Clinical Trials Logo

Clinical Trial Summary

The purpose of this study is to evaluate the effect of escalating doses of cystine biding thiol drugs, including tiopronin and d-penicillamine, on the urinary cystine capacity, which is a measure of the amount of cystine in the urine, in patients with cystinuria. The overall goal will be to help guide therapy and ultimately minimize unnecessary side effects caused by larger doses.


Clinical Trial Description

Cystinuria is a rare genetic cause of kidney stones that leads to significant morbidity due to the recurrent nature of the disease. As a result, a significant part of treatment is focused on prevention of stone formation. Current methods of prevention include increasing fluid intake, dietary modifications, alkali therapy, and cystine binding thiol drugs (CBTDs), which help increase the solubility of cystine in the urine. At present, the dosing of CBTDs is empiric, and not titrated to a specific measured effect. Our primary objective will be to measure the effect of increasing doses of CBTDs on cystine capacity. The investigators predict that higher dosages of these medications will lead to a more positive urinary cystine capacity, or the ability of urine to take up more cystine (and therefore decreased risk of stone formation). However, the dose at which increasing dosages of the medications cease to provide additional benefit is unknown. The investigators will directly measure the cystine capacity in the urine in response to increasing doses of medications to determine if there is a dose at which the maximum benefit of the drug exists. This is a cross-over trial of escalating doses. There will be four parts to the study. In the first part, subjects will stop taking CBTDs for 7 days, and perform a 24-hour urine collection on day 7. In part 2, they will take their usual CBTD (either tiopronin or d-penicillamine) 1 gram per day for 7 days. They will perform another 24-hour urine collection on day 7 of this study period. In part 3, they will take their usual CBTD at 2 grams per day for 7 days and in part 4 they will take their CBTD at 3 grams per day for 7 days. Again, they will perform 24-hour urine collections on day 7 of each period. The order in which the parts of the study are performed will be randomized for each subject. The mean cystine capacities in each part of the study will be compared to determine the effect of drug dosage on urinary cystine capacity. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT02125721
Study type Interventional
Source NYU Langone Health
Contact
Status Completed
Phase Phase 4
Start date June 2012
Completion date December 2017

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
Recruiting NCT04818034 - The Effect of Sodium-glucose Cotransporter (SGLT) 2 Inhibitors on Cystine Stone Formation: A Preliminary Study Phase 2
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
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