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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
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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

Drug Information available for: Tolvaptan

Arms and Interventions
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Arm Intervention/treatment
Tolvaptan Drug: Tolvaptan


Outcome Measures
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Primary Outcome Measures :
  1. 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 :
  1. 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
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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
Genetic Diseases, Inborn
Tolvaptan
Antidiuretic Hormone Receptor Antagonists
Molecular Mechanisms of Pharmacological Action
Natriuretic Agents
Physiological Effects of Drugs