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Pharmacokinetics and Pharmacodynamics of Desmopressin Oral Lyophilisate Formulation in the Paediatric Population

Primary Purpose

Monosymptomatic Nocturnal Enuresis

Status

Completed

Phase

Phase 4

Locations

Belgium

Study Type

Interventional

Intervention

desmopressin

About this trial

This is an interventional diagnostic trial for Monosymptomatic Nocturnal Enuresis focused on measuring Desmopressin, Paediatrics, urinary concentration test

Eligibility Criteria

6 Months - 8 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Children with an uro- and/or nephropathy who need an urinary concentration test OR Children with monosymptomatic nocturnal enuresis (based on nocturnal polyuria) with treatment failure on desmopressin tablet
Otherwise healthy children (on medical history and physical examination)
Parents or legal guardian of the child signed the informed consent form
Age: between 6 months and 8 years
Minimum weight: 8 kg

Exclusion Criteria:

Diabetes insipidus
Renal failure (eGFR<60ml/min/1,73m²)
Current urinary tract infection
Syndrome of inappropriate antidiuretic hormone secretion
Heart failure
Clinical significant medical conditions (renal, hepatic, gastro-intestinal, pulmonary, cardiac, endocrinologic) that might interfere with the clinical endpoints
Sensitivity to desmopressin or excipients of the oral lyophilisate formulation
Use of antibiotics, diuretics or other drugs that can influence diuresis (tricyclic antidepressants, chlorpropamide, oxcarbazepine, selective serotonin reuptake inhibitors, chlorpromazine and carbamazepine).
Use of drugs that influence intestinal motility (such as loperamide)
Anomalies of the mouth that might interfere with the intake / absorption of the medication

Sites / Locations

Ghent University Hospital - Department of Paediatric Nephrology

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Patients needing an urinary concentration test

Patients suffering from treatment resistant nocturnal enuresis

Arm Description

Patients who need a urinary concentration test because of uro- or nephropathy (age: 6 months - 8 year)

Patients suffering from treatment resistant nocturnal enuresis (age: 5 - 8 year)

Outcomes

Primary Outcome Measures

Desmopressine concentrations

The assessed pharmacokinetic parameters are desmopressine concentrations at timepoints 0, 1/4, 1/2, 1, 2, 3, 5, 6 and 7h.

Secondary Outcome Measures

Efficacy - urinary volume.

PD of desmopressin in children with nocturnal enuresis that is resistant to treatment with desmopressin tablet. First PD parameter is antidiuretic effect. This is urinary volume per hour.

Efficacy - osmolality in urine.

PD of desmopressin in children with nocturnal enuresis that is resistant to treatment with desmopressin tablet. Second PD parameter is urinary concentration capacity. This is osmolality in urine.

Urinary concentration test

Evaluation of the oral lyophilisate formulation of desmopressin for the urinary concentration test. Assessed parameter is urinary concentration capacity.

Safety of desmopressin in children as assessed by registration of adverse events.

Registration of adverse events

Safety of desmopressin in children as assessed by the measurement of natremia.

Measurement of natremia

Full Information

NCT ID

NCT02584231

First Posted

September 10, 2015

Last Updated

February 4, 2019

Sponsor

University Ghent

1. Study Identification

Unique Protocol Identification Number

NCT02584231

Brief Title

Pharmacokinetics and Pharmacodynamics of Desmopressin Oral Lyophilisate Formulation in the Paediatric Population

Official Title

Pharmacokinetics and Pharmacodynamics of Desmopressin Oral Lyophilisate Formulation in the Paediatric Population

Study Type

Interventional

2. Study Status

Record Verification Date

February 2019

Overall Recruitment Status

Completed

Study Start Date

September 9, 2015 (Actual)

Primary Completion Date

March 19, 2018 (Actual)

Study Completion Date

February 1, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

University Ghent

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

Patients suffering from nocturnal enuresis (starting from the age of 5 till adulthood) are all treated with the same dose of desmopressin, i.e. 120mcg once daily. In treatment resistant enuresis, this dose is doubled: those patients take 240mcg once daily. A pilot study performed at our department showed a correlation between weight and plasma concentration when a fixed dose of desmopressin oral lyophilisate formulation was given to the pediatric patient (older than 6 years). This study will investigate the pharmacokinetics (PK) and pharmacodynamics (PD) of desmopressin in young children, less than 8 years old. Additionally, the efficacy of desmopressin oral lyophilisate formulation in urinary concentration testing will be evaluated

