Study With Two Different Doses of Desmopressin Orally Lyophilisate Tablet With Nocturnal Enuresis (PNE)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Ferring Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01078753
First received: March 1, 2010
Last updated: September 27, 2011
Last verified: September 2011
Results First Received: September 27, 2011  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Investigator);   Primary Purpose: Treatment
Condition: Nocturnal Enuresis
Interventions: Drug: Desmopressin
Drug: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment

174 subjects consented and entered the 14-day screening period during which Baseline characteristics regarding enuretic episodes were established. Eligible patients who met the criterion were randomized 1:1 to receive either desmopressin or matching placebo.

Randomization was stratified by gender, age and number of wet nights at Baseline.


Reporting Groups
  Description
Desmopressin During treatment period I participants received 120 μg per day desmopressin oral lyophilisate tablet for 14 days. Participants for whom treatment was effective (a reduction of ≥ 75% from Baseline in the number of wet nights), and who showed no problems with tolerability, continued to receive the same treatment for a further 14 days in treatment period II. Participants for whom efficacy was inadequate (a reduction of <75% from Baseline in the number of wet nights), but who showed no tolerability problems, received an increased dose of desmopressin oral lyophilisate tablet 240 µg for 14 days in treatment period II.
Placebo Participants received matching placebo tablets during treatment periods I and II according to the same efficacy criteria as participants in the Desmopressin treatment group.

Participant Flow for 2 periods

Period 1:   Treatment Period I
    Desmopressin     Placebo  
STARTED     45     44  
Full Analysis Set     44 [1]   44  
COMPLETED     45     44  
NOT COMPLETED     0     0  
[1] One patient was excluded from the full analysis set due to failure to satisfy the entry criteria.

Period 2:   Treatment Period II
    Desmopressin     Placebo  
STARTED     45 [1]   44  
Full Analysis Set     44 [2]   44  
COMPLETED     45     44  
NOT COMPLETED     0     0  
[1] Desmopressin 120 µg - 4 participants Desmopressin 240 µg - 41 participants
[2] One patient was excluded from the full analysis set due to failure to satisfy the entry criteria.



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Desmopressin During treatment period I participants received 120 μg per day desmopressin oral lyophilisate tablet for 14 days. Participants for whom treatment was effective (a reduction of ≥ 75% from Baseline in the number of wet nights), and who showed no problems with tolerability, continued to receive the same treatment for a further 14 days in treatment period II. Participants for whom efficacy was inadequate (a reduction of <75% from Baseline in the number of wet nights), but who showed no tolerability problems, received an increased dose of desmopressin oral lyophilisate tablet 240 µg for 14 days in treatment period II.
Placebo Participants received matching placebo tablets during treatment periods I and II according to the same efficacy criteria as participants in the Desmopressin treatment group.
Total Total of all reporting groups

Baseline Measures
    Desmopressin     Placebo     Total  
Number of Participants  
[units: participants]
  45     44     89  
Age  
[units: years]
Mean ± Standard Deviation
  8.2  ± 1.6     8.1  ± 1.4     8.1  ± 1.5  
Age, Customized  
[units: participants]
     
6 to 9 years old     36     36     72  
10 to15 years old     9     8     17  
Gender  
[units: participants]
     
Female     17     21     38  
Male     28     23     51  
Region of Enrollment  
[units: participants]
     
Japan     45     44     89  
Height  
[units: cm]
Mean ± Standard Deviation
  128.14  ± 11.30     127.68  ± 8.61     127.92  ± 10.01  
Weight  
[units: kg]
Mean ± Standard Deviation
  27.59  ± 8.40     27.01  ± 5.32     27.30  ± 7.01  
Body Mass Index (BMI)  
[units: kg/m˄2]
Mean ± Standard Deviation
  16.46  ± 2.47     16.43  ± 1.77     16.44  ± 2.14  
Number of Wet Nights during Baseline [1]
[units: days]
Mean ± Standard Deviation
  12.2  ± 1.5     12.4  ± 1.5     12.3  ± 1.5  
Number of Wet Nights during Baseline by stratification criteria [2]
[units: participants]
     
10 to 13 wet nights     32     30     62  
14 wet nights     13     14     27  
Prior treatment of Nocturnal Enuresis with Drugs  
[units: participants]
     
No prior treatment     45     44     89  
Prior treatment received     0     0     0  
Prior treatment of Nocturnal Enuresis with Therapy [3]
[units: participants]
     
No prior therapy     0     0     0  
Prior therapy     45     44     89  
[1] Number of Wet Nights recorded during the 14-day Baseline period.
[2] The number of wet nights recorded during the 14-day Baseline period is reported for each of the stratification categories.
[3] Therapies for treatment of Nocturnal Enuresis might include life-skill guidance, bladder control training, psychotherapy, or behaviour conditioning therapy.



  Outcome Measures
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1.  Primary:   Change in the Number of Wet Nights Between Baseline and Treatment Period II   [ Time Frame: Baseline (14-day period prior to starting study treatment) and Treatment Period II (weeks 3-4 after treatment initiation). ]

2.  Secondary:   Change in Number of Wet Nights Between Baseline and Treatment Period I   [ Time Frame: Baseline (14-day period prior to starting study treatment) and Treatment Period I (weeks 1-2 after treatment initiation). ]

3.  Secondary:   Change in Number of Wet Nights Between Treatment Periods I and II   [ Time Frame: Treatment Period I (weeks 1-2) and Treatment Period II (weeks 3-4) ]


  Serious Adverse Events


  Other Adverse Events


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Limitations and Caveats
Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.  


Results Point of Contact:  
Name/Title: Clinical Development Support
Organization: Ferring Pharmaceuticals
e-mail: DK0-Disclosure@ferring.com


No publications provided


Responsible Party: Ferring Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01078753     History of Changes
Other Study ID Numbers: FE992026 CS35
Study First Received: March 1, 2010
Results First Received: September 27, 2011
Last Updated: September 27, 2011
Health Authority: Japan: Pharmaceuticals and Medical Devices Agency