Hyper- and Hypokalemic Periodic Paralysis Study



Status:Completed
Conditions:Neurology
Therapuetic Areas:Neurology
Healthy:No
Age Range:18 - Any
Updated:4/21/2016
Start Date:June 2007
End Date:May 2013

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Dichlorphenamide vs. Placebo for Periodic Paralysis

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.

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.

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)

1. Prolonged QT interval or complex ventricular ectopy between attacks

2. Distinctive physical features (2 of the following 5)

1. Low set ears

2. Short stature

3. Hypo-/micrognathia

4. Clinodactyly

5. Hypo-/hypertelorism

3. 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
We found this trial at
11
sites
75 Francis street
Boston, Massachusetts 02115
(617) 732-5500
Brigham and Women's Hosp Boston’s Brigham and Women’s Hospital (BWH) is an international leader in...
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Boston, MA
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60 Crittenden Blvd # 70
Rochester, New York 14642
(585) 275-2121
University of Rochester The University of Rochester is one of the country's top-tier research universities....
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Rochester, NY
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281 W. Lane Ave
Columbus, Ohio 43210
(614) 292-6446
Ohio State University The Ohio State University’s main Columbus campus is one of America’s largest...
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Columbus, OH
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Dallas, TX
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3901 Rainbow Blvd
Kansas City, Kansas 66160
(913) 588-5000
University of Kansas Medical Center The University of Kansas Medical Center serves Kansas through excellence...
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Kansas City, KS
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Los Angeles, California 90095
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Los Angeles, CA
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630 W 168th St
New York, New York
212-305-2862
Columbia University Medical Center Situated on a 20-acre campus in Northern Manhattan and accounting for...
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New York, NY
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Rochester, Minnesota 55905
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Rochester, MN
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San Donato, Milan
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San Donato,
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San Francisco, California 94143
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San Francisco, CA
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St Louis, Missouri 63108
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St Louis, MO
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