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

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ClinicalTrials.gov Identifier: NCT02538016

Recruitment Status : Completed

First Posted : September 2, 2015

Results First Posted : June 4, 2020

Last Update Posted : July 10, 2020

Sponsor:

Collaborator:

Otsuka America Pharmaceutical

Information provided by (Responsible Party):

Caleb Nelson, Boston Children's Hospital

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.

Condition or disease	Intervention/treatment	Phase
Cystinuria	Drug: Tolvaptan	Not Applicable

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	4 participants
Allocation:	N/A
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
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 Start Date :	October 2016
Actual Primary Completion Date :	December 2018
Actual Study Completion Date :	December 2018

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Kidney stones Cystinuria

MedlinePlus related topics: Urine and Urination

Drug Information available for: Tolvaptan

Genetic and Rare Diseases Information Center resources: Cystinuria

U.S. FDA Resources

Arms and Interventions

Arm	Intervention/treatment
Tolvaptan	Drug: Tolvaptan

Outcome Measures

Primary Outcome Measures :

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.

Secondary Outcome Measures :

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).

Eligibility Criteria

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Ages Eligible for Study:	12 Years to 29 Years (Child, Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02538016

Locations

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

Sponsors and Collaborators

Caleb Nelson

Otsuka America Pharmaceutical

Investigators

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Principal Investigator:	Caleb Nelson, MD, MPH	Boston Children's Hospital

Study Documents (Full-Text)

Documents provided by Caleb Nelson, Boston Children's Hospital:

Study Protocol and Statistical Analysis Plan [PDF] September 23, 2019

More Information

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Responsible Party:	Caleb Nelson, MD, MPH, Boston Children's Hospital
ClinicalTrials.gov Identifier:	NCT02538016
Other Study ID Numbers:	16726
First Posted:	September 2, 2015 Key Record Dates
Results First Posted:	June 4, 2020
Last Update Posted:	July 10, 2020
Last Verified:	May 2020

Keywords provided by Caleb Nelson, Boston Children's Hospital:


Cystinuria Kidney Stones

Additional relevant MeSH terms:

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Urolithiasis Urinary Calculi Cystinuria Urologic Diseases Calculi Pathological Conditions, Anatomical Renal Aminoacidurias Renal Tubular Transport, Inborn Errors Kidney Diseases	Metabolism, Inborn Errors Genetic Diseases, Inborn Metabolic Diseases Tolvaptan Antidiuretic Hormone Receptor Antagonists Molecular Mechanisms of Pharmacological Action Natriuretic Agents Physiological Effects of Drugs

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