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Completed

NCT Number: NCT03663855

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

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.

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Key information

Conditions

Cystinuria Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Kidney Diseases Male Urogenital Diseases Renal Aminoacidurias Renal Tubular Transport, Inborn Errors Urogenital Diseases Urologic Diseases

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

New York University School of Medicine

New York, 10016, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Tiopronin

Drug

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 outcomes

  1. Change in Cystine Capacity From Baseline

    Time frame: Baseline; Day 7

    This measure reflects the ability of urine to take up more cystine

Sponsors and collaborators

Lead sponsor

NYU Langone Health

Other

Registry information

Important dates

Study start
2018
Primary completion
2019
Study completion
2019
First posted
Sep 10, 2018
Registry last updated
Dec 17, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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