Patients with hyperkalemic (HYP) and hypokalemic (HOP) periodic paralysis. MedDRA version: 12.1 Level: LLT Classification code 10016208 Term: Familial periodic paralysis
Conditions
Interventions
Product Name: Dichlorphenamide
Product Code: DCP
Pharmaceutical Form: Tablet
INN or Proposed INN: Dichlorphenamide
CAS Number: 120-97-8
Sponsors
University of Rochester
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Inclusion criteria for Hyperkalemic Periodic Paralysis (HYP): 1. Genetically definite, clinically definite or clinically probable HYP. 2. Male and female participants, age 18 and older who are able to comply with the study procedures. 3. Participants will be required to have distinct regular episodes of weakness by history with an average frequency of >1 /week and 1 /week and =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Exclusion Criteria for Hyperkalemic Periodic Paralysis: None of the following can be present: 1. Evidence for Andersen-Tawil syndrome (any one of the following 3 criteria) • Prolonged QT interval or complex ventricular ectopy between attacks • KIR 2.1 gene mutation • Distinctive physical features (2 out of 5) o Low set ears o Short stature o Hypo-/micro-gnathia o Clinodactyly o Hypo-/hypertelorism 2. Coincidental renal, hepatic, restrictive or obstructive lung disease, active thyroid, or heart disease 3. Chronic, non-congestive, angle-closure glaucoma 4. 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 5. History of life-threatening episodes of respiratory muscle weakness or cardiac arrhythmias during attacks (prior to treatment) 6. History of worsening symptoms with the use of CAI’s 7. Any other neuromuscular disease Exclusion criteria for Hypokalemic Periodic Paralysis: None of the following can be present: 1. Known mutation in the a subunit of sodium channel. 2. 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 out of 5) o Low set ears o Short stature o Hypo-/micrognathia o Clinodactyly o Hypo-/hypertelorism • KIR 2.1 gene mutation 3. Coincidental renal, hepatic, active thyroid disease, restrictive or obstructive lung disease, or heart disease 4. Chronic, non-congestive, angle-closure glaucoma 5. 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 6. History of life-threatening episodes of respiratory muscle weakness or cardiac arrhythmias during attacks (prior to treatment) 7. History of worsening symptoms with the use of CAI’s 8. Any other neuromuscular disease
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess whether dichlorphenamide lowers the rate of attacks of weakness in HYP and HOP participants as measured by participant self-report over the last 8 weeks of a 9-week double blind period.; Secondary Objective: 1. To test whether a larger percentage of placebo-treated HOP participants will reach the endpoint of acute worsening than of participants taking DCP. 2. To test whether the mean scores on composite strength measures, muscle mass, and from the physical health and mental health summary scales of the SF-36 will be significantly higher at the end of the 9-weeks in participants taking DCP than in participants taking placebo. 3. To test whether, after 12 months of open label treatment with DCP, HYP and HOP participants will have a positive mean change from baseline in composite strength measures and muscle mass during attack-free intervals. ;Primary end point(s): The primary outcome variable of the initial 9-week comparisons of DCP, and placebo will be the number of attacks/week over the last 8 weeks. | — |
Countries
United Kingdom
Outcome results
None listed