Dichlorphenamide vs. Placebo for Periodic Paralysis
Acronym
HYP-HOP
Organization
University of RochesterOTHER
Status Module
Record Verification Date
May 2017
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Jun 2007
Primary Completion Date
Apr 2013Actual
Completion Date
May 2013Actual
First Submitted Date
Jun 27, 2007
First Submission Date that Met QC Criteria
Jun 28, 2007
First Posted Date
Jun 29, 2007Estimated
Results Waived
Not provided
Results First Submitted Date
Apr 30, 2014
Results First Submitted that Met QC Criteria
Apr 30, 2014
Results First Posted Date
May 30, 2014Estimated
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
May 15, 2017
Last Update Posted Date
Jun 14, 2017Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Robert Griggs, MD, Principal Investigator, University of RochesterPrincipal Investigator
Lead Sponsor
University of RochesterOTHER
Collaborators
Name
Class
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Not provided
Is FDA Regulated Device
Not provided
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
The purpose of this study is to compare Dichlorphenamide with placebo (an inactive substance) for prevention of episodes and for improvement of strength in hyperkalemic (HYP) and hypokalemic (HOP) periodic paralysis. This study will also look at the long-term effects of Dichlorphenamide in periodic paralysis.
Detailed Description
Periodic paralysis is a relatively rare, life-long disorder characterized by intermittent bouts of paralysis, progressive weakness, and diminished quality of life. Two drugs, acetazolamide (ACZ) and dichlorphenamide, have been prescribed to treat the disorder, however, dichlorphenamide is no longer available.
In this multi-center, parallel, randomized trial researchers will compare the effects of dichlorphenamide vs. placebo in patients with hyperkalemic (HYP) and hypokalemic (HOP) periodic paralysis.
The trial consists of two 9-week studies-one study will enroll persons with hyperkalemic periodic paralysis and the other study will enroll persons with hypokalemic periodic paralysis. Participants will be randomly assigned to one of two treatment groups: dichlorphenamide or placebo (an inactive substance). During the studies, participants will be asked to keep a daily diary to record the time, length, and severity of each episode of weakness (attack). The study coordinator will contact participants weekly to review the diary information.
The 9-week phase will be followed by a 1-year open-label dichlorphenamide extension without placebo to determine the long-term effects of dichlorphenamide on the course of the disease and on inter-attack weakness.
Duration of the trial for participants is approximately 65 weeks, including a screening phase to determine eligibility, the first 9-week treatment phase, and the one-year open-label extension phase.
Conditions Module
Conditions
Hyperkalemic Periodic Paralysis
Hypokalemic Periodic Paralysis
Keywords
periodic paralysis
dichlorphenamide
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 3
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
71Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
HYP Dichlorphenamide
Active Comparator
Hyperkalemic participants were randomized to Dichlorphenamide for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.
Drug: Dichlorphenamide (double-blind)
Drug: Dichlorphenamide (open-label)
HYP Placebo
Placebo Comparator
Hyperkalemic participants were randomized to Placebo for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.
Drug: Placebo (double-blind)
Drug: Dichlorphenamide (open-label)
HOP Dichlorphenamide
Active Comparator
Hypokalemic participants were randomized to Dichlorphenamide for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.
Drug: Dichlorphenamide (double-blind)
Drug: Dichlorphenamide (open-label)
HOP Placebo
Placebo Comparator
Hypokalemic participants were randomized to Placebo for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.
Drug: Placebo (double-blind)
Drug: Dichlorphenamide (open-label)
Interventions
Name
Type
Description
Arm Group Labels
Other Names
Dichlorphenamide (double-blind)
Drug
50mg tablet; maximum dosage 400mg/day
HOP Dichlorphenamide
HYP Dichlorphenamide
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
HYP Attack Rate
The number of distinct attacks per week over the final 8 weeks (Weeks 2-9) of the double-blind treatment period as self-reported by HYP participants.
8 weeks
HOP Attack Rate
The number of distinct attacks per week over the final 8 weeks (Weeks 2-9) of the double-blind treatment period as self-reported by HOP participants.
8 weeks
Secondary Outcomes
Measure
Description
Time Frame
HYP Severity-weighted Attack Rate
HYP participant severity-weighted attack rate is defined as the sum of average attack severity across all distinct attacks over the final 8 weeks (Weeks 2-9) of the double-blind treatment period divided by the number of weeks that the subject was followed. Attack severity (scored as 1-10 with increasing severity) is self-reported.
8 weeks
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Inclusion Criteria:
Genetically definite, clinically definite or clinically probable Hyperkalemic or Hypokalemic Periodic Paralysis as outlined in the protocol
Male and female participants, age 18 and older who are able to comply with the study conditions.
Participants who have distinct regular episodes of weakness with an average frequency of > or = to 1 a week and < or = to 3 a day either on or off treatment, whichever is higher
Normal thyroid-stimulating hormone (TSH) level
Exclusion Criteria:
Evidence for Andersen-Tawil syndrome (any one of the following 3 criteria)
Prolonged QT interval or complex ventricular ectopy between attacks
Distinctive physical features (2 of the following 5)
Low set ears
Short stature
Hypo-/micrognathia
Clinodactyly
Hypo-/hypertelorism
KIR 2.1 gene mutation
Coincidental renal, hepatic, active thyroid disease, restrictive or obstructive lung disease, other neuromuscular disease, or heart disease
Chronic, non-congestive, angle-closure glaucoma
Use of any of the following medications for reasons other than treatment of periodic paralysis: diuretics, antiarrhythmics, corticosteroids, beta-blockers, calcium channel blockers, antiepileptics, magnesium
History of life-threatening episodes of respiratory muscle weakness or cardiac arrhythmias during attacks
Pregnancy
Known mutation in the alpha subunit of the sodium channel gene in hypokalemic periodic paralysis patients
Accepts Healthy Volunteers
No
Sex
All
Sex/Gender Based
Not provided
Sex/Gender Description
Not provided
Minimum Age
18 Years
Maximum Age
Not provided
Standard Ages
AdultOlder Adult
Study Population
Not provided
Sampling Method
Not provided
Contacts/Locations Module
Central Contacts
Not provided
Overall Officials
Name
Affiliation
Role
Robert C. Griggs, M.D.
