Disease Directory Gitelman Syndrome
Renal

Gitelman Syndrome

Also known as: familial hypokalemia-hypomagnesemia, SLC12A3 mutation, Gitelman-Bartter

Prevalence

Approximately 1–10 per 40,000; one of the more common inherited tubular disorders

Onset

Adolescence to adulthood (rarely childhood)

Type

Rare autosomal recessive renal tubular disorder

Gene

SLC12A3

About Gitelman Syndrome

Gitelman syndrome is an autosomal recessive disorder caused by loss-of-function mutations in SLC12A3 encoding the thiazide-sensitive sodium-chloride cotransporter (NCC) in the distal convoluted tubule, resulting in salt wasting, hypokalaemia, hypomagnesaemia, and metabolic alkalosis. Unlike Bartter syndrome, Gitelman syndrome typically presents in older children or adults with milder symptoms including muscle weakness, fatigue, salt craving, and tetany. Renal function is usually preserved, and the condition is often diagnosed incidentally on routine electrolyte testing.

Common Clinical Features

Hypokalaemia (often asymptomatic or with fatigue) Hypomagnesaemia Salt craving Muscle weakness, cramps, and tetany Metabolic alkalosis Hypocalciuria (distinguishing from Bartter syndrome) Chondrocalcinosis in adulthood (long-term hypomagnesaemia)

Clinical Trial Eligibility Tips

What to know before applying to Gitelman Syndrome trials.

SLC12A3 biallelic mutation confirmation is the gold standard for diagnosis; clinical biochemistry alone (hypokalaemia, hypomagnesaemia, hypocalciuria) supports but does not confirm eligibility for genetic trials.

Baseline electrolyte levels on current supplementation must be characterised; some trials require a defined wash-out period from potassium or magnesium supplements before screening.

Chondrocalcinosis assessment may be required for trials evaluating long-term hypomagnesaemia complications; include joint imaging if clinically indicated.

Patient Resources

Patient Organization

Rare Renal Disease Foundation

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Natural History Registry

ERKNet Registry

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Orphanet

European reference resource for rare diseases (ORPHA:358)

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NORD

National Organization for Rare Disorders

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Find recruiting Gitelman Syndrome trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for Gitelman Syndrome. Updated daily.

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