Acetazolamide (AZ) for Management of Alkalosis in Bartter Syndrome

Study Overview
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Brief Summary
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In this prospective controlled cross over clinical trial, the investigators aim to evaluate the efficacy and safety of acetazolamide for the management of metabolic alkalosis in children with Bartter syndrome. Urine and blood electrolytes will be measured before and after acetazolamide treatment. The primary end point is a change in polyuria, hypokalemia, and metabolic alkalosis.

Detailed Description
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Bartter syndrome is a hereditary salt-loosing tubulopathy caused by several gene mutations encoding the sodium reabsorption in the thick ascending limb of loop of Henle, with poor response to treatment. The effects of inhibition of proximal tubular reabsorption of bicarbonate by acetazolamide have not been previously studied in Batter patients. The present study is designed to assess he efficacy and safety of acetazolamide for the management of children with Bartter syndrome. The primary end point is change in polyuria, hypokalemia, and metabolic alkalosis. In this prospective observational crossover clinical trial, patients between ages 1 and 10 years with clinical diagnosis of Bartter syndrome (hypokalemia, metabolic alkalosis, normal blood pressure, elevated urine chloride >20 milliequivalent per liter, high serum aldosterone and plasma renin levels) will be enrolled in a 4- week clinical trial. After initial clinical and laboratory evaluations, patients will receive acetazolamide 5.0 mg/kg orally as a single daily dose and each patient will act as his/her own control. Renal electrolyte and 24-hour urine output will be measured at baseline and after the 4 weeks acetazolamide treatment.

Official Title
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Acetazolamide (AZ) for Management of Refractory Hypokalemia Metabolic Alkalosis in Bartter Syndrome

Conditions
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Bartter Syndrome

Intervention / Treatment
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* Drug: Acetazolamide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Hypokalemia metabolic alkalosis normal blood pressure random urine chloride >20 milliequivalent per liter (mEq/L) Elevated serum aldosterone and renin levels Exclusion Criteria: Hypertension History of emesis Prior use of laxatives Cystic fibrosis ofpancrease

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 10 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Acetazolamide<br>Oral administration of acetazolamide 5 mg/kg/day for 4 weeks | Drug: Acetazolamide<br>* Correction of metabolic alkalosis by inhibition of the filtered bicarbonate load reabsorption in the proximal tubules using acetazolamide (AZ)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Metabolic alkalosis | Change in serum bicarbonate level | 4 weeks | 
| Urine output | Change in 24-hr urine volume | 4 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Acetazolamide, Hypokalemic metabolic alkalosis, Bartter syndrome