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Hyper- and Hypokalemic Periodic Paralysis Study (HYP-HOP)
This study is currently recruiting participants.
Verified by University of Rochester, February 2010
First Received: June 27, 2007   Last Updated: February 4, 2010   History of Changes
Sponsor: University of Rochester
Collaborator: National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by: University of Rochester
ClinicalTrials.gov Identifier: NCT00494507
  Purpose

The purpose of this study is to compare Dichlorphenamide with placebo (an inactive substance) for prevention of episodes and for improvement of strength in periodic paralysis. This study will also look at the long-term effects of Dichlorphenamide in periodic paralysis.


Condition Intervention Phase
Periodic Paralysis
Drug: Dichlorphenamide
Drug: Placebo
Phase III

Study Type: Interventional
Study Design: Treatment, Randomized, Double Blind (Subject, Investigator), Placebo Control, Parallel Assignment, Efficacy Study
Official Title: Dichlorphenamide vs. Placebo for Periodic Paralysis

Resource links provided by NLM:


Further study details as provided by University of Rochester:

Primary Outcome Measures:
  • The number of attacks/week over the last 8 weeks of the initial 9-week study phase. [ Time Frame: 8 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Efficacy: severity-weighted attack rate; muscle strength and mass measures; changes in health-related quality-of-life; intolerable increase in attack frequency or severity necessitating withdrawal from the 9-week phase (HOP trial only). [ Time Frame: 8 to 61 weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 140
Study Start Date: June 2007
Estimated Study Completion Date: November 2012
Estimated Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Dichlorphenamide: Active Comparator Drug: Dichlorphenamide
has been prescribed to treat periodic paralysis, but is no longer available
Placebo: Placebo Comparator
inactive substance
Drug: Placebo
an inactive substance

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 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 computer diary to record the time, length, and severity of each episode of weakness. 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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Genetically definite, clinically definite or clinically probable HYP or HOP 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 out of 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00494507

Contacts
Contact: Patty Smith 585-275-4339

Locations
United States, California
University of California-San Francisco Recruiting
San Francisco, California, United States, 94143
Contact: Claudia Villierme     415-476-2662        
Principal Investigator: Jeffrey Ralph, MD            
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Victoria Watts     913-588-5479        
Principal Investigator: Richard Barohn, MD            
United States, Massachusetts
Brigham & Women's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Kristen Whiteside     617-525-6763        
Principal Investigator: Anthony Amato, MD            
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Janet Fisher     507-538-2433        
Principal Investigator: Brian Crum, MD            
United States, New York
University of Rochester Recruiting
Rochester, New York, United States, 14642
Contact: Jill Scheltz     585-276-5432        
Principal Investigator: Emma Ciafaloni, MD            
Columbia University Medical Center Recruiting
New York, New York, United States, 10032
Contact: Kate Dalton     212-305-2027        
Principal Investigator: Jinsy Andrews, MD            
United States, Ohio
Ohio State University Recruiting
Columbus, Ohio, United States, 43210
Contact: Amy Bartlett     614-366-9050        
Principal Investigator: John Kissel, MD            
United States, Texas
University of Texas Southwestern-Dallas Recruiting
Dallas, Texas, United States, 75390
Contact: Nina Gorham     214-648-0462        
Principal Investigator: Stephen C. Cannon, MD            
France
Hospital Pitie-Salpetriere, Salpetriere Not yet recruiting
Paris, France
Contact: Savine Vicart     33 140 778119        
Principal Investigator: Bertrand Fontaine, MD            
Italy, Milan
University of Milan Not yet recruiting
San Donato, Milan, Italy
Contact: Valeria Sansone     39 02 5607450        
Principal Investigator: Giovanni Meola, MD            
United Kingdom
Institute of Neurology-Queen's Square Not yet recruiting
London, United Kingdom
Contact: Gisela Barreto     011 44 207 837 3611 ext 3888        
Principal Investigator: Michael Hanna, MD            
Sponsors and Collaborators
University of Rochester
Investigators
Principal Investigator: Robert C. Griggs, M.D. University of Rochester
Investigator: Rabi Tawil, M.D. Co-Principal Investigator, University of Rochester
Investigator: Michael McDermott, Ph.D. Biostatistician, University of Rochester
  More Information

No publications provided

Responsible Party: University of Rochester ( Robert C. Griggs, MD )
Study ID Numbers: R01NS045686-02
Study First Received: June 27, 2007
Last Updated: February 4, 2010
ClinicalTrials.gov Identifier: NCT00494507     History of Changes
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Rochester:
periodic paralysis
dichlorphenamide

Additional relevant MeSH terms:
Metabolic Diseases
Molecular Mechanisms of Pharmacological Action
Dichlorphenamide
Nervous System Diseases
Hypokalemic Periodic Paralysis
Enzyme Inhibitors
Metal Metabolism, Inborn Errors
Pharmacologic Actions
Paralysis
Metabolism, Inborn Errors
Signs and Symptoms
Carbonic Anhydrase Inhibitors
Paralyses, Familial Periodic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Neurologic Manifestations

ClinicalTrials.gov processed this record on February 08, 2010