University of Rochester
Principal Investigator
Rabi Tawil, M.D.
Co-Principal Investigator, University of Rochester
Principal Investigator
Michael McDermott, Ph.D.
Biostatistician, University of Rochester
Locations
Facility
Status
City
State
ZIP
Country
Contacts
UCLA Neurology
Los Angeles
California
90095
United States
University of California-San Francisco
References Module
No data available
No data is available for this block.
IPD Sharing Statement Module
No data available
No data is available for this block.
Results Section
Participant Flow Module
Pre-assignment Details
The original trial design included an Acetazolamide (ACZ) drug arm which was subsequently removed. Five participants randomized to ACZ and one ineligible participant are not included in the trial results reported here.
Recruitment Details
Not provided
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
HYP Dichlorphenamide
Hyperkalemic participants were randomized to Dichlorphenamide for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.
Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day
Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day
Periods
Title
Milestones
Reasons Not Completed
Double-Blind Phase
Type
Comment
Milestone Data
STARTED
Baseline Characteristics Module
Baseline Analysis Population Description
Not provided
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
5
More Info Module
Limitations and Caveats
Annotation Section
No data available
No data is available for this block.
Document Section
No data available
No data is available for this block.
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Canada
France
Submission Tracking
No data available
No data is available for this block.
Condition Browse Module
MeSH Terms
Intervention Browse Module
MeSH Terms
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Not provided
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
Double
Masking Description
Not provided
Who Masked
ParticipantInvestigator
Daranide
Placebo (double-blind)
Drug
Inactive substance manufactured to look like Dichlorphenamide 50mg tablet
HOP Placebo
HYP Placebo
Dichlorphenamide (open-label)
Drug
50mg tablet; maximum dosage 400mg/day
HOP Dichlorphenamide
HOP Placebo
HYP Dichlorphenamide
HYP Placebo
Daranide
HOP Severity-weighted Attack Rate
HOP participant severity-weighted attack rate is defined as the sum of average attack severity across all distinct attacks over the final 8 weeks (Weeks 2-9) of the double-blind treatment period divided by the number of weeks that the subject was followed. Attack severity (scored as 1-10 with increasing severity) is self-reported.
8 weeks
HYP Attack Duration
HYP participant total attack duration per week, defined as the sum of attack durations across all distinct attacks over the final 8 weeks (Weeks 2-9) of the double-blind treatment period divided by the number of weeks that the subject was followed.
8 weeks
HOP Attack Duration
HOP participant total attack duration per week, defined as the sum of attack durations across all distinct attacks over the final 8 weeks (Weeks 2-9) of the double-blind treatment period divided by the number of weeks that the subject was followed.
8 weeks
HYP Endpoint of Acute Worsening
Increase in attack frequency or severity in HYP participants necessitating withdrawal from the initial nine-week double-blind treatment period and moving directly into the open-label phase.
0-9 weeks
HOP Endpoint of Acute Worsening
Increase in attack frequency or severity in HOP participants necessitating withdrawal from the initial nine-week double-blind treatment period and moving directly into the open-label phase.
0-9 weeks
HYP Change From Baseline to Week 9 in Average Manual Muscle Testing (MMT) Score
The strength of each of 26 individual muscles was graded using a modified 13-point Medical Research Council scale ranging from 0-5. Recorded grades were converted to numerical values as follows prior to averaging across muscles to form a composite score: 0 = 0; 1 = 1; 2- = 1.67; 2 = 2; 2+ = 2.33; 3- = 2.67; 3 = 3; 3+ = 3.33; 4- = 3.67; 4 = 4; 4+ = 4.33; 5- = 4.67; 5 = 5. A higher score represents a better outcome, i.e. 5 = normal strength.
The following muscles were tested: shoulder abductor (left/right), elbow extensor (left/right), elbow flexor (left/right), wrist extensor (left/right), wrist flexor (left/right), hip flexor (left/right), hip extensor (left/right), hip abductor (left/right), knee extensor (left/right), knee flexor (left/right), ankle dorsiflexor (left/right), ankle plantar flexor (left/right), neck extensor, neck flexor.
Baseline and 9 weeks
HOP Change From Baseline to Week 9 in Average Manual Muscle Testing (MMT) Score
The strength of each of 26 individual muscles was graded using a modified 13-point Medical Research Council scale ranging from 0-5. Recorded grades were converted to numerical values as follows prior to averaging across muscles to form a composite score: 0 = 0; 1 = 1; 2- = 1.67; 2 = 2; 2+ = 2.33; 3- = 2.67; 3 = 3; 3+ = 3.33; 4- = 3.67; 4 = 4; 4+ = 4.33; 5- = 4.67; 5 = 5. A higher score represents a better outcome, i.e. 5 = normal strength.
The following muscles were tested: shoulder abductor (left/right), elbow extensor (left/right), elbow flexor (left/right), wrist extensor (left/right), wrist flexor (left/right), hip flexor (left/right), hip extensor (left/right), hip abductor (left/right), knee extensor (left/right), knee flexor (left/right), ankle dorsiflexor (left/right), ankle plantar flexor (left/right), neck extensor, neck flexor.
