Clinical Trials Logo

Clinical Trial Summary

Cystinuria is an inherited autosomal recessive disorder of the kidney that is the result of an inability to reabsorb cystine from the urine. Supersaturation of cystine in the urine produces crystals that precipitate and form stones in the kidney, which can be a cause of obstruction, infection, and chronic kidney disease. Cystine stones constitute a major health challenge for affected individuals with cystinuria because of the frequent recurrence of painful symptoms and the current absence of effective, patient-accepting treatment. A mainstay of therapy is breaking or preventing the cystine bond on the molecular level such that cystine (which is formed from the joining of two cysteine amino acids and their corresponding sulfur atoms) cannot precipitate in the urine. It is hypothesized that a glucose molecule may be able to do this if introduced into the urine. SGLT-2 inhibitors are a class of drug that are FDA approved to treat diabetes mellitus (DM) and heart failure by inhibiting an enzyme in the kidney that allows for reabsorption of glucose from the urine. This effectively increases the concentration of glucose in the urine. Our hypothesis suggests that administration of this drug to patients with cystine will introduce sufficient glucose into the urine to prevent the formation of cystine stones. To date, there has been no published data on the effectiveness of this therapy for this indication, although the dosage and administration would be identical to that already approved by the FDA for the treatment of DM and heart failure.


Clinical Trial Description

This is a single center, proof of concept prospective cohort trial designed to assess the effect of daily oral administration of dapagliflozin on cystine formation in freshly voided urine. Five subjects are planned, each with previously diagnosed cystinuria and without current treatment except with potassium citrate medication. Total duration of subject participation with be up to four weeks. Total duration of the study is expected to be 6 months. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04818034
Study type Interventional
Source University of California, San Francisco
Contact Victoria Hogue
Phone 415-302-7443
Email Victoria.Hogue@ucsf.edu
Status Recruiting
Phase Phase 2
Start date May 2, 2025
Completion date June 1, 2026

See also
  Status Clinical Trial Phase
Completed NCT02120105 - Cystine Capacity Clinical Study (CysCap)
Completed NCT02538016 - TCUPS- Tolvaptan Use in Cystinuria and Urolithiasis: A Pilot Study N/A
Recruiting NCT06065852 - National Registry of Rare Kidney Diseases
Recruiting NCT00169806 - Randall's Plaque Study: Pathogenesis and Relationship to Nephrolithiasis N/A
Completed NCT03663855 - Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria Phase 2
Recruiting NCT02780297 - Prospective Research Rare Kidney Stones (ProRKS)
Recruiting NCT02026388 - Rare Kidney Stone Consortium Biobank
Withdrawn NCT04137978 - Study Evaluating Patients With Cystinuria Phase 2/Phase 3
Recruiting NCT02942420 - Bucillamine Phase 2 Trial in Patients With Cystinuria Phase 2
Completed NCT05048563 - Registry of Thiola EC Therapy
Not yet recruiting NCT04147871 - Study Evaluating Patients With Cystinuria and Efficacy and Safety Exploratory Study in the Youngest Children Phase 2/Phase 3
Completed NCT02125721 - Effect of Increasing Doses of Cystine Binding Thiol Drugs on Cystine Capacity in Patients With Cystinuria Phase 4
Active, not recruiting NCT02910531 - Lipoic Acid Supplement for Cystine Stone Phase 2
Recruiting NCT00588562 - Rare Kidney Stone Consortium Patient Registry
Completed NCT02124395 - Health-related Quality of Life in Rare Kidney Stone
Not yet recruiting NCT05058859 - Long Term Clinical Efficacy of Sodium-glucose Cotransporter-2 (SGLT-2) Inhibitor in Cystinurics Phase 2
Active, not recruiting NCT03539926 - This Study Evaluates the Superiority of Daily Self-pH Monitorization of Lit-control®pH Meter Compared to the Monitorization of Reactive Strips (Standard of Care). N/A
Completed NCT00381849 - Use of an Herbal Preparation to Prevent and Dissolve Kidney Stones Phase 1/Phase 2
Completed NCT03836144 - Effect of Urine Alkalinazation on Urinary Inflammatory Markers in Patients With Cystinuria