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

Phase 2

Recruiting

• Conditions: Acquired Nephrogenic Diabetes Insipidus; Congenital Nephrogenic Diabetes Insipidus; Autosomal Dominant Polycystic Kidney Disease; Nephrogenic Diabetes Insipidus
• Interventions: Drug: PB

• Registration Number: NCT05190744
• Lead Sponsor: Mayo Clinic

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-12-29
• Study First Submitted QC: 2021-12-29
• Study First Posted: 2022-01-13
• Study Start: 2022-09-01
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-18
• Last Update Posted: 2024-04-22
• Primary Completion: 2025-09-30
• Study Completion: 2025-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 20

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Polyuric subjects with Autosomal Dominant Polycystic Kidney Disease treated with Tolvaptan	PB	Polyuric subjects with autosomal dominant polycystic kidney disease on chronic tolvaptan treatment will be treated with PB
Polyuric subject secondary to lithium administration	PB	Polyuric subject post lithium administration will receive PB
Polyuric subjects with Hereditary Nephrogenic Diabetes Insipidus	PB	Polyuric subjects with hereditary nephrogenic diabetes insipidus with loss of function of arginine vasopressin receptor 2 (AVPR2) or aquaporin 2 (AQP2) will be treated with PB

Primary Outcome Measures

Name	Time	Method
Change in urine osmolality	Baseline, 90 days	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

Name	Time	Method
Change in urine output	Baseline, day 15, day 45, day 75	Measured in milliliters per day (ml/day) by 24 hour urine collection

Trial Locations

Locations (1)

Mayo Clinic; 🇺🇸 Jacksonville, Florida, United States