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

NCT02584231 | Completed Phase 4

• 2 other identifiers: EC/2015/06162014-005200-13
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

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

Conditions

Monosymptomatic Nocturnal Enuresis

Keywords

Desmopressin; Paediatrics; urinary concentration test

Outcome Measures

Primary Outcomes (1)

• Desmopressine concentrations

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

  24 hours

Secondary Outcomes (5)

• Efficacy - urinary volume.

  24 hours

• Efficacy - osmolality in urine.

  24 hours

• Urinary concentration test

  24 hours

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

  24 hours

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

  24 hours

Study Arms (2)

Patients needing an urinary concentration test

EXPERIMENTAL

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

Drug: desmopressin

Patients suffering from treatment resistant nocturnal enuresis

EXPERIMENTAL

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

Drug: desmopressin

Interventions

desmopressin DRUG

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

Also known as: dDAVP

Patients needing an urinary concentration test; Patients suffering from treatment resistant nocturnal enuresis

Eligibility Criteria

• Age: 6 Months - 8 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University Ghent: lead

Study Sites (1)

Ghent University Hospital - Department of Paediatric Nephrology

Ghent, 9000, Belgium

Location

Related Publications (1)

• 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.

  PMID: 31347012 DERIVED

MeSH Terms

Interventions

Deamino Arginine Vasopressin

Intervention Hierarchy (Ancestors)

Arginine Vasopressin; Vasopressins; Pituitary Hormones, Posterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Neuropeptides; Peptides; Amino Acids, Peptides, and Proteins; Oligopeptides; Nerve Tissue Proteins; Proteins

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof Johan Vande Walle

Study Record Dates

• First Submitted: September 10, 2015
• First Posted: October 22, 2015
• Study Start: September 9, 2015
• Primary Completion: March 19, 2018
• Study Completion: February 1, 2019
• Last Updated: February 6, 2019

Record last verified: 2019-02

Locations

BE (1)