in percent of peak) AUC from two hours until four hours post-treatment. o Safety of bumetanide to include self-reported side effects and changes of serum potassium. ; Secondary end point(s): 1)To assess the efficacy of bumetanide in reducing the duration of a localised attack; 2)To assess the efficacy of bumetanide in reducing the severity of a focal attack early (0-2hr) after treatment administration; 3)To assess the efficacy of bumetanide in reducing the severity of a focal attack late (2-4hr) after treatment administration; 4)To assess the safety of bumetanide in hypokalaemic periodic paralysis;	—

Measure

Time frame

Timepoint(s) of evaluation of this end point: o The effect of treatment on focal attack duration. This will be measured as the time between treatment administration until CMAP returns to 65% peak CMAP. o The initial effect of treatment on severity of a focal attack within the first two hours. This will be measured as CMAP amplitude (in percent of peak) area under the curve (AUC) from treatment administration until two hours post-treatment. o The late effect of treatment on severity of a focal attack two to four hours post treatment. This will be measured as CMAP amplitude (in percent of peak) AUC from two hours until four hours post-treatment. o Safety of bumetanide to include self-reported side effects and changes of serum potassium. ; Secondary end point(s): 1)To assess the efficacy of bumetanide in reducing the duration of a localised attack; 2)To assess the efficacy of bumetanide in reducing the severity of a focal attack early (0-2hr) after treatment administration; 3)To assess the efficacy of bumetanide in reducing the severity of a focal attack late (2-4hr) after treatment administration; 4)To assess the safety of bumetanide in hypokalaemic periodic paralysis;

—

Countries

United Kingdom

Contacts

Public ContactSponsor Regulatory Advisor

University College London - Joint Research Office

a.akinyemi@ucl.ac.uk02076796469

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026