Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria
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|ClinicalTrials.gov Identifier: NCT03663855|
Recruitment Status : Completed
First Posted : September 10, 2018
Last Update Posted : January 13, 2020
The purpose of this study is to determine the minimum effective dose of the cysteine binding thiol drug (CBTD) Tiopronin on urine cystine capacity, which is a measure of cystine solubility in the urine, in patients with cystinuria to evaluate the effect of escalating doses of cystine binding thiol drugs on the cystine capacity of the urine. The overall goal will be to help guide therapy and ultimately minimize unnecessary side effects caused by larger dosages.
Cystinuria is a rare genetic disease that can lead to significant morbidity in affected patients due to the recurrent nature of the disease. This study will follow the levels of urine cystine capacity in order to help guide treatment and to use lower than usually prescribed Tiopronin doses to decrease the potential side effects while maintaining therapeutic benefit. This will increase adherence with the medications by decreasing the burden of the large number of pills that need to be taken daily.
|Condition or disease||Intervention/treatment||Phase|
|Cystinuria||Drug: 7 Days Tiopronin Drug: 500 mg Tiopronin Drug: 1 grams per day of Tiopronin Drug: 2 grams per day of Tiopronin||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||11 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria|
|Actual Study Start Date :||November 1, 2018|
|Actual Primary Completion Date :||September 10, 2019|
|Actual Study Completion Date :||September 10, 2019|
|Experimental: Cystinuria Patients||
Drug: 7 Days Tiopronin
On day 7 of this period,patients will be asked to perform a 24-hour urine collection to measure the urinary cystine levels.
Drug: 500 mg Tiopronin
5 tablets a day (200mg tab AM and 300mg tab PM). On day 7 of this period, you will perform a 24-hour urine collection.
Drug: 1 grams per day of Tiopronin
5 tabs twice a day (100mg per tab). Patients will have another 24-hour urine collection on day 7 of this period.
Drug: 2 grams per day of Tiopronin
10 tabs twice a day (100mg per tab). On day 7 of this period, patients will perform the final 24-hour urine collection.
- Measure of Cystine Capacity [ Time Frame: 12 Months ]This measure reflects the ability of urine to take up more cystine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03663855
|United States, New York|
|New York University School of Medicine|
|New York, New York, United States, 10016|
|Principal Investigator:||David Goldfarb, MD||NYU Langone Health|