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

June 25, 2020 updated by: Caleb Nelson

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

4

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years to 29 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Tolvaptan
Drug: Tolvaptan

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
23 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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).
11 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Caleb Nelson

Collaborators

Otsuka America Pharmaceutical

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2016

Primary Completion (Actual)

December 1, 2018

Study Completion (Actual)

December 1, 2018

Study Registration Dates

First Submitted

July 21, 2015

First Submitted That Met QC Criteria

August 31, 2015

First Posted (Estimate)

September 2, 2015

Study Record Updates

Last Update Posted (Actual)

July 10, 2020

Last Update Submitted That Met QC Criteria

June 25, 2020

Last Verified

May 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 16726

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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