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

Primary Purpose

Cystinuria

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Tiopronin
Sponsored by
NYU Langone Health
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystinuria

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • New York University School of Medicine

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Cystinuria Patients

Arm Description

Outcomes

Primary Outcome Measures

Change in Cystine Capacity From Baseline
This measure reflects the ability of urine to take up more cystine

Secondary Outcome Measures

Full Information

First Posted
September 6, 2018
Last Updated
November 18, 2021
Sponsor
NYU Langone Health
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1. Study Identification

Unique Protocol Identification Number
NCT03663855
Brief Title
Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria
Official Title
Effect of Increasing Doses of Tiopronin on Cystine Capacity in Patients With Cystinuria
Study Type
Interventional

2. Study Status

Record Verification Date
November 2021
Overall Recruitment Status
Completed
Study Start Date
November 1, 2018 (Actual)
Primary Completion Date
September 10, 2019 (Actual)
Study Completion Date
September 10, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
NYU Langone Health

4. Oversight

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
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystinuria

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Cystinuria Patients
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Tiopronin
Intervention Description
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)
Primary Outcome Measure Information:
Title
Change in Cystine Capacity From Baseline
Description
This measure reflects the ability of urine to take up more cystine
Time Frame
Baseline; Day 7

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
David Goldfarb, MD
Organizational Affiliation
NYU Langone Health
Official's Role
Principal Investigator
Facility Information:
Facility Name
New York University School of Medicine
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
All of the individual participant data collected during the trial, after deidentification. Researchers who provide a methodologically sound proposal.
IPD Sharing Time Frame
Beginning 9 months and ending 36 months following article publication.
IPD Sharing 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

Learn more about this trial

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

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