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

2024-517438-16-00 Therapeutic exploratory (Phase II) Ended

Start 12 Sep 2024 · End 19 Feb 2026 · Status Ended · 1 EU/EEA countries · 1 sites

Overview

Sponsor-declared trial summary

Phase Therapeutic exploratory (Phase II)
Status Ended
Participants planned 10
Countries 1
Sites 1

Nephrogenic diabetes insipidus

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 mutati…

Key facts

Sponsor
Odense University Hospital
Participant type
Patients
Age range
18-64 years, 65+ years
Gender
Male
Therapeutic area
Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trial duration
12 Sep 2024 → 19 Feb 2026
Decision date (initial)
2024-09-12
Transition trial
Yes
Low-intervention
No
Rare-disease indication
Yes
Vulnerable population
No

External identifiers

EU CT number
2024-517438-16-00
EudraCT number
2020-002204-38

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

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.

Conditions and MedDRA coding

Nephrogenic diabetes insipidus

VersionLevelCodeTermSystem organ class
20.0 PT 10068304 Congenital nephrogenic diabetes insipidus 100000004850

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

  1. Male
  2. Age > 18 year
  3. Mentally capable of understanding the test
  4. Nephrogenic diabetes insipidus diagnosed by genetic defect in AVPR2

Exclusion criteria 4

  1. Other known relevant disease including malignity
  2. Contraindications to fluconazole
  3. Prolonged QT interval on baseline ECG
  4. Concurrent treatment with drugs metabolized by CYP3A4, CYP2C19 or CYP2C9

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. Spontaneous urinary volume and urinary concentration ability (osmolality).

Secondary endpoints 1

  1. Plasma osmolality, abundance of AQP2 in urinary exosomes, plasma vasopressin/copeptin concentrations, plasma electrolytes, micturition frequency

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Clotrimazole

SCP1086356 · ATC

Active substance
Clotrimazole
Route of administration
ORAL
Max daily dose
400 mg milligram(s)
Max total dose
400
Max treatment duration
3 Week(s)
Authorisation status
Authorised
ATC code
J02AC01 — FLUCONAZOLE
Marketing authorisation
-
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Odense University Hospital

Sponsor organisation
Odense University Hospital
Address
J B Winsloews Vej 4
City
Odense C
Postcode
5000
Country
Denmark

Scientific contact point

Organisation
Odense University Hospital
Contact name
Gitte Rye Hinrichs

Public contact point

Organisation
Odense University Hospital
Contact name
Gitte Rye Hinrichs

Third parties 1

OrganisationCity, countryDuties
Odense University Hospital
ORG-100007716
Odense C, Denmark On site monitoring, E-data capture, Code 8

Locations

1 EU/EEA country · 1 investigational sites

By country

CountryMS statusPlanned subjectsSites
Denmark Ended 10 1
Rest of world 0

Investigational sites

Denmark

1 site · Ended
Odense University Hospital
Department of Nephrology, J B Winsloews Vej 4, 5000, Odense C

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Country Trial startTrial end Recruitment startRecruitment end Early termination
Denmark 2024-09-12

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 6 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

TypeTitleVersion
Protocol (for publication) Protocol 6
Recruitment arrangements (for publication) Recruitement procedures 2
Subject information and informed consent form (for publication) Informed concent form 1
Subject information and informed consent form (for publication) Subject Information version 4 1
Summary of Product Characteristics (SmPC) (for publication) SMPC_produktresume_fluconazol 1
Synopsis of the protocol (for publication) Protokolresume 1

Application history

2 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2024-08-23 Denmark Acceptable
2024-09-12
2024-09-12
2 SUBSTANTIAL MODIFICATION SM-2 2025-11-11 Denmark Acceptable
2025-12-08
2025-12-09