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

Primary Purpose

Cystinuria

Status

Completed

Phase

Not Applicable

Locations

United States

Study Type

Interventional

Intervention

Tolvaptan

About this trial

This is an interventional treatment trial for Cystinuria focused on measuring Cystinuria, Kidney Stones

Eligibility Criteria

12 Years - 29 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Boston Children's Hospital

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Tolvaptan

Arm Description

Outcomes

Primary Outcome Measures

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

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 Outcome Measures

Urine Osmolality at High Dose (Day 8)

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).

Full Information

NCT ID

NCT02538016

First Posted

July 21, 2015

Last Updated

June 25, 2020

Sponsor

Caleb Nelson

Collaborators

Otsuka America Pharmaceutical

1. Study Identification

Unique Protocol Identification Number

NCT02538016

Brief Title

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

Acronym

TCUPS

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

May 2020

Overall Recruitment Status

Completed

Study Start Date

October 2016 (undefined)

Primary Completion Date

December 2018 (Actual)

Study Completion Date

December 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

Caleb Nelson

Collaborators

Otsuka America Pharmaceutical

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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.

6. Conditions and Keywords

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

Cystinuria

Keywords

Cystinuria, Kidney Stones

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

4 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Tolvaptan

Arm Type

Other

Intervention Type

Drug

Intervention Name(s)

Tolvaptan

Primary Outcome Measure Information:

Title

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

Description

Time Frame

23 days

Secondary Outcome Measure Information:

Title

Urine Osmolality at High Dose (Day 8)

Description

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).

Time Frame

11 days

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Maximum Age & Unit of Time

29 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Caleb Nelson, MD, MPH

Organizational Affiliation

Boston Children's Hospital

Official's Role

Principal Investigator

Facility Information:

Facility Name

Boston Children's Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

12. IPD Sharing Statement

Learn more about this trial

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

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