We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05190744
Recruitment Status : Recruiting
First Posted : January 13, 2022
Last Update Posted : January 19, 2023
Sponsor:
Collaborator:
Hopital du Sacre-Coeur de Montreal
Information provided by (Responsible Party):
Fouad T. Chebib, 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.

Condition or disease Intervention/treatment Phase
Autosomal Dominant Polycystic Kidney Disease Nephrogenic Diabetes Insipidus Acquired Nephrogenic Diabetes Insipidus Congenital Nephrogenic Diabetes Insipidus Drug: PB Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
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)
Actual Study Start Date : September 1, 2022
Estimated Primary Completion Date : January 2024
Estimated Study Completion Date : January 2024


Arm Intervention/treatment
Experimental: Polyuric subjects with Hereditary Nephrogenic Diabetes Insipidus
Polyuric subjects with hereditary nephrogenic diabetes insipidus with loss of function of AVPR2 or AQP2 will be treated with PB
Drug: PB
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.

Experimental: Polyuric subjects with Autosomal Dominant Polycystic Kidney Disease treated with Tolvaptan
Polyuric subjects with autosomal dominant polycystic kidney disease on chronic tolvaptan treatment will be treated with PB
Drug: PB
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.

Experimental: Polyuric subject secondary to lithium administration
Polyuric subject post lithium administration will receive PB
Drug: PB
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 Measures :
  1. Change in urine osmolality [ Time Frame: 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 :
  1. Change in urine output [ Time Frame: Baseline, day 15, day 45, day 75 ]
    Measured in milliliters per day (ml/day) by 24 hour urine collection



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

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): NCT05190744


Contacts
Layout table for location contacts
Contact: Cameron King 904-953-4254 King.Cameron@mayo.edu
Contact: Trinity Hooks 904-953-3057 Hooks.Trinity@mayo.edu

Locations
Layout table for location information
United States, Florida
Mayo Clinic Recruiting
Jacksonville, Florida, United States, 32224
Contact: Clinical Studies Unit    904-953-4254    King.Cameron@mayo.edu   
Contact: Trinity Hooks    904-953-3057    Hooks.Trinity@mayo.edu   
Principal Investigator: Fouad Chebib, MD         
Sponsors and Collaborators
Mayo Clinic
Hopital du Sacre-Coeur de Montreal
Investigators
Layout table for investigator information
Principal Investigator: Fouad Chebib, MD Mayo Clinic
Additional Information:
Layout table for additonal information
Responsible Party: Fouad T. Chebib, Principal Investigator, Mayo Clinic
ClinicalTrials.gov Identifier: NCT05190744    
Other Study ID Numbers: 21-005437
First Posted: January 13, 2022    Key Record Dates
Last Update Posted: January 19, 2023
Last Verified: January 2023

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Fouad T. Chebib, Mayo Clinic:
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
Additional relevant MeSH terms:
Layout table for MeSH terms
Kidney Diseases
Polycystic Kidney Diseases
Polycystic Kidney, Autosomal Dominant
Diabetes Insipidus
Diabetes Insipidus, Nephrogenic
Diabetes Mellitus
Glucose Metabolism Disorders
Metabolic Diseases
Endocrine System Diseases
Urologic Diseases
Congenital Abnormalities
Kidney Diseases, Cystic
Abnormalities, Multiple
Ciliopathies
Genetic Diseases, Inborn
Pituitary Diseases