Disease Directory Cystinuria
Renal

Cystinuria

Also known as: cystine urolithiasis, SLC3A1/SLC7A9 deficiency, recurrent cystine stones

Prevalence

Approximately 1 in 7,000

Onset

Childhood to early adulthood (first stone event often in 2nd decade)

Type

Rare inherited aminoaciduria

Gene

SLC3A1, SLC7A9

About Cystinuria

Cystinuria is an autosomal recessive disorder of renal tubular transport affecting the reabsorption of cystine and dibasic amino acids, resulting in abnormally high urinary cystine excretion and recurrent cystine kidney stone formation. Cystine stones are characteristically radiopaque, large, staghorn-forming, and resistant to extracorporeal shockwave lithotripsy, frequently requiring urological intervention. Chronic stone burden and obstructive nephropathy contribute to progressive renal impairment in many patients.

Common Clinical Features

Recurrent cystine nephrolithiasis (often from childhood) Renal colic and flank pain Haematuria Urinary tract infections Obstructive nephropathy Staghorn calculi on imaging Progressive decline in renal function with repeated obstruction

Clinical Trial Eligibility Tips

What to know before applying to Cystinuria trials.

Urinary cystine quantification (24-hour urine cystine excretion) is the central biomarker for both diagnosis and trial eligibility; establish reliable baseline measurements.

Stone analysis confirming cystine composition, along with SLC3A1 or SLC7A9 genotyping, strengthens eligibility for genotype-specific studies.

Fluid intake and urinary pH are modifiable variables that affect stone risk; trials may specify minimum urine output targets as inclusion criteria or ask that current urine alkalinisation therapy be documented.

Patient Resources

Patient Organization

Cystinuria Support Network

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Orphanet

European reference resource for rare diseases (ORPHA:214)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

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Find recruiting Cystinuria trials

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

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