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

Not Applicable

Completed

• Conditions: Cystinuria
• Interventions: Drug: Tolvaptan

• Registration Number: NCT02538016
• Lead Sponsor: Caleb Nelson

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2015-07-21
• Study First Submitted QC: 2015-08-31
• Study First Posted: 2015-09-02
• Study Start: 2016-10
• Primary Completion: 2018-12
• Study Completion: 2018-12
• Status Verified: 2020-05
• Results First Submitted: 2020-05-01
• Results First Submitted QC: 2020-05-19
• Results First Posted: 2020-06-04
• Last Update Submitted: 2020-06-25
• Last Update Posted: 2020-07-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 4

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Tolvaptan	Tolvaptan	-

Primary Outcome Measures

Name	Time	Method
Urinary Cystine Supersaturation (mg/L) at High Dose (Day7-8)	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 Outcome Measures

Name	Time	Method
Urine Osmolality at High Dose (Day 8)	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).

Trial Locations

Locations (1)

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States