Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria
Status:
Completed
Trial end date:
2019-09-10
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 2
Details
Lead Sponsor:
New York University School of Medicine NYU Langone Health