Hyper- and Hypokalemic Periodic Paralysis Study

• Status: Completed
• Trial end date: 2013-05-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Rochester
• Collaborator: National Institute of Neurological Disorders and Stroke (NINDS)
• Treatments: Dichlorphenamide

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)

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