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

Cystinuria Clinical Trial

— 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02538016
• Other study ID #: 16726
• Status: Completed
• Phase: N/A
• Start date: October 2016
• Est. completion date: December 2018

Study information

• Verified date: May 2020
• Source: Boston Children’s Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: December 2018
• Est. primary completion date: December 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 29 Years

Eligibility

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

Related Conditions & MeSH terms

• Cystinuria
• Urolithiasis

Intervention

Drug:
• Tolvaptan

Locations

Country	Name	City	State
United States	Boston Children's Hospital	Boston	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
Caleb Nelson	Otsuka America Pharmaceutical

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	23 days
Secondary	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).	11 days