Treatment of the genetic disease nephrogenic diabetes insipidus with the antifungal drug Fluconazole

Treatment of congenital nephrogenic diabetes insipidus with fluconazole, an antifungal medication

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2020-002204-38-DK

Enrollment

Registered

2020-05-07

Start date

2020-11-24

Completion date

Unknown

Last updated

2024-09-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Nephrogenic diabetes insipidus MedDRA version: 21.0 Level: LLT Classification code 10012600 Term: Diabetes insipidus nephrogenic System Organ Class: 100000004857

Interventions

Pharmaceutical Form: Capsule, hard INN or Proposed INN: Fluconazole Other descriptive name: FLUCONAZOLE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 200-

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: - Male - Age > 18 year - Mentally capable of understanding the test - Nephrogenic diabetes insipidus diagnosed by genetic defect in AVPR2 Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 9 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1

Exclusion criteria

Exclusion criteria: - Other known relevant disease including malignity - Contraindications to fluconazole - Prolonged QT interval on baseline ECG - Concurrent treatment with drugs metabolized by CYP3A4, CYP2C19 or CYP2C9

Design outcomes

Primary

Measure	Time frame
Main Objective: The overall aim is to improve quality of life and relieve of the severe impairment associated with NDI in patients. This is achieved by testing if the antifungal drug fluconazole can reduce diuresis and improve the urinary concentration ability in patients with nephrogenic diabetes insipidus due to an AVPR2 gene mutation. ;Secondary Objective: Not applicable;Primary end point(s): 1. Diuresis (mL/day): Evaluated weekly by 24hour urine samples 2. Urinary concentration ability evaluated by a water deprivation test before and after the intervention ;Timepoint(s) of evaluation of this end point: 1. Weekly throughout the intervention 2. Before and after the intervention	—

Measure

Time frame

Main Objective: The overall aim is to improve quality of life and relieve of the severe impairment associated with NDI in patients. This is achieved by testing if the antifungal drug fluconazole can reduce diuresis and improve the urinary concentration ability in patients with nephrogenic diabetes insipidus due to an AVPR2 gene mutation. ;Secondary Objective: Not applicable;Primary end point(s): 1. Diuresis (mL/day): Evaluated weekly by 24hour urine samples 2. Urinary concentration ability evaluated by a water deprivation test before and after the intervention ;Timepoint(s) of evaluation of this end point: 1. Weekly throughout the intervention 2. Before and after the intervention

—

Secondary

Measure	Time frame
Secondary end point(s): Secondary outcomes: 1. Urinary osmolality 2. Plasma osmolality (mmol/kg): Evaluated by weekly blood samples 3. Plasma electrolytes 4. Micturition frequency 5. Abundance of AQP2 in urinary exosomes: Spot urine samples before and after the intervention 6. Plasma vasopressin and copeptin concentrations;Timepoint(s) of evaluation of this end point: 1. Weekly throughout the intervention 2. Weekly throughout the intervention 3. Weekly throughout the intervention 4. Daily throughout the intervention 5. Before and after the intervention 6. Weekly throughout the intervention	—

Measure

Time frame

Secondary end point(s): Secondary outcomes: 1. Urinary osmolality 2. Plasma osmolality (mmol/kg): Evaluated by weekly blood samples 3. Plasma electrolytes 4. Micturition frequency 5. Abundance of AQP2 in urinary exosomes: Spot urine samples before and after the intervention 6. Plasma vasopressin and copeptin concentrations;Timepoint(s) of evaluation of this end point: 1. Weekly throughout the intervention 2. Weekly throughout the intervention 3. Weekly throughout the intervention 4. Daily throughout the intervention 5. Before and after the intervention 6. Weekly throughout the intervention

—

Countries

Denmark

Contacts

Public ContactDepartment of Nephrology

Odense University Hospital

line.mortensen@rsyd.dk

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026