Baseline and 9 weeks
HYP Change From Baseline to Week 9 in Average Maximum Voluntary Isometric Contraction Testing (MVICT) Scores
The strength of each of 10 muscles was measured using quantitative myometry and expressed as the number of standard deviations from normal (Z-score) given the participant's age, gender, and height. The scores were averaged across muscles to form a composite MVICT score: average standardized MVICT score. A positive Z-score indicates a better outcome.
The following muscles were tested: elbow extensor (left/right), elbow flexor (left/right), knee extensor (left/right), knee flexor (left/right), and hand grip (left/right).
Baseline and 9 weeks
HOP Change From Baseline to Week 9 in Average Maximum Voluntary Isometric Contraction Testing (MVICT) Scores
The strength of each of 10 muscles was measured using quantitative myometry and expressed as the number of standard deviations from normal (Z-score) given the participant's age, gender, and height. The scores were averaged across muscles to form a composite MVICT score: average standardized MVICT score. A positive Z-score indicates a better outcome.
The following muscles were tested: elbow extensor (left/right), elbow flexor (left/right), knee extensor (left/right), knee flexor (left/right), and hand grip (left/right).
Baseline and 9 weeks
HYP Change From Baseline to Week 9 in Average Maximum Voluntary Isometric Contraction Testing Percent of Predicted Normal
The strength of each of 10 muscles was measured using quantitative myometry and expressed as the percent of predicted normal given the participant's age, gender, and height. The scores were averaged across muscles to form a composite MVICT score: average percent of predicted normal score. A higher number indicates a better outcome.
The following muscles were tested: elbow extensor (left/right), elbow flexor (left/right), knee extensor (left/right), knee flexor (left/right), and hand grip (left/right).
Baseline and 9 weeks
HOP Change From Baseline to Week 9 in Average Maximum Voluntary Isometric Contraction Testing Percent of Predicted Normal
The strength of each of 10 muscles was measured using quantitative myometry and expressed as the percent of predicted normal given the participant's age, gender, and height. The scores were averaged across muscles to form a composite MVICT score: average percent of predicted normal score. A higher number indicates a better outcome.
The following muscles were tested: elbow extensor (left/right), elbow flexor (left/right), knee extensor (left/right), knee flexor (left/right), and hand grip (left/right).
Baseline and 9 weeks
HYP Change From Baseline to Week 9 in Lean Body Mass
Lean body mass was measured by dual-energy X-ray absorptiometry (DEXA).
Baseline and 9 weeks
HOP Change From Baseline to Week 9 in Lean Body Mass
Lean body mass was measured by dual-energy X-ray absorptiometry (DEXA).
Baseline and 9 weeks
HYP Change From Baseline to Week 9 in SF-36 Physical Component Summary Score
The Medical Outcomes Study Short Form (SF-36) questionnaire is a widely used profile measure of generic health-related quality of life. The 36 questions are allocated to eight scales: physical function (PF), physical role (RP), bodily pain (BP), general health perceptions (GH), vitality (VT), social function (SF), mental health (MH), and emotional role (RE). The physical component summary score is calculated from the four scales of PF, RP,BP, and GH. Scores are constructed as a T-score with a mean of 50 and standard deviation of 10 and no minimum or maximum score. Higher scores are associated with better quality of life.
Baseline and 9 weeks
HOP Change From Baseline to Week 9 in SF-36 Physical Component Summary Score
The Medical Outcomes Study Short Form (SF-36) questionnaire is a widely used profile measure of generic health-related quality of life. The 36 questions are allocated to eight scales: physical function (PF), physical role (RP), bodily pain (BP), general health perceptions (GH), vitality (VT), social function (SF), mental health (MH), and emotional role (RE). The physical component summary score is calculated from the four scales of PF, RP, BP, and GH. Scores are constructed as a T-score with a mean of 50 and standard deviation of 10 and no minimum or maximum score. Higher scores are associated with better quality of life.
Baseline and 9 weeks
HYP Change From Baseline to Week 9 in SF-36 Mental Health Component Summary Score
The Medical Outcomes Study Short Form (SF-36) questionnaire is a widely used profile measure of generic health-related quality of life. The 36 questions are allocated to eight scales: physical function (PF), physical role (RP), bodily pain (BP), general health perceptions (GH), vitality (VT), social function (SF), mental health (MH), and emotional role (RE). The mental health component summary score is calculated from the four scales of VT, SF, MH, and RE. Scores are constructed as a T-score with a mean of 50 and standard deviation of 10 and no minimum or maximum score. Higher scores are associated with better quality of life.
Baseline and 9 weeks
HOP Change From Baseline to Week 9 in SF-36 Mental Health Component Summary Score
The Medical Outcomes Study Short Form (SF-36) questionnaire is a widely used profile measure of generic health-related quality of life. The 36 questions are allocated to eight scales: physical function (PF), physical role (RP), bodily pain (BP), general health perceptions (GH), vitality (VT), social function (SF), mental health (MH), and emotional role (RE). The mental health component summary score is calculated from the four scales of VT, SF, MH, and RE. Scores are constructed as a T-score with a mean of 50 and standard deviation of 10 and no minimum or maximum score. Higher scores are associated with better quality of life.
Baseline and 9 weeks
San Francisco
California
94143
United States
University of Kansas Medical Center
Kansas City
Kansas
66160
United States
Brigham & Women's Hospital
Boston
Massachusetts
02115
United States
Mayo Clinic
Rochester
Minnesota
55905
United States
Washington University School of Medicine
St Louis
Missouri
63110
United States
Columbia University Medical Center
New York
New York
10032
United States
University of Rochester
Rochester
New York
14642
United States
Ohio State University
Columbus
Ohio
43210
United States
University of Texas Southwestern-Dallas
Dallas
Texas
75390
United States
University of Milan
San Donato
Milan
Italy
Institute of Neurology-Queen's Square
London
United Kingdom
FG001
HYP Placebo
Hyperkalemic participants were randomized to Placebo for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.
Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet
Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day
FG002
HOP Dichlorphenamide
Hypokalemic participants were randomized to Dichlorphenamide for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.
Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day
Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day
FG003
HOP Placebo
Hypokalemic participants were randomized to Placebo for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.
Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet
Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day
FG00012 subjects
FG0019 subjects
FG00224 subjects
FG00320 subjects
Reached Endpoint of Acute Worsening
FG0000 subjects
FG0012 subjects
FG0020 subjects
FG0035 subjects
COMPLETED
FG0009 subjects
FG0019 subjects
FG00222 subjects
FG00319 subjects
NOT COMPLETED
FG0003 subjects
FG0010 subjects
FG0022 subjects
FG0031 subjects
Type
Comment
Reasons
Adverse Event
FG0002 subjects
FG0010 subjects
FG0021 subjects
FG0030 subjects
Withdrawal by Subject
FG0001 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
Subject Non-compliance
FG0000 subjects
FG0010 subjects
FG0021 subjects
FG0030 subjects
Negative DNA test
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0031 subjects
Open-Label Phase
Type
Comment
Milestone Data
STARTED
FG0009 subjects
FG0018 subjects1 subject randomized to Acetazolamide in this phase is not included-drug arm was removed from trial.
FG00222 subjects
FG00319 subjects
COMPLETED
FG0009 subjects
FG0017 subjects
FG00217 subjects
FG00314 subjects
NOT COMPLETED
FG0000 subjects
FG0011 subjects
FG0025 subjects
FG0035 subjects
Type
Comment
Reasons
Adverse Event
FG0000 subjects
FG0011 subjects
FG0025 subjects
FG003
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
HYP Dichlorphenamide
Hyperkalemic participants were randomized to Dichlorphenamide for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.
Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day
Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day
BG001
HYP Placebo
Hyperkalemic participants were randomized to Placebo for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.
Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet
Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day
BG002
HOP Dichlorphenamide
Hypokalemic participants were randomized to Dichlorphenamide for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.
Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day
Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day
BG003
HOP Placebo
Hypokalemic participants were randomized to Placebo for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.
Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet
Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day
BG004
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG00012
BG0019
BG00224
BG00320
BG00465
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Standard Deviation
years
Title
Denominators
Categories
Title
Measurements
BG00040.6± 10.3
BG00145.2± 17.7
BG00244.8± 14.6
BG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG0006
BG0016
BG002
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
HYP Attack Rate
The number of distinct attacks per week over the final 8 weeks (Weeks 2-9) of the double-blind treatment period as self-reported by HYP participants.
Posted
Median
Inter-Quartile Range
attacks per week
8 weeks
ID
Title
Description
OG000
HYP Dichlorphenamide
Hyperkalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day.
OG001
HYP Placebo
Hyperkalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet.
Units
Counts
Participants
OG00012
OG0019
Title
Denominators
Categories
Title
Measurements
OG0000.9(0.4 to 1.5)
OG0014.8(0.5 to 7.1)
Primary
HOP Attack Rate
The number of distinct attacks per week over the final 8 weeks (Weeks 2-9) of the double-blind treatment period as self-reported by HOP participants.
Posted
Median
Inter-Quartile Range
attacks per week
8 weeks
ID
Title
Description
OG000
HOP Dichlorphenamide
Hypokalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day.
OG001
HOP Placebo
Hypokalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet.
Units
Counts
Participants
OG000
Secondary
HYP Severity-weighted Attack Rate
HYP participant severity-weighted attack rate is defined as the sum of average attack severity across all distinct attacks over the final 8 weeks (Weeks 2-9) of the double-blind treatment period divided by the number of weeks that the subject was followed. Attack severity (scored as 1-10 with increasing severity) is self-reported.
Posted
Median
Inter-Quartile Range
severity-weighted attacks per week
8 weeks
ID
Title
Description
OG000
HYP Dichlorphenamide
Hyperkalemic participants were randomized to Dichlorphenamide for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.
Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day
Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day
OG001
HYP Placebo
Hyperkalemic participants were randomized to Placebo for a 9 week double-blind phase. All participants then received Dichlorphenamide for a 52 week open-label phase.
Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet
Dichlorphenamide (open-label): 50mg tablet; maximum dosage 400mg/day
Units
Counts
Secondary
HOP Severity-weighted Attack Rate
HOP participant severity-weighted attack rate is defined as the sum of average attack severity across all distinct attacks over the final 8 weeks (Weeks 2-9) of the double-blind treatment period divided by the number of weeks that the subject was followed. Attack severity (scored as 1-10 with increasing severity) is self-reported.
Posted
Median
Inter-Quartile Range
severity-weighted attacks per week
8 weeks
ID
Title
Description
OG000
HOP Dichlorphenamide
Hypokalemic participants were randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day.
OG001
HOP Placebo
Hypokalemic participants were randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet.
Units
Counts
Participants
OG000
Secondary
HYP Attack Duration
HYP participant total attack duration per week, defined as the sum of attack durations across all distinct attacks over the final 8 weeks (Weeks 2-9) of the double-blind treatment period divided by the number of weeks that the subject was followed.
Posted
Median
Inter-Quartile Range
hours per week
8 weeks
ID
Title
Description
OG000
HYP Dichlorphenamide
Hyperkalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day.
OG001
HYP Placebo
Hyperkalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet.
