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Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03663855
Recruitment Status : Completed
First Posted : September 10, 2018
Last Update Posted : January 13, 2020
Sponsor:
Information provided by (Responsible Party):
NYU Langone Health

Brief 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.


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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 11 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

Drug Information available for: Tiopronin

Arm Intervention/treatment
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.




Primary Outcome Measures :
  1. Measure of Cystine Capacity [ Time Frame: 12 Months ]
    This measure reflects the ability of urine to take up more cystine



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

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


Locations
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United States, New York
New York University School of Medicine
New York, New York, United States, 10016
Sponsors and Collaborators
NYU Langone Health
Investigators
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Principal Investigator: David Goldfarb, MD NYU Langone Health
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Responsible Party: NYU Langone Health
ClinicalTrials.gov Identifier: NCT03663855    
Other Study ID Numbers: 18-00642
First Posted: September 10, 2018    Key Record Dates
Last Update Posted: January 13, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: All of the individual participant data collected during the trial, after deidentification. Researchers who provide a methodologically sound proposal.
Supporting Materials: Clinical Study Report (CSR)
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Cystinuria
Renal Aminoacidurias
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases