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

Autosomal Dominant Polycystic Kidney Disease Clinical Trial

— 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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05190744
• Other study ID #: 21-005437
• Status: Recruiting
• Phase: Phase 2
• Start date: September 1, 2022
• Est. completion date: December 31, 2025

Study information

• Verified date: April 2024
• Source: Mayo Clinic
• Contact: Trinity Hooks
• Phone: 904-953-3057
• Email: Hooks.Trinity[@]mayo.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: December 31, 2025
• Est. primary completion date: September 30, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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 (Glomerular filtration rate) = 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.

Related Conditions & MeSH terms

• Autosomal Dominant Polycystic Kidney Disease
• Diabetes Insipidus
• Diabetes Insipidus, Nephrogenic
• Diabetes Mellitus
• Kidney Diseases
• Nephrogenic Diabetes Insipidus
• Polycystic Kidney Diseases
• Polycystic Kidney, Autosomal Dominant

Intervention

Drug:
• PB
500mg twice daily (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.

Locations

Country	Name	City	State
United States	Mayo Clinic	Jacksonville	Florida

Sponsors (2)

Lead Sponsor	Collaborator
Mayo Clinic	Hopital du Sacre-Coeur de Montreal

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Baseline, 90 days
Secondary	Change in urine output	Measured in milliliters per day (ml/day) by 24 hour urine collection	Baseline, day 15, day 45, day 75