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

NCT ID: NCT02584231

Last Updated: 2019-02-06

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 25 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-09-09
• Study Completion Date: 2019-02-01

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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: DIAGNOSTIC
• Blinding Strategy: NONE

Study Groups

Patients needing an urinary concentration test

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

Group Type: EXPERIMENTAL

desmopressin

Intervention Type: 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

Patients suffering from treatment resistant nocturnal enuresis

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

Group Type: EXPERIMENTAL

desmopressin

Intervention Type: 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

Interventions

desmopressin

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

Intervention Type: DRUG

Other Intervention Names

dDAVP

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

• Minimum Eligible Age: 6 Months
• Maximum Eligible Age: 8 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University Ghent

OTHER

Sponsor Role: lead

Responsible Party

Pediatrie

Prof Johan Vande Walle

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Ghent University Hospital - Department of Paediatric Nephrology

Ghent, , Belgium

Countries

Belgium

References

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.

Reference Type: DERIVED

PMID: 31347012 (View on PubMed)

Other Identifiers

2014-005200-13

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

EC/2015/0616

Identifier Type: -

Identifier Source: org_study_id