Units
Counts
Participants
OG000
Secondary
HOP Attack Duration
HOP participant total attack duration per week, defined as the sum of attack durations across all distinct attacks over the final 8 weeks (Weeks 2-9) of the double-blind treatment period divided by the number of weeks that the subject was followed.
Posted
Median
Inter-Quartile Range
hours per week
8 weeks
ID
Title
Description
OG000
HOP Dichlorphenamide
Hypokalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day.
OG001
HOP Placebo
Hypokalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet.
Units
Counts
Participants
OG000
Secondary
HYP Endpoint of Acute Worsening
Increase in attack frequency or severity in HYP participants necessitating withdrawal from the initial nine-week double-blind treatment period and moving directly into the open-label phase.
Posted
Number
participants
0-9 weeks
ID
Title
Description
OG000
HYP Dichlorphenamide
Hyperkalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day.
OG001
HYP Placebo
Hyperkalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet.
Units
Counts
Participants
OG000
Secondary
HOP Endpoint of Acute Worsening
Increase in attack frequency or severity in HOP participants necessitating withdrawal from the initial nine-week double-blind treatment period and moving directly into the open-label phase.
Posted
Number
participants
0-9 weeks
ID
Title
Description
OG000
HOP Dichlorphenamide
Hypokalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day.
OG001
HOP Placebo
Hypokalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet.
Units
Counts
Participants
OG000
Secondary
HYP Change From Baseline to Week 9 in Average Manual Muscle Testing (MMT) Score
The strength of each of 26 individual muscles was graded using a modified 13-point Medical Research Council scale ranging from 0-5. Recorded grades were converted to numerical values as follows prior to averaging across muscles to form a composite score: 0 = 0; 1 = 1; 2- = 1.67; 2 = 2; 2+ = 2.33; 3- = 2.67; 3 = 3; 3+ = 3.33; 4- = 3.67; 4 = 4; 4+ = 4.33; 5- = 4.67; 5 = 5. A higher score represents a better outcome, i.e. 5 = normal strength.
The following muscles were tested: shoulder abductor (left/right), elbow extensor (left/right), elbow flexor (left/right), wrist extensor (left/right), wrist flexor (left/right), hip flexor (left/right), hip extensor (left/right), hip abductor (left/right), knee extensor (left/right), knee flexor (left/right), ankle dorsiflexor (left/right), ankle plantar flexor (left/right), neck extensor, neck flexor.
Participants with evaluable data at both timepoints
Posted
Mean
Standard Deviation
units on a scale
Baseline and 9 weeks
ID
Title
Description
OG000
HYP Dichlorphenamide
Hyperkalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day
OG001
HYP Placebo
Hyperkalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet
Secondary
HOP Change From Baseline to Week 9 in Average Manual Muscle Testing (MMT) Score
The strength of each of 26 individual muscles was graded using a modified 13-point Medical Research Council scale ranging from 0-5. Recorded grades were converted to numerical values as follows prior to averaging across muscles to form a composite score: 0 = 0; 1 = 1; 2- = 1.67; 2 = 2; 2+ = 2.33; 3- = 2.67; 3 = 3; 3+ = 3.33; 4- = 3.67; 4 = 4; 4+ = 4.33; 5- = 4.67; 5 = 5. A higher score represents a better outcome, i.e. 5 = normal strength.
The following muscles were tested: shoulder abductor (left/right), elbow extensor (left/right), elbow flexor (left/right), wrist extensor (left/right), wrist flexor (left/right), hip flexor (left/right), hip extensor (left/right), hip abductor (left/right), knee extensor (left/right), knee flexor (left/right), ankle dorsiflexor (left/right), ankle plantar flexor (left/right), neck extensor, neck flexor.
Participants with evaluable data at both timepoints
Posted
Mean
Standard Deviation
units on a scale
Baseline and 9 weeks
ID
Title
Description
OG000
HOP Dichlorphenamide
Hypokalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day
OG001
HOP Placebo
Hypokalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet
Secondary
HYP Change From Baseline to Week 9 in Average Maximum Voluntary Isometric Contraction Testing (MVICT) Scores
The strength of each of 10 muscles was measured using quantitative myometry and expressed as the number of standard deviations from normal (Z-score) given the participant's age, gender, and height. The scores were averaged across muscles to form a composite MVICT score: average standardized MVICT score. A positive Z-score indicates a better outcome.
The following muscles were tested: elbow extensor (left/right), elbow flexor (left/right), knee extensor (left/right), knee flexor (left/right), and hand grip (left/right).
Participants with evaluable data at both timepoints
Posted
Mean
Standard Deviation
Z-score
Baseline and 9 weeks
ID
Title
Description
OG000
HYP Dichlorphenamide
Hyperkalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day
OG001
HYP Placebo
Hyperkalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet
Units
Counts
Secondary
HOP Change From Baseline to Week 9 in Average Maximum Voluntary Isometric Contraction Testing (MVICT) Scores
The strength of each of 10 muscles was measured using quantitative myometry and expressed as the number of standard deviations from normal (Z-score) given the participant's age, gender, and height. The scores were averaged across muscles to form a composite MVICT score: average standardized MVICT score. A positive Z-score indicates a better outcome.
The following muscles were tested: elbow extensor (left/right), elbow flexor (left/right), knee extensor (left/right), knee flexor (left/right), and hand grip (left/right).
Participants with evaluable data at both timepoints
Posted
Mean
Standard Deviation
Z-score
Baseline and 9 weeks
ID
Title
Description
OG000
HOP Dichlorphenamide
Hypokalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day.
OG001
HOP Placebo
Hypokalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet.
Units
Counts
Secondary
HYP Change From Baseline to Week 9 in Average Maximum Voluntary Isometric Contraction Testing Percent of Predicted Normal
The strength of each of 10 muscles was measured using quantitative myometry and expressed as the percent of predicted normal given the participant's age, gender, and height. The scores were averaged across muscles to form a composite MVICT score: average percent of predicted normal score. A higher number indicates a better outcome.
