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Salt Supplementation in Gitelman Syndrome

Primary Purpose

Gitelman Syndrome

Status
Recruiting
Phase
Not Applicable
Locations
Netherlands
Study Type
Interventional
Intervention
Salt (NaCl)
Placebo
Sponsored by
Radboud University Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Gitelman Syndrome

Eligibility Criteria

16 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • RadboudumcRecruiting
  • Erasmus MCRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

Salt (NaCl)

Placebo

Arm Description

12 grams (12 capsules) of NaCl per day Note: participants will be treated with active and placebo comparator (N-of-1 trial design)

12 capsules of placebo per day Note: participants will be treated with active and placebo comparator (N-of-1 trial design)

Outcomes

Primary Outcome Measures

Change in serum Potassium
Change in serum potassium
Change in symptoms measured by personalized symptom score sheet
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

Secondary Outcome Measures

Change in serum sodium
Change in serum sodium
Change in serum magnesium
Change in serum magnesium
Change in serum chloride
Change in serum chloride
Change in serum bicarbonate
Change in serum bicarbonate
Change in serum calcium
Change in serum calcium
Change in serum creatinine
Change in serum creatinine
Change in serum aldosterone
Change in serum aldosterone
Change in serum renin
Change in serum renin
Change in fractional urinary excretion of potassium
Change in fractional urinary excretion of potassium
Change in fractional urinary excretion of sodium
Change in fractional urinary excretion of sodium
Change in fractional urinary excretion of chloride
Change in fractional urinary excretion of chloride
Change in fractional urinary excretion of magnesium
Change in fractional urinary excretion of magnesium
Change in urinary calcium/creatinine ratio
Change in urinary calcium/creatinine ratio
Change in urinary excretion of renin
Change in urinary excretion of renin
Change in urinary excretion of aldosterone
Change in urinary excretion of aldosterone
Gitelman symptom questionnaire
With the Gitelman symptom questionnaire, symptoms can be scored for frequency of apperance and severity. This questionnaire also contains the 36-Item Short Form Health Survey (RAND SF-36) quality of life questionnaire
Blood pressure (including orthostatic hypotension measurement)
Measurement of blood pressure including measurement of orthostatic blood pressure
Weight
Body Weight
Muscle strength
Muscle strength will be measured by hand grip dynamometer

Full Information

First Posted
July 18, 2021
Last Updated
October 25, 2022
Sponsor
Radboud University Medical Center
Collaborators
Dutch Kidney Foundation
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1. Study Identification

Unique Protocol Identification Number
NCT04995627
Brief Title
Salt Supplementation in Gitelman Syndrome
Official Title
Exploratory Study Into the Effect of Salt Supplementation in Gitelman Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
October 2022
Overall Recruitment Status
Recruiting
Study Start Date
April 19, 2021 (Actual)
Primary Completion Date
July 1, 2023 (Anticipated)
Study Completion Date
July 1, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Radboud University Medical Center
Collaborators
Dutch Kidney Foundation

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gitelman Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Crossover Assignment
Model Description
Single subject multiple randomized double-blinded multi-crossover placebo-controlled trials (N-of-1 trials)
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Salt (NaCl)
Arm Type
Active Comparator
Arm Description
12 grams (12 capsules) of NaCl per day Note: participants will be treated with active and placebo comparator (N-of-1 trial design)
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
12 capsules of placebo per day Note: participants will be treated with active and placebo comparator (N-of-1 trial design)
Intervention Type
Dietary Supplement
Intervention Name(s)
Salt (NaCl)
Intervention Description
12 grams of salt (NaCl) per day
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Placebo
Primary Outcome Measure Information:
Title
Change in serum Potassium
Description
Change in serum potassium
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Change in symptoms measured by personalized symptom score sheet
Description
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
Time Frame
Weekly (week 0 till week 24)
Secondary Outcome Measure Information:
Title
Change in serum sodium
Description
Change in serum sodium
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Change in serum magnesium
Description
Change in serum magnesium
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Change in serum chloride
Description
Change in serum chloride
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Change in serum bicarbonate
Description
Change in serum bicarbonate
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Change in serum calcium
Description
Change in serum calcium
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Change in serum creatinine
Description
Change in serum creatinine
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Change in serum aldosterone
Description
Change in serum aldosterone
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Change in serum renin
Description
Change in serum renin
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Change in fractional urinary excretion of potassium
Description
Change in fractional urinary excretion of potassium
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Change in fractional urinary excretion of sodium
Description
Change in fractional urinary excretion of sodium
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Change in fractional urinary excretion of chloride
Description
Change in fractional urinary excretion of chloride
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Change in fractional urinary excretion of magnesium
Description
Change in fractional urinary excretion of magnesium
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Change in urinary calcium/creatinine ratio
Description
Change in urinary calcium/creatinine ratio
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Change in urinary excretion of renin
Description
Change in urinary excretion of renin
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Change in urinary excretion of aldosterone
Description
Change in urinary excretion of aldosterone
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Gitelman symptom questionnaire
Description
With the Gitelman symptom questionnaire, symptoms can be scored for frequency of apperance and severity. This questionnaire also contains the 36-Item Short Form Health Survey (RAND SF-36) quality of life questionnaire
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Blood pressure (including orthostatic hypotension measurement)
Description
Measurement of blood pressure including measurement of orthostatic blood pressure
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Weight
Description
Body Weight
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24
Title
Muscle strength
Description
Muscle strength will be measured by hand grip dynamometer
Time Frame
Baseline, week 4, week 8, week 12, week 16, week 20, week 24

10. Eligibility

Sex
All
Minimum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Maartje FA Verploegen, MD
Phone
+31243614761
Email
Maartje.Verploegen@radboudumc.nl
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Tom Nijenhuis, MD PhD
Organizational Affiliation
Radboud University Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Radboudumc
City
Nijmegen
State/Province
Gelderland
ZIP/Postal Code
6500HB
Country
Netherlands
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Maartje FA Verploegen, MD
Phone
+31243614761
Email
Maartje.Verploegen@radboudumc.nl
First Name & Middle Initial & Last Name & Degree
Tom Nijenhuis, MD PhD
Phone
+31243614761
Email
Tom.Nijenhuis@radboudumc.nl
Facility Name
Erasmus MC
City
Rotterdam
State/Province
Zuid-Holland
ZIP/Postal Code
3000CA
Country
Netherlands
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ewout J Hoorn, MD PhD
Phone
+31107040565
Email
e.j.hoorn@erasmusmc.nl

12. IPD Sharing Statement

Plan to Share IPD
Undecided

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Salt Supplementation in Gitelman Syndrome

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