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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

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

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

NCT ID

NCT05190744

First Posted

December 29, 2021

Last Updated

January 18, 2023

Sponsor

Mayo Clinic

Collaborators

Hopital du Sacre-Coeur de Montreal

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

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)

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

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

King.Cameron@mayo.edu

First Name & Middle Initial & Last Name or Official Title & Degree

Trinity Hooks

Phone

904-953-3057

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

King.Cameron@mayo.edu

First Name & Middle Initial & Last Name & Degree

Trinity Hooks

Phone

904-953-3057

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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