A Phase 2 interventional study of Tiopronin in Cystinuria, sponsored by NYU Langone Health. Completed at 1 site in United States. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2021-12-17.
Sponsored by NYU Langone Health · Phase 2, Interventional, and Treatment
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.
Exclusion Criteria:
Drug: Tiopronin
Phase 1: No medication, Phase 2: 500mg PO daily x 7 days (total 500mg), Phase 3: 500mg PO BID x 7 days (total 1g), Phase 4: 1g PO BID x 7 days (total 2g)
Change in Cystine Capacity From Baseline
This measure reflects the ability of urine to take up more cystine
Time frame: Baseline; Day 7
| Milestone | Cystinuria Patients |
|---|---|
| Started | 10 |
| Completed | 10 |
| Not completed | 0 |
This measure reflects the ability of urine to take up more cystine
| mg/L | Cystinuria Patients |
|---|---|
| Phase 1 | -63.6 ± 24.8 |
| Phase 2 | 76 ± 18.1 |
| Phase 3 | 53.4 ± 17.7 |
| Phase 4 | 103.1 ± 27.4 |
Collected over 30 days. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cystinuria Patients | 0/10 (0%) | 0/10 (0%) | 0/10 (0%) |
Multiple efforts to contact the PI/study team for the relevant data have failed. Therefore, per intervention data are not available to be reported for this assessment.
| Age, Continuous(years) | Cystinuria Patients |
|---|---|
| Mean | 45.3 ± 15.42 |
| Sex: Female, Male(Participants) | Cystinuria Patients |
|---|---|
| Female | 5 |
| Male | 5 |
| Race (NIH/OMB)(Participants) | Cystinuria Patients |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 1 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 9 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | Cystinuria Patients |
|---|---|
| United States | 10 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes — All of the individual participant data collected during the trial, after deidentification. Researchers who provide a methodologically sound proposal.
Supporting information: Csr, Analytic code
This study is completed, as verified in Nov 2021. You cannot join it, but the record below documents what was studied.
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