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Completed

NCT Number: NCT02538016

TCUPS- Tolvaptan Use in Cystinuria and Urolithiasis: A Pilot Study

The goal of this research project is to establish that in short-term use, tolvaptan is a safe and potentially effective new therapy for cystinuria, by conducting a short-term pilot study of the safety and tolerability of this drug, and assess impact on urinary stone risk parameters, among adolescent and young adult patients with clinical cystinuria.

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Key information

Conditions

Cystinuria Calculi Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Kidney Calculi Kidney Diseases Male Urogenital Diseases Nephrolithiasis Pathological Conditions, Anatomical Pathological Conditions, Signs and Symptoms Renal Aminoacidurias Renal Tubular Transport, Inborn Errors Urinary Calculi Urogenital Diseases Urolithiasis Urologic Diseases

Age range

12 year–29 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Males and females age 12 - 29 years
  • Weight ≥ 25kg (55 lbs)
  • Confirmed cystinuria diagnosis
  • Specific blood test levels (done within the past 6 months)

Exclusion criteria

  • Concurrent non-renal disease that might increase risk of complications due to aquaresis
  • Liver or biliary disease (chronic or acute)
  • Malabsorption syndrome or other gastrointestinal condition that may interfere with response to therapy
  • Non-cutaneous malignancy within last 5 years
  • History of adverse reaction or allergy to Tolvaptan or other arginine vasopressin V2-receptor antagonists

Treatment and study plan

Tolvaptan

Drug

Primary outcomes

  1. Urinary Cystine Supersaturation (mg/L) at High Dose (Day7-8)

    Time frame: 23 days

    The primary outcome was urinary cystine supersaturation as measured by "cystine capacity". This proprietary test (Litholink Corp., Chicago IL) is reported as a value in mg/L above or below zero, with positive values indicating urine undersaturated with cystine, while negative values indicate that the urine is supersaturated with cystine. Four 24-hour urine samples were obtained during the study: one at baseline 3-6 days prior to the measurement, one on day 3-4 of the dosing period, one on day 7-8 of the dosing period, and one 3-6 days after the washout period. Each 24 hour urine sample was sent individually for analysis by Litholink Corp, which performs the cystine assays.

Secondary outcomes

  1. Urine Osmolality at High Dose (Day 8)

    Time frame: 11 days

    Secondary outcomes included serum sodium and other electrolyte levels. Serum electrolytes for each subject before, during, and after tolvaptan treatment (Sodium/Potassium/Chloride in mmol/L).

Sponsors and collaborators

Lead sponsor

Caleb Nelson

Other

Collaborators

  • Otsuka America Pharmaceutical

Registry information

Official study title

Use of Tolvaptan, a Vasopressin Antagonist, to Increase Urine Dilution and Reduce Cystine Urolithiasis Among Patients With Homozygous Cystinuria: a Pilot Investigation

Acronym: TCUPS

Important dates

Study start
2016
Primary completion
2018
Study completion
2018
First posted
Sep 2, 2015
Registry last updated
Jul 10, 2020

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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