Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria
| Status: | Recruiting |
|---|---|
| Conditions: | Nephrology |
| Therapuetic Areas: | Nephrology / Urology |
| Healthy: | No |
| Age Range: | 18 - 80 |
| Updated: | 1/23/2019 |
| Start Date: | November 1, 2018 |
| End Date: | December 2020 |
| Contact: | Frank Modersitzki |
| Email: | Frank.Modersitzki@nyumc.org |
| Phone: | 212 263 0744 |
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.
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.
Inclusion Criteria:
- Patients with a confirmed laboratory diagnosis of cystinuria who meet the following
criteria: (1) stone analysis demonstrating cystine as a component, or (2) increased
urinary cystine excretion (>250mg/24hrs in adults).
- A medical regimen that includes Tiopronin.
- Willing to use a medically accepted form of birth control, if female and of child
bearing- potential
- Ability to reliably urinate in a collection vessel and measure urine volume.
- Ability to give informed consent.
- Documentation of a stable complete blood count (CBC) and urinalysis (UA) in the six
month period prior to the date of enrollment.
- Enrollment in Rare Kidney Stone Consortium (RKSC) Protocol 6401 (Cystinuria Registry)
Exclusion Criteria:
- Women who are pregnant, breastfeeding, or trying to become pregnant
- Patients with renal colic
- Patients who are scheduled to undergo a surgical procedure
- Inability to give informed consent
We found this trial at
1
site
550 1st Ave
New York, New York 10016
New York, New York 10016
(212) 263-7300
Principal Investigator: David Goldfarb, MD
Phone: 212-263-0744
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