Exploratory Study Into the Effect of Salt Supplementation in Gitelman Syndrome
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Enrollment
- 21
- Locations
- 3
- Primary Endpoint
- Change in symptoms measured by personalized symptom score sheet
Study Overview
Brief Summary
The purpose of this study is to determine the effect of salt (NaCl) supplementation on serum potassium and clinical signs and symptoms in patients with Gitelman syndrome.
Detailed Description
Single subject multiple randomized double-blinded multi-crossover placebo-controlled trials (N-of-1 trials) will be performed. Participants will enroll the study after giving written informed consent. The individual trials will consist of 3 treatment blocks which each contain a 4-week treatment period of salt supplementation (12 grams of NaCl) and a 4-week treatment period of placebo, in a randomized, blinded order. After each period of 4 weeks, outcomes measures will be measured.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Crossover
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 16 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Genetically-proven, symptomatic Gitelman syndrome
- •Written informed consent
Exclusion Criteria
- •inability to discontinue potassium-sparing diuretics, mineralocorticoid antagonists and NSAIDs; this means inability to reach a potassium level of 2.5 mmol/L or higher with maximally tolerable potassium supplementation after discontinuation of potassium-sparing diuretics
- •pregnancy
Arms & Interventions
Salt (NaCl)
12 grams (12 capsules) of NaCl per day
Note: participants will be treated with active and placebo comparator (N-of-1 trial design)
Intervention: Salt (NaCl) (Dietary Supplement)
Placebo
12 capsules of placebo per day
Note: participants will be treated with active and placebo comparator (N-of-1 trial design)
Intervention: Placebo (Other)
Outcomes
Primary Outcomes
Change in symptoms measured by personalized symptom score sheet
Time Frame: Weekly (week 0 till week 24)
Symptoms will be scored via a personalized symptom score sheet, which will be composed with the individual participant. Symptoms will be scored at a range from 0 - 10
Change in serum Potassium
Time Frame: Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Change in serum potassium
Secondary Outcomes
- Change in serum bicarbonate(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in serum renin(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in fractional urinary excretion of potassium(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in fractional urinary excretion of sodium(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in fractional urinary excretion of magnesium(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in urinary excretion of aldosterone(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Gitelman symptom questionnaire(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Weight(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in serum chloride(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in fractional urinary excretion of chloride(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in urinary calcium/creatinine ratio(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in urinary excretion of renin(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in serum sodium(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in serum magnesium(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in serum calcium(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in serum creatinine(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Change in serum aldosterone(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Blood pressure (including orthostatic hypotension measurement)(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)
- Muscle strength(Baseline, week 4, week 8, week 12, week 16, week 20, week 24)