Effect of Potassium and Acetazolamide on People With Andersen-Tawil Syndrome
NCT00839501 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 3
Last updated 2012-01-20
Summary
Andersen-Tawil Syndrome (ATS) is a rare genetic disorder that causes episodes of muscle weakness, potentially life-threatening changes in heart rhythm, and skeletal developmental abnormalities. The cause of some ATS cases remains unknown, and no specific treatments have been established. The purpose of this study is to determine whether potassium supplements and/or the medication acetazolamide affect the duration of muscle weakness and heart rhythm abnormalities in people with ATS.
Conditions
- Andersen-Tawil Syndrome
Interventions
- DIETARY_SUPPLEMENT
-
Potassium
40 mEq twice daily in pill form during the first 3-day inpatient visit, followed by 40 mEq twice daily in liquid form during the first 18-week treatment period, as is randomly scheduled, and continued during the second 18-week treatment period as long as there are no limiting side effects
- DRUG
-
Acetazolamide
250 mg twice daily, orally, during the second 3-day inpatient visit and during the second 18-week treatment period, as is randomly scheduled
Sponsors & Collaborators
-
Office of Rare Diseases (ORD)
collaborator NIH -
Rare Diseases Clinical Research Network
collaborator NETWORK -
National Institute of Neurological Disorders and Stroke (NINDS)
collaborator NIH -
University of Rochester
lead OTHER
Principal Investigators
-
Paul Twydell, DO · University of Rochester School of Medicine & Dentistry
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- DOUBLE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 10 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2008-12-31
- Primary Completion
- 2011-01-31
- Completion
- 2011-01-31
Countries
- United States
Study Locations
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