Phase 3 N=65 Randomized Double-blind Treatment

Hyper- and Hypokalemic Periodic Paralysis Study

Hyperkalemic Periodic Paralysis · Hypokalemic Periodic Paralysis

Bottom Line

View on ClinicalTrials.gov: NCT00494507 ↗

Enrolled (actual)

Serious AEs

4.1%

Results posted

May 2014

Primary outcome: Primary: HYP Attack Rate — 0.9; 4.8 attacks per week

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Dichlorphenamide (double-blind) (Drug); Placebo (double-blind) (Drug); Dichlorphenamide (open-label) (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: University of Rochester
Primary completion: Apr 2013

Outcome Measures

Outcome	Result	p-value
PRIMARY HYP Attack Rate	0.9; 4.8	—
PRIMARY HOP Attack Rate	0.3; 2.4	—
SECONDARY HYP Severity-weighted Attack Rate	1.0; 5.8	—
SECONDARY HOP Severity-weighted Attack Rate	0.6; 5.7	—
SECONDARY HYP Attack Duration	10.5; 39.4	—
SECONDARY HOP Attack Duration	2.7; 26.2	—
SECONDARY HYP Endpoint of Acute Worsening	0; 2	—
SECONDARY HOP Endpoint of Acute Worsening	0; 5	—
SECONDARY HYP Change From Baseline to Week 9 in Average Manual Muscle Testing (MMT) Score	0.13; 0.00	—
SECONDARY HOP Change From Baseline to Week 9 in Average Manual Muscle Testing (MMT) Score	0.05; -0.08	—
SECONDARY HYP Change From Baseline to Week 9 in Average Maximum Voluntary Isometric Contraction Testing (MVICT) Scores	0.78; 0.55	—
SECONDARY HOP Change From Baseline to Week 9 in Average Maximum Voluntary Isometric Contraction Testing (MVICT) Scores	-0.08; -0.27	—
SECONDARY HYP Change From Baseline to Week 9 in Average Maximum Voluntary Isometric Contraction Testing Percent of Predicted Normal	10.84; 10.17	—
SECONDARY HOP Change From Baseline to Week 9 in Average Maximum Voluntary Isometric Contraction Testing Percent of Predicted Normal	-0.81; -2.94	—
SECONDARY HYP Change From Baseline to Week 9 in Lean Body Mass	-1.31; -1.52	—
SECONDARY HOP Change From Baseline to Week 9 in Lean Body Mass	-0.78; 0.44	—
SECONDARY HYP Change From Baseline to Week 9 in SF-36 Physical Component Summary Score	3.16; -0.11	—
SECONDARY HOP Change From Baseline to Week 9 in SF-36 Physical Component Summary Score	4.83; -3.75	—
SECONDARY HYP Change From Baseline to Week 9 in SF-36 Mental Health Component Summary Score	-3.77; 2.65	—
SECONDARY HOP Change From Baseline to Week 9 in SF-36 Mental Health Component Summary Score	-1.10; -5.65	—

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.

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00494507). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.