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PB to Treat Hereditary Nephrogenic Diabetes Insipidus, ADPKD Treated With Tolvaptan, and Severely Polyuric Patients With Previous Lithium Administration (SerendipityPB1)

Primary Purpose

Autosomal Dominant Polycystic Kidney Disease, Nephrogenic Diabetes Insipidus, Acquired Nephrogenic Diabetes Insipidus

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
PB
Sponsored by
Mayo Clinic
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional supportive care trial for Autosomal Dominant Polycystic Kidney Disease focused on measuring Tolvaptan, ADPKD, Polycystic Kidney Disease, NDI, Nephrogenic diabetes insipidus, Inherited nephrogenic diabetes insipidus, Diabetes Insipidus, Lithium, Lithium-induced nephrogenic diabetes insipidus, Vasopressin receptor antagonist, Tolvaptan-induced aquaresis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Diagnosis of nephrogenic diabetes insipidus (NDI) (congenital, tolvaptan-induced, or lithium-induced).
  • Morning Uosm < 300 mOsm/kg H2O.
  • Participating in tolvaptan arm.
  • Males for NDI.
  • Autosomal Dominant Polycystic Kidney Disease (ADPKD).
  • Lithium-induced NDI.
  • GFR ≥ 30 ml/min.
  • If hypertensive, blood pressure controlled on antihypertensives (< 130/80 mm Hg) at least 30 days before day 1.
  • Capable of providing consent.
  • Capable of providing urine samples as dictated by the protocol.

Exclusion Criteria:

  • History of acute gout attack in the past 30 days.
  • Uncontrolled hyperuricemia or active gout.
  • Known urinary retention, urinary incontinence or bladder dysfunction.
  • Other significant chronic medical disease (heart failure, diabetes mellitus, liver disease, transient or persistent elevated transaminases.
  • History of hepatotoxicity related to tolvaptan.
  • Allergy to interventional drug (PB).
  • History of persistent hyponatremia.

Sites / Locations

  • Mayo ClinicRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

Polyuric subjects with Hereditary Nephrogenic Diabetes Insipidus

Polyuric subjects with Autosomal Dominant Polycystic Kidney Disease treated with Tolvaptan

Polyuric subject secondary to lithium administration

Arm Description

Polyuric subjects with hereditary nephrogenic diabetes insipidus with loss of function of AVPR2 or AQP2 will be treated with PB

Polyuric subjects with autosomal dominant polycystic kidney disease on chronic tolvaptan treatment will be treated with PB

Polyuric subject post lithium administration will receive PB

Outcomes

Primary Outcome Measures

Change in urine osmolality
Measured in milliosmoles per kilogram of water (mOsm/kg) from a urine specimen and is a measure of the concentration of osmotically active particles, principally sodium, chloride, potassium, and urea

Secondary Outcome Measures

Change in urine output
Measured in milliliters per day (ml/day) by 24 hour urine collection

Full Information

First Posted
December 29, 2021
Last Updated
January 18, 2023
Sponsor
Mayo Clinic
Collaborators
Hopital du Sacre-Coeur de Montreal
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1. Study Identification

Unique Protocol Identification Number
NCT05190744
Brief Title
PB to Treat Hereditary Nephrogenic Diabetes Insipidus, ADPKD Treated With Tolvaptan, and Severely Polyuric Patients With Previous Lithium Administration
Acronym
SerendipityPB1
Official Title
A Multi-center, Open-Label, Exploratory Study to Assess the Efficacy of PB in Decreasing the Urine Output and Increasing the Urine Osmolality in Patients With Hereditary Nephrogenic Diabetes Insipidus, Patients With Autosomal Dominant Polycystic Kidney Disease Treated With Tolvaptan, And Severely Polyuric Patients With Previous Lithium Administration (Serendipity-PB1)
Study Type
Interventional

2. Study Status

Record Verification Date
January 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 1, 2022 (Actual)
Primary Completion Date
January 2024 (Anticipated)
Study Completion Date
January 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Mayo Clinic
Collaborators
Hopital du Sacre-Coeur de Montreal

