Ongoing Clinical Trials for Congenital Nephrogenic Diabetes Insipidus
This article provides information about 1 ongoing clinical trial for Congenital Nephrogenic Diabetes Insipidus, a rare condition where the kidneys cannot properly concentrate urine, leading to excessive urination and constant thirst. The trial is exploring a new potential treatment approach using fluconazole, a medication typically used for fungal infections.
Clinical trial locations
Study of Fluconazole Treatment for Patients with Congenital Nephrogenic Diabetes Insipidus
This clinical trial is investigating whether fluconazole, a medication commonly used to treat fungal infections, can help reduce excessive urination and improve the body’s ability to concentrate urine in people with Congenital Nephrogenic Diabetes Insipidus. This condition occurs when the kidneys fail to respond to a hormone called vasopressin that normally helps the body retain water, leading to the production of large amounts of dilute urine and persistent thirst.
Inclusion criteria: This trial is open to male participants who are over 18 years old and have a confirmed diagnosis through genetic testing showing a mutation in the AVPR2 gene, which causes the body not to respond properly to the hormone that controls water balance. Participants must be able to understand and follow study instructions and be mentally capable of providing informed consent.
Exclusion criteria: The study excludes male patients younger than 18 or older than 65 years, as well as all female patients. People who do not have a confirmed AVPR2 gene mutation cannot participate. Those currently taking medications that could interact with fluconazole, or who have severe liver or kidney problems unrelated to the condition, are not eligible. Patients with allergies to fluconazole or similar antifungal medications, those unable to provide informed consent, anyone participating in other clinical trials, or those with uncontrolled heart conditions are also excluded.
Main focus and goals: The study aims to determine whether fluconazole can improve quality of life and reduce the severe symptoms experienced by people with this condition. During the trial, researchers will monitor how much urine patients produce naturally and how well their bodies can concentrate this urine. Additional measurements include blood tests to check salt levels and other substances, tracking urination frequency, analyzing urine samples for specific proteins, and measuring blood hormone levels. The treatment period lasts for 3 days, during which patients receive fluconazole taken by mouth at doses up to 400 milligrams per day.
Investigational drug: Fluconazole is being studied for a new potential use in treating this condition. While it is well-known as an antifungal medication, this trial explores whether it can help the kidneys better concentrate urine and reduce the excessive urination that characterizes the condition. This novel application could potentially offer a new treatment option for people living with this rare genetic disorder.
Summary
Currently, there is one clinical trial available for patients with Congenital Nephrogenic Diabetes Insipidus, taking place in Denmark. This trial represents an innovative approach to treating this rare condition by repurposing fluconazole, an antifungal medication, for a completely different therapeutic purpose. The study specifically focuses on patients with the AVPR2 gene mutation and is limited to male participants over 18 years of age. The trial involves a relatively short treatment period of 3 days, with careful monitoring of urine production and concentration. This research could potentially provide a new treatment option for a condition that currently has limited therapeutic choices, offering hope to patients who struggle with the challenging symptoms of excessive urination and constant thirst.