The following muscles were tested: elbow extensor (left/right), elbow flexor (left/right), knee extensor (left/right), knee flexor (left/right), and hand grip (left/right).
Participants with evaluable data at both timepoints
Posted
Mean
Standard Deviation
average percent of predicted normal
Baseline and 9 weeks
ID
Title
Description
OG000
HYP Dichlorphenamide
Hyperkalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day
OG001
HYP Placebo
Hyperkalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet
Units
Secondary
HOP Change From Baseline to Week 9 in Average Maximum Voluntary Isometric Contraction Testing Percent of Predicted Normal
The strength of each of 10 muscles was measured using quantitative myometry and expressed as the percent of predicted normal given the participant's age, gender, and height. The scores were averaged across muscles to form a composite MVICT score: average percent of predicted normal score. A higher number indicates a better outcome.
The following muscles were tested: elbow extensor (left/right), elbow flexor (left/right), knee extensor (left/right), knee flexor (left/right), and hand grip (left/right).
Participants with evaluable data at both timepoints
Posted
Mean
Standard Deviation
average percent of predicted normal
Baseline and 9 weeks
ID
Title
Description
OG000
HOP Dichlorphenamide
Hypokalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day
OG001
HOP Placebo
Hypokalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet
Units
Secondary
HYP Change From Baseline to Week 9 in Lean Body Mass
Lean body mass was measured by dual-energy X-ray absorptiometry (DEXA).
Participants who had lean body mass data available at baseline and week 9.
Posted
Mean
Standard Deviation
kg
Baseline and 9 weeks
ID
Title
Description
OG000
HYP Dichlorphenamide
Hyperkalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day.
OG001
HYP Placebo
Hyperkalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet.
Units
Counts
Participants
OG000
Secondary
HOP Change From Baseline to Week 9 in Lean Body Mass
Lean body mass was measured by dual-energy X-ray absorptiometry (DEXA).
Participants who had lean body mass data available at baseline and week 9.
Posted
Mean
Standard Deviation
kg
Baseline and 9 weeks
ID
Title
Description
OG000
HOP Dichlorphenamide
Hypokalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day.
OG001
HOP Placebo
Hypokalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet.
Units
Counts
Participants
OG000
Secondary
HYP Change From Baseline to Week 9 in SF-36 Physical Component Summary Score
The Medical Outcomes Study Short Form (SF-36) questionnaire is a widely used profile measure of generic health-related quality of life. The 36 questions are allocated to eight scales: physical function (PF), physical role (RP), bodily pain (BP), general health perceptions (GH), vitality (VT), social function (SF), mental health (MH), and emotional role (RE). The physical component summary score is calculated from the four scales of PF, RP,BP, and GH. Scores are constructed as a T-score with a mean of 50 and standard deviation of 10 and no minimum or maximum score. Higher scores are associated with better quality of life.
Participants with evaluable data at both timepoints
Posted
Mean
Standard Deviation
T-score
Baseline and 9 weeks
ID
Title
Description
OG000
HYP Dichlorphenamide
Hyperkalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day.
OG001
HYP Placebo
Hyperkalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet.
Secondary
HOP Change From Baseline to Week 9 in SF-36 Physical Component Summary Score
The Medical Outcomes Study Short Form (SF-36) questionnaire is a widely used profile measure of generic health-related quality of life. The 36 questions are allocated to eight scales: physical function (PF), physical role (RP), bodily pain (BP), general health perceptions (GH), vitality (VT), social function (SF), mental health (MH), and emotional role (RE). The physical component summary score is calculated from the four scales of PF, RP, BP, and GH. Scores are constructed as a T-score with a mean of 50 and standard deviation of 10 and no minimum or maximum score. Higher scores are associated with better quality of life.
Participants with evaluable data at both timepoints
Posted
Mean
Standard Deviation
T-score
Baseline and 9 weeks
ID
Title
Description
OG000
HOP Dichlorphenamide
Hypokalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day.
OG001
HOP Placebo
Hypokalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet.
Secondary
HYP Change From Baseline to Week 9 in SF-36 Mental Health Component Summary Score
The Medical Outcomes Study Short Form (SF-36) questionnaire is a widely used profile measure of generic health-related quality of life. The 36 questions are allocated to eight scales: physical function (PF), physical role (RP), bodily pain (BP), general health perceptions (GH), vitality (VT), social function (SF), mental health (MH), and emotional role (RE). The mental health component summary score is calculated from the four scales of VT, SF, MH, and RE. Scores are constructed as a T-score with a mean of 50 and standard deviation of 10 and no minimum or maximum score. Higher scores are associated with better quality of life.
Participants with evaluable data at both timepoints
Posted
Mean
Standard Deviation
T-score
Baseline and 9 weeks
ID
Title
Description
OG000
HYP Dichlorphenamide
Hyperkalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day.
OG001
HYP Placebo
Hyperkalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet.
Secondary
HOP Change From Baseline to Week 9 in SF-36 Mental Health Component Summary Score
The Medical Outcomes Study Short Form (SF-36) questionnaire is a widely used profile measure of generic health-related quality of life. The 36 questions are allocated to eight scales: physical function (PF), physical role (RP), bodily pain (BP), general health perceptions (GH), vitality (VT), social function (SF), mental health (MH), and emotional role (RE). The mental health component summary score is calculated from the four scales of VT, SF, MH, and RE. Scores are constructed as a T-score with a mean of 50 and standard deviation of 10 and no minimum or maximum score. Higher scores are associated with better quality of life.
