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

NCT02538016 | Completed Results DMC

• 1 other identifier: 16726
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

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.

Conditions

Cystinuria

Keywords

Cystinuria; Kidney Stones

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Urine Osmolality at High Dose (Day 8)

  11 days

Study Arms (1)

Tolvaptan

OTHER

Drug: Tolvaptan

Interventions

Tolvaptan DRUG

Tolvaptan

Eligibility Criteria

• Age: 12 Years - 29 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Caleb Nelson: lead
• Otsuka America Pharmaceutical: collaborator

Study Sites (1)

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

MeSH Terms

Conditions

Cystinuria; Kidney Calculi

Interventions

Tolvaptan

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Benzazepines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Alyssia Venna
• Organization: Boston Children's Hospital

Alyssia.Venna@childrens.harvard.edu

617-919-6352

Study Officials

• Caleb Nelson, MD, MPH

  Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: MD, MPH

Study Record Dates

• First Submitted: July 21, 2015
• First Posted: September 2, 2015
• Study Start: October 1, 2016
• Primary Completion: December 1, 2018
• Study Completion: December 1, 2018
• Last Updated: July 10, 2020
• Results First Posted: June 4, 2020

Record last verified: 2020-05

Locations

US (1)