Clinical trial NCT00209261: A 6-week Open Label Cross-over Study With 2 Different Daily Doses of Minirin® Oral Lyophilisate in Children and Adolescents With Primary Nocturnal Enuresis (PNE)
Official title: A 6-week Open Label Cross-over Study With 2 Different Daily Doses of Minirin® Oral Lyophilisate (120 μg and 240 μg) and 2 Different Daily Doses of Minirin® Tablet (0.2 mg and 2 x 0.2 mg) in Children and Adolescents With Primary Nocturnal Enuresis (PNE)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Primary Nocturnal Enuresis
Lead sponsor: Ferring Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2005-09-21
Enrollment: 221
Sex: ALL
Minimum age (years): 5
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: To evaluate the preference of subjects for Minirin® oral lyophilisate treatment compared with Minirin® tablet treatment after 6 weeks.

To compare efficacy of the 2 formulations at the end of the 6-week treatment period using diary card data.

To compare ease of use of both formulations at 3 and 6 weeks using a VAS-scale. To validate a PNE Quality of Life (QoL) questionnaire. To evaluate safety. To compare compliance with the 2 formulations

Detailed description: To evaluate the preference of subjects for Minirin® oral lyophilisate treatment compared with Minirin® tablet treatment after 6 weeks.

To compare efficacy of the 2 formulations at the end of the 6-week treatment period using diary card data.

To compare ease of use of both formulations at 3 and 6 weeks using a VAS-scale. To validate a PNE Quality of Life (QoL) questionnaire. To evaluate safety. To compare compliance with the 2 formulations

Eligibility criteria: Inclusion Criteria:

* Children suffering from primary nocturnal enuresis with no organic pathology.
* Children of either sex, not below 5 and not above 15 years of age (not below 6 years in The Netherlands and France).
* Children with a minimum of 6 wet nights in 2 weeks.

Exclusion Criteria:

* Children who have previously been treated with desmopressin or other medications for nocturnal enuresis or enuresis alarms.
* Children receiving substances that are known or suspected to potentiate antidiuretic hormone, e.g. SSRI, tricyclic antidepressant drugs, chlorpromazine and carbamazepine.
* Diagnosed renal diabetes insipidus or central diabetes insipidus with an AVP (arginine vasopressin) deficiency.
* Proven urinary tract infection within the past month or a documented positive urine culture at the start of the study

Primary outcomes:
measure: The primary endpoint will be the difference in the proportion of subjects who preferred each formulation at the end of the 6-week treatment period.
timeFrame: 6 weeks (melt) and 6 weeks (tablet). Total 12 weeks.
Secondary outcomes:
measure: Efficacy will be measured as the difference in the incidence of bedwetting episodes during each 3-week treatment period, compared between formulations.
timeFrame: 3 weeks
measure: The subjects will be asked to rate the ease of use of each formulation using a 100 mm Visual Analogue Scale (VAS), with 0 = I find it very easy to use this medicine and 100 = I find it very difficult to use this medicine.
timeFrame: 3 weeks
measure: Subjects will be asked to complete a QoL questionnaire at visit 2 and visit 3.
timeFrame: 3 weeks
measure: Safety comparison between the 2 formulations.
timeFrame: 6 weeks
measure: Comparison of compliance between the 2 formulations.
timeFrame: 6 weeks
measure: The subjects will be asked a question regarding which treatment (Minirin® oral lyophilisate or Minirin® tablet) they preferred at the end of 6 weeks treatment.
timeFrame: 6 weeks (melt) and 6 weeks (tablet). Total 12 weeks.
Other outcomes:
Locations:
city: Paris
country: France
facility: Service de Chirurrgie Viscerale Pediatrique Hospital Necker-Enfants Malades
Study officials:
affiliation: Ferring Pharmaceuticals
name: Clinical Development Support
role: STUDY_DIRECTOR
References:
citation: Caldwell PH, Codarini M, Stewart F, Hahn D, Sureshkumar P. Alarm interventions for nocturnal enuresis in children. Cochrane Database Syst Rev. 2020 May 4;5(5):CD002911. doi: 10.1002/14651858.CD002911.pub3.
citation: Juul KV, Van Herzeele C, De Bruyne P, Goble S, Walle JV, Norgaard JP. Desmopressin melt improves response and compliance compared with tablet in treatment of primary monosymptomatic nocturnal enuresis. Eur J Pediatr. 2013 Sep;172(9):1235-42. doi: 10.1007/s00431-013-1992-9. Epub 2013 May 16.