Participants with evaluable data at both timepoints
Posted
Mean
Standard Deviation
T-score
Baseline and 9 weeks
ID
Title
Description
OG000
HOP Dichlorphenamide
Hypokalemic participants randomized to Dichlorphenamide for a 9 week double-blind phase. Dichlorphenamide (double-blind): 50mg tablet; maximum dosage 400mg/day.
OG001
HOP Placebo
Hypokalemic participants randomized to Placebo for a 9 week double-blind phase. Placebo (double-blind): Inactive substance manufactured to look like Dichlorphenamide 50mg tablet.
Time Frame
Not provided
Description
Adverse events were assessed via weekly phone calls with participants and at all study visits.
One HOP participant inadvertently remained on placebo during the open-label phase and therefore is not reported in the Other Adverse Events for the HOP Open-Label Dichlorphenamide group.
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
HYP Double-Blind Dichlorphenamide
Hyperkalemic participants were randomized to Dichlorphenamide for the 9 week double-blind phase.
1
12
9
12
EG001
HYP Double-Blind Placebo
Hyperkalemic participants were randomized to Placebo for the 9 week double-blind phase.
0
9
3
9
EG002
HYP Open-Label Dichlorphenamide
Hyperkalemic participants received Dichlorphenamide for the 52 week open-label phase.
0
17
12
17
EG003
HOP Double-Blind Dichlorphenamide
Hypokalemic participants were randomized to Dichlorphenamide for the 9 week double-blind phase.
0
24
20
24
EG004
HOP Double-Blind Placebo
Hypokalemic participants were randomized to Placebo for the 9 week double-blind phase.
1
20
11
20
EG005
HOP Open-Label Dichlorphenamide
Hypokalemic participants received Dichlorphenamide for the 52 week open-label phase.
3
40
32
40
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Humerus fracture
Injury, poisoning and procedural complications
MedDRA
Systematic Assessment
EG0000 events0 affected12 at risk
EG0010 events0 affected9 at risk
EG0020 events0 affected17 at risk
EG0030 events0 affected24 at risk
EG004
Road traffic accident
Injury, poisoning and procedural complications
MedDRA
Systematic Assessment
Occurred in the pre-treatment screening period.
EG0001 events1 affected12 at risk
EG0010 events0 affected9 at risk
EG0020 events0 affected17 at risk
EG003
Thyroid cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA
Systematic Assessment
EG0000 events0 affected12 at risk
EG0010 events0 affected9 at risk
EG0020 events0 affected17 at risk
EG003
Adenocarcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA
Systematic Assessment
EG0000 events0 affected12 at risk
EG0010 events0 affected9 at risk
EG0020 events0 affected17 at risk
EG003
Cauda equina syndrome
Nervous system disorders
MedDRA
Systematic Assessment
EG0000 events0 affected12 at risk
EG0010 events0 affected9 at risk
EG0020 events0 affected17 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA
Systematic Assessment
EG0001 events1 affected12 at risk
EG0010 events0 affected9 at risk
EG0020 events0 affected17 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
EAR PAIN
Ear and labyrinth disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG0030 affected24 at risk
EG0040 affected20 at risk
EG0050 affected40 at risk
TINNITUS
Ear and labyrinth disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
EYE PAIN
Eye disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
LACRIMATION INCREASED
Eye disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
PHOTOPSIA
Eye disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
VISION BLURRED
Eye disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
VISUAL DISTURBANCE
Eye disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
ABDOMINAL PAIN UPPER
Gastrointestinal disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
CONSTIPATION
Gastrointestinal disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
DIARRHOEA
Gastrointestinal disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
DRY MOUTH
Gastrointestinal disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
GASTROOESOPHAGEAL REFLUX DISEASE
Gastrointestinal disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
NAUSEA
Gastrointestinal disorders
MedDRA (11.0)
Systematic Assessment
EG0002 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
VOMITING
Gastrointestinal disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
ASTHENIA
General disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0011 affected9 at risk
EG0021 affected17 at risk
EG003
FATIGUE
General disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
MALAISE
General disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
OEDEMA
General disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
OEDEMA PERIPHERAL
General disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
PAIN
General disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
PYREXIA
General disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
CORNEAL GRAFT REJECTION
Immune system disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
CELLULITIS
Infections and infestations
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
CYSTITIS ESCHERICHIA
Infections and infestations
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
INFECTION
Infections and infestations
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
INFLUENZA
Infections and infestations
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
NASOPHARYNGITIS
Infections and infestations
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
PHARYNGITIS STREPTOCOCCAL
Infections and infestations
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
TONSILLITIS
Infections and infestations
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
CONCUSSION
Injury, poisoning and procedural complications
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
FALL
Injury, poisoning and procedural complications
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
HEAD INJURY
Injury, poisoning and procedural complications
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
MUSCLE STRAIN
Injury, poisoning and procedural complications
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
RIB FRACTURE
Injury, poisoning and procedural complications
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
SPINAL FRACTURE
Injury, poisoning and procedural complications
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
BLOOD CHOLESTEROL INCREASED
Investigations
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
BLOOD GLUCOSE INCREASED
Investigations
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
WEIGHT DECREASED
Investigations
MedDRA (11.0)
Systematic Assessment
EG0002 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
ANOREXIA
