Clinical trial NCT02584231: 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
Overall status: COMPLETED
Phase: PHASE4
Conditions: Monosymptomatic Nocturnal Enuresis
Keywords: Desmopressin
Paediatrics
urinary concentration test
Lead sponsor: University Hospital, Ghent
Sponsor class: OTHER
First posted: 2015-10-22
Enrollment: 25
Sex: ALL
Minimum age (years): 6
Maximum age (years): 8
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: DIAGNOSTIC

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


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

Primary outcomes:
measure: Desmopressine concentrations
timeFrame: 24 hours
Secondary outcomes:
measure: Efficacy - urinary volume.
timeFrame: 24 hours
measure: Efficacy - osmolality in urine.
timeFrame: 24 hours
measure: Urinary concentration test
timeFrame: 24 hours
measure: Safety of desmopressin in children as assessed by registration of adverse events.
timeFrame: 24 hours
measure: Safety of desmopressin in children as assessed by the measurement of natremia.
timeFrame: 24 hours
Other outcomes:
Locations:
city: Ghent
country: Belgium
facility: Ghent University Hospital - Department of Paediatric Nephrology
Study officials:
References:
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.