Monosymptomatic Nocturnal EnuresisPhase 4NCT02584231
Pharmacokinetics and Pharmacodynamics of Desmopressin Oral Lyophilisate Formulation in the Paediatric Population
A Phase 4 study testing desmopressin in people with Monosymptomatic Nocturnal Enuresis, run by University Ghent at 1 site in Belgium.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 months to 8 years
- Conditions the study is for
- Monosymptomatic Nocturnal Enuresis
- What is being tested?
- desmopressin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteBelgium
- Study dates
- 9 Sep 2015to 1 Feb 2019
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 8 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
Belgium1 site
Ghent University Hospital - Department of Paediatric Nephrology
Ghent, 9000
About this study
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
- Being tested
- desmopressin
- Keywords
- Desmopressin, Paediatrics, urinary concentration test
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
desmopressinDrug
Also called dDAVP
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
| Study group | Role | What they receive |
|---|---|---|
| Patients needing an urinary concentration test | Experimental | Patients who need a urinary concentration test because of uro- or nephropathy (age: 6 months - 8 year) Receives: Drug: desmopressin |
| Patients suffering from treatment resistant nocturnal enuresis | Experimental | Patients suffering from treatment resistant nocturnal enuresis (age: 5 - 8 year) Receives: Drug: desmopressin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Desmopressine concentrations
The assessed pharmacokinetic parameters are desmopressine concentrations at timepoints 0, 1/4, 1/2, 1, 2, 3, 5, 6 and 7h.
Measured: 24 hours
Secondary outcomes (5)
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.
Measured: 24 hours
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.
Measured: 24 hours
Urinary concentration test
Evaluation of the oral lyophilisate formulation of desmopressin for the urinary concentration test. Assessed parameter is urinary concentration capacity.
Measured: 24 hours
Safety of desmopressin in children as assessed by registration of adverse events.
Registration of adverse events
Measured: 24 hours
Safety of desmopressin in children as assessed by the measurement of natremia.
Measurement of natremia
Measured: 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2015 (estimated)
- Study start9 Sep 2015
- Primary completion19 Mar 2018
- Study completion1 Feb 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Monosymptomatic Nocturnal EnuresisStudies of the same condition, open and under way.
- More trials run by University GhentThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Ghent
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Pediatrie, University Hospital, Ghent
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02584231
- Sponsor's protocol ID
- EC/2015/0616
- Other IDs
- 2014-005200-13, 2014
- Status last verified
- Feb 2019
- First submitted
- 10 Sep 2015
- Last updated
- 6 Feb 2019
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 31347012
Study at a glance
From the registry record, last updated by the sponsor on 6 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pharmacokinetics and Pharmacodynamics of Desmopressin Oral Lyophilisate Formulation in the Paediatric Population
- ClinicalTrials.gov ID
- NCT02584231
- Acronym / sponsor ID
- EC/2015/0616
- Conditions
- Monosymptomatic Nocturnal Enuresis
- Keywords
- Desmopressin, Paediatrics, urinary concentration test
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- University GhentUniversity, hospital or other
- Study start
- 9 Sep 2015 (actual)
- Primary completion
- 19 Mar 2018 (actual)
- Study completion
- 1 Feb 2019 (actual)
- First posted
- 22 Oct 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients needing an urinary concentration test | Experimental | Drug: desmopressinPatients who need a urinary concentration test because of uro- or nephropathy (age: 6 months - 8 year) |
| Patients suffering from treatment resistant nocturnal enuresis | Experimental | Drug: desmopressinPatients suffering from treatment resistant nocturnal enuresis (age: 5 - 8 year) |
Who may qualify
- Ages
- 6 months to 8 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (10 listed)
- Diabetes insipidus
- Renal failure (eGFR<60ml/min/1,73m²)
- Current urinary tract infection
- Syndrome of inappropriate antidiuretic hormone secretion
- Heart failure
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| desmopressinAlso: dDAVPOne 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 | Drug | Patients needing an urinary concentration test, Patients suffering from treatment resistant nocturnal enuresis |
Primary outcome
Desmopressine concentrations
Time frame: 24 hours
The assessed pharmacokinetic parameters are desmopressine concentrations at timepoints 0, 1/4, 1/2, 1, 2, 3, 5, 6 and 7h.
5 secondary outcomes
Efficacy - urinary volume.
Time frame: 24 hours
Efficacy - osmolality in urine.
Time frame: 24 hours
Urinary concentration test
Time frame: 24 hours
Safety of desmopressin in children as assessed by registration of adverse events.
Time frame: 24 hours
Safety of desmopressin in children as assessed by the measurement of natremia.
Time frame: 24 hours
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Feb 2019.
- Derived from this study: PubMed 31347012Michelet 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing desmopressin in people with Monosymptomatic Nocturnal Enuresis, run by University Ghent at 1 site in Belgium.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Desmopressine concentrations (time frame: 24 hours). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02584231, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Feb 2019.