Metabolism and nutrition disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
ARTHRALGIA
Musculoskeletal and connective tissue disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
MUSCLE SPASMS
Musculoskeletal and connective tissue disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
MUSCLE TWITCHING
Musculoskeletal and connective tissue disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
MUSCULAR WEAKNESS
Musculoskeletal and connective tissue disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
MUSCULOSKELETAL DISCOMFORT
Musculoskeletal and connective tissue disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
MUSCULOSKELETAL STIFFNESS
Musculoskeletal and connective tissue disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
MYALGIA
Musculoskeletal and connective tissue disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
PAIN IN EXTREMITY
Musculoskeletal and connective tissue disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
COGNITIVE DISORDER
Nervous system disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
DIZZINESS
Nervous system disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
DYSARTHRIA
Nervous system disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
DYSGEUSIA
Nervous system disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
HEADACHE
Nervous system disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0011 affected9 at risk
EG0021 affected17 at risk
EG003
HYPOAESTHESIA
Nervous system disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
LETHARGY
Nervous system disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
MEMORY IMPAIRMENT
Nervous system disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0011 affected9 at risk
EG0022 affected17 at risk
EG003
PARAESTHESIA
Nervous system disorders
MedDRA (11.0)
Systematic Assessment
EG0008 affected12 at risk
EG0013 affected9 at risk
EG0029 affected17 at risk
EG003
POLYNEUROPATHY
Nervous system disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
CONFUSIONAL STATE
Psychiatric disorders
MedDRA (11.0)
Systematic Assessment
EG0002 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
DEPRESSED MOOD
Psychiatric disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
DEPRESSION
Psychiatric disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
NERVOUSNESS
Psychiatric disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
NIGHTMARE
Psychiatric disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
NEPHROLITHIASIS
Renal and urinary disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
DYSPNOEA
Respiratory, thoracic and mediastinal disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
PHARYNGOLARYNGEAL PAIN
Respiratory, thoracic and mediastinal disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
ACNE
Skin and subcutaneous tissue disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
DRY SKIN
Skin and subcutaneous tissue disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
OILY SKIN
Skin and subcutaneous tissue disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
PHOTOSENSITIVITY REACTION
Skin and subcutaneous tissue disorders
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0020 affected17 at risk
EG003
PRURITUS
Skin and subcutaneous tissue disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
RASH
Skin and subcutaneous tissue disorders
MedDRA (11.0)
Systematic Assessment
EG0001 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
FOOT OPERATION
Surgical and medical procedures
MedDRA (11.0)
Systematic Assessment
EG0000 affected12 at risk
EG0010 affected9 at risk
EG0021 affected17 at risk
EG003
Sample sizes in the trial were limited by slow recruitment and the trial was concluded before attainment of the target numbers of subjects. Statistical analyses were conducted using smaller group sizes than planned, particularly for HYP subjects.
Certain Agreements
Are all PI(s) employees of the sponsor?
Yes
Restriction Type
Not provided
Results Disclosure Restriction on PI(s)?
Not provided
Other Details
Not provided
Point of Contact
Title
Organization
Phone
Extension
Email
Robert Griggs, MD
University of Rochester
585-275-3707
robert_griggs@urmc.rochester.edu
ID
Term
D020513
Paralysis, Hyperkalemic Periodic
D020514
Hypokalemic Periodic Paralysis
Ancestor Terms
ID
Term
D010245
Paralyses, Familial Periodic
D009135
Muscular Diseases
D009140
Musculoskeletal Diseases
D009468
Neuromuscular Diseases
D009422
Nervous System Diseases
D008664
Metal Metabolism, Inborn Errors
D008661
Metabolism, Inborn Errors
D030342
Genetic Diseases, Inborn
D009358
Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D008659
Metabolic Diseases
D009750
Nutritional and Metabolic Diseases
Browse Leaves
Not provided
Browse Branches
Not provided
ID
Term
D004005
Dichlorphenamide
D004311
Double-Blind Method
Ancestor Terms
ID
Term
D013449
Sulfonamides
D000577
Amides
D009930
Organic Chemicals
D013450
Sulfones
D013457
Sulfur Compounds
D015340
Epidemiologic Research Design
D004812
Epidemiologic Methods
D008919
Investigative Techniques
D012107
Research Design
D008722
Methods
D017531
Health Care Evaluation Mechanisms
D011787
Quality of Health Care
D017530
Health Care Quality, Access, and Evaluation
D011634
Public Health
D004778
Environment and Public Health
Browse Leaves
Not provided
Browse Branches
Not provided
3 subjects
Worsening Disease
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0032 subjects
44.0
± 15.6
BG00444.2± 14.3
8
BG0034
BG00424
Male
BG0006
BG0013
BG00216
BG00316
BG00441
24
OG00120
Title
Denominators
Categories
Title
Measurements
OG0000.3(0.1 to 1.6)
OG0012.4(0.5 to NA)The upper quartile for the placebo group could not be calculated because more than 25% of placebo-treated subjects reached the endpoint of acute worsening.
Participants
OG00012
OG0019
Title
Denominators
Categories
Title
Measurements
OG0001.0(0.4 to 2.9)
OG0015.8(1.4 to 28.0)
24
OG00120
Title
Denominators
Categories
Title
Measurements
OG0000.6(0.2 to 3.9)
OG0015.7(1.2 to NA)The upper quartile for the placebo group could not be calculated because more than 25% of placebo-treated subjects reached the endpoint of acute worsening and thus were assigned an arbitrarily large attack duration for purposes of analysis.
12
OG0019
Title
Denominators
Categories
Title
Measurements
OG00010.5(2.5 to 21.3)
OG00139.4(6.2 to 139.4)
24
OG00120
Title
Denominators
Categories
Title
Measurements
OG0002.7(0.4 to 13.4)
OG00126.2(4.0 to NA)The upper quartile for the placebo group could not be calculated because more than 25% of placebo-treated subjects reached the endpoint of acute worsening and thus were assigned an arbitrarily large attack duration for purposes of analysis.