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this research is to study the effectiveness and safety of the medication PB in slowing the frequent urination related to tolvaptan as long-term treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD), or frequent urination related to inherited nephrogenic diabetes insipidus as an inherited condition or as an acquired condition from prior treatment with lithium.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autosomal Dominant Polycystic Kidney Disease, Nephrogenic Diabetes Insipidus, Acquired Nephrogenic Diabetes Insipidus, Congenital Nephrogenic Diabetes Insipidus
Keywords
Tolvaptan, ADPKD, Polycystic Kidney Disease, NDI, Nephrogenic diabetes insipidus, Inherited nephrogenic diabetes insipidus, Diabetes Insipidus, Lithium, Lithium-induced nephrogenic diabetes insipidus, Vasopressin receptor antagonist, Tolvaptan-induced aquaresis

7. Study Design

Primary Purpose
Supportive Care
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Polyuric subjects with Hereditary Nephrogenic Diabetes Insipidus
Arm Type
Experimental
Arm Description
Polyuric subjects with hereditary nephrogenic diabetes insipidus with loss of function of AVPR2 or AQP2 will be treated with PB
Arm Title
Polyuric subjects with Autosomal Dominant Polycystic Kidney Disease treated with Tolvaptan
Arm Type
Experimental
Arm Description
Polyuric subjects with autosomal dominant polycystic kidney disease on chronic tolvaptan treatment will be treated with PB
Arm Title
Polyuric subject secondary to lithium administration
Arm Type
Experimental
Arm Description
Polyuric subject post lithium administration will receive PB
Intervention Type
Drug
Intervention Name(s)
PB
Intervention Description
500mg BID followed by 1000mg BID. The dose of PB inducing the maximal increase in urine osmolality will be continued for up to three months providing that no side effects are observed including clinical and laboratory surveillance.
Primary Outcome Measure Information:
Title
Change in urine osmolality
Description
Measured in milliosmoles per kilogram of water (mOsm/kg) from a urine specimen and is a measure of the concentration of osmotically active particles, principally sodium, chloride, potassium, and urea
Time Frame
Baseline, 90 days
Secondary Outcome Measure Information:
Title
Change in urine output
Description
Measured in milliliters per day (ml/day) by 24 hour urine collection
Time Frame
Baseline, day 15, day 45, day 75

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Diagnosis of nephrogenic diabetes insipidus (NDI) (congenital, tolvaptan-induced, or lithium-induced). Morning Uosm < 300 mOsm/kg H2O. Participating in tolvaptan arm. Males for NDI. Autosomal Dominant Polycystic Kidney Disease (ADPKD). Lithium-induced NDI. GFR ≥ 30 ml/min. If hypertensive, blood pressure controlled on antihypertensives (< 130/80 mm Hg) at least 30 days before day 1. Capable of providing consent. Capable of providing urine samples as dictated by the protocol. Exclusion Criteria: History of acute gout attack in the past 30 days. Uncontrolled hyperuricemia or active gout. Known urinary retention, urinary incontinence or bladder dysfunction. Other significant chronic medical disease (heart failure, diabetes mellitus, liver disease, transient or persistent elevated transaminases. History of hepatotoxicity related to tolvaptan. Allergy to interventional drug (PB). History of persistent hyponatremia.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Cameron King
Phone
904-953-4254
Email
King.Cameron@mayo.edu
First Name & Middle Initial & Last Name or Official Title & Degree
Trinity Hooks
Phone
904-953-3057
Email
Hooks.Trinity@mayo.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Fouad Chebib, MD
Organizational Affiliation
Mayo Clinic
Official's Role
Principal Investigator
Facility Information:
Facility Name
Mayo Clinic
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32224
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Clinical Studies Unit
Phone
904-953-4254
Email
King.Cameron@mayo.edu
First Name & Middle Initial & Last Name & Degree
Trinity Hooks
Phone
904-953-3057
Email
Hooks.Trinity@mayo.edu
First Name & Middle Initial & Last Name & Degree
Fouad Chebib, MD

12. IPD Sharing Statement

Links:
URL
https://www.mayo.edu/research/clinical-trials
Description
Mayo Clinic Clinical Trials

Learn more about this trial

PB to Treat Hereditary Nephrogenic Diabetes Insipidus, ADPKD Treated With Tolvaptan, and Severely Polyuric Patients With Previous Lithium Administration

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