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Monosymptomatic Nocturnal Enuresis

Keywords

Desmopressin, Paediatrics, urinary concentration test

7. Study Design

Primary Purpose

Diagnostic

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

25 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Patients needing an urinary concentration test

Arm Type

Experimental

Arm Description

Patients who need a urinary concentration test because of uro- or nephropathy (age: 6 months - 8 year)

Arm Title

Patients suffering from treatment resistant nocturnal enuresis

Arm Type

Experimental

Arm Description

Patients suffering from treatment resistant nocturnal enuresis (age: 5 - 8 year)

Intervention Type

Drug

Intervention Name(s)

desmopressin

Other Intervention Name(s)

dDAVP

Intervention Description

One time dosing of desmopressin oral lyophilisate formulation. The dose is age-dependant: >6 months and < 2years: 60µg; ≥2 years and <4 years: 120µg PO and ≥4 years and <8 years: 240 µg PO. There will be blood sampling and urine sampling for PK and PD/safety

Primary Outcome Measure Information:

Title

Desmopressine concentrations

Description

The assessed pharmacokinetic parameters are desmopressine concentrations at timepoints 0, 1/4, 1/2, 1, 2, 3, 5, 6 and 7h.

Time Frame

24 hours

Secondary Outcome Measure Information:

Title

Efficacy - urinary volume.

Description

PD of desmopressin in children with nocturnal enuresis that is resistant to treatment with desmopressin tablet. First PD parameter is antidiuretic effect. This is urinary volume per hour.

Time Frame

24 hours

Title

Efficacy - osmolality in urine.

Description

PD of desmopressin in children with nocturnal enuresis that is resistant to treatment with desmopressin tablet. Second PD parameter is urinary concentration capacity. This is osmolality in urine.

Time Frame

24 hours

Title

Urinary concentration test

Description

Evaluation of the oral lyophilisate formulation of desmopressin for the urinary concentration test. Assessed parameter is urinary concentration capacity.

Time Frame

24 hours

Title

Safety of desmopressin in children as assessed by registration of adverse events.

Description

Registration of adverse events

Time Frame

24 hours

Title

Safety of desmopressin in children as assessed by the measurement of natremia.

Description

Measurement of natremia

Time Frame

24 hours

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Months

Maximum Age & Unit of Time

8 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Children with an uro- and/or nephropathy who need an urinary concentration test OR Children with monosymptomatic nocturnal enuresis (based on nocturnal polyuria) with treatment failure on desmopressin tablet Otherwise healthy children (on medical history and physical examination) Parents or legal guardian of the child signed the informed consent form Age: between 6 months and 8 years Minimum weight: 8 kg Exclusion Criteria: Diabetes insipidus Renal failure (eGFR<60ml/min/1,73m²) Current urinary tract infection Syndrome of inappropriate antidiuretic hormone secretion Heart failure Clinical significant medical conditions (renal, hepatic, gastro-intestinal, pulmonary, cardiac, endocrinologic) that might interfere with the clinical endpoints Sensitivity to desmopressin or excipients of the oral lyophilisate formulation Use of antibiotics, diuretics or other drugs that can influence diuresis (tricyclic antidepressants, chlorpropamide, oxcarbazepine, selective serotonin reuptake inhibitors, chlorpromazine and carbamazepine). Use of drugs that influence intestinal motility (such as loperamide) Anomalies of the mouth that might interfere with the intake / absorption of the medication

Facility Information:

Facility Name

Ghent University Hospital - Department of Paediatric Nephrology

City

Ghent

ZIP/Postal Code

9000

Country

Belgium

12. IPD Sharing Statement

Citations:

PubMed Identifier

31347012

Citation

Michelet R, Dossche L, Van Herzeele C, De Bruyne P, Gasthuys E, Van Bocxlaer J, Vande Walle J, Vermeulen A. An Integrated Paediatric Population PK/PD Analysis of dDAVP: How do PK Differences Translate to Clinical Outcomes? Clin Pharmacokinet. 2020 Jan;59(1):81-96. doi: 10.1007/s40262-019-00798-6.

Results Reference

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Pharmacokinetics and Pharmacodynamics of Desmopressin Oral Lyophilisate Formulation in the Paediatric Population

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