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.
21 studies on the registry are indexed under Cystinuria; 5 are open to participants now.
Browse Cystinuria studies →NYU Langone Health is the lead sponsor of 1,430 studies on the registry; 258 are open to participants now.
Of its 235 completed or terminated interventional studies of FDA-regulated products, 197 (84%) have results posted.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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 |
PDFs hosted by the registry, ClinicalTrials.gov.
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: Clinical study report, Analytic code
Time frame: Beginning 9 months and ending 36 months following article publication.
Access criteria: Proposals may be submitted up to 36 months following article publication. After 36 months the data will be available in our University's data warehouse but without investigator support other than deposited metadata. Information regarding submitting requests and accessing data may be found at (Link to be provided
Ran from Nov 2018 to primary completion in Sep 2019, 10 months.
Results were first posted in Dec 2021, 2 years 3 months after primary completion.
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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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Studies of the drug tested here: Tiopronin