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

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ClinicalTrials.gov Identifier: NCT01078753
Recruitment Status : Completed
First Posted : March 2, 2010
Results First Posted : November 2, 2011
Last Update Posted : November 2, 2011
Sponsor:
Information provided by (Responsible Party):
Ferring Pharmaceuticals

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Nocturnal Enuresis
Interventions Drug: Desmopressin
Drug: Placebo
Enrollment 89
Recruitment Details  
Pre-assignment Details

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.

Arm/Group Title Desmopressin Placebo
Hide Arm/Group Description 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. Participants received matching placebo tablets during treatment periods I and II according to the same efficacy criteria as participants in the Desmopressin treatment group.
Period Title: Treatment Period I
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 Title: Treatment Period II
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.
Arm/Group Title Desmopressin Placebo Total
Hide Arm/Group Description 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. 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 of all reporting groups
Overall Number of Baseline Participants 45 44 89
Hide Baseline Analysis Population Description
[Not Specified]
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 45 participants 44 participants 89 participants
8.2  (1.6) 8.1  (1.4) 8.1  (1.5)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 45 participants 44 participants 89 participants
6 to 9 years old 36 36 72
10 to15 years old 9 8 17
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants 44 participants 89 participants
Female
17
  37.8%
21
  47.7%
38
  42.7%
Male
28
  62.2%
23
  52.3%
51
  57.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Japan Number Analyzed 45 participants 44 participants 89 participants
45 44 89
Height  
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 45 participants 44 participants 89 participants
128.14  (11.30) 127.68  (8.61) 127.92  (10.01)
Weight  
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 45 participants 44 participants 89 participants
27.59  (8.40) 27.01  (5.32) 27.30  (7.01)
Body Mass Index (BMI)  
Mean (Standard Deviation)
Unit of measure:  Kg/m˄2
Number Analyzed 45 participants 44 participants 89 participants
16.46  (2.47) 16.43  (1.77) 16.44  (2.14)
Number of Wet Nights during Baseline   [1] 
Mean (Standard Deviation)
Unit of measure:  Days
Number Analyzed 45 participants 44 participants 89 participants
12.2  (1.5) 12.4  (1.5) 12.3  (1.5)
[1]
Measure Description: Number of Wet Nights recorded during the 14-day Baseline period.
Number of Wet Nights during Baseline by stratification criteria   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 45 participants 44 participants 89 participants
10 to 13 wet nights 32 30 62
14 wet nights 13 14 27
[1]
Measure Description: The number of wet nights recorded during the 14-day Baseline period is reported for each of the stratification categories.
Prior treatment of Nocturnal Enuresis with Drugs  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 45 participants 44 participants 89 participants
No prior treatment 45 44 89
Prior treatment received 0 0 0
Prior treatment of Nocturnal Enuresis with Therapy   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 45 participants 44 participants 89 participants
No prior therapy 0 0 0
Prior therapy 45 44 89
[1]
Measure Description: Therapies for treatment of Nocturnal Enuresis might include life-skill guidance, bladder control training, psychotherapy, or behaviour conditioning therapy.
1.Primary Outcome
Title Change in the Number of Wet Nights Between Baseline and Treatment Period II
Hide Description The number of wet nights was recorded by participants (or their caregivers) in a daily diary. The difference was calculated from the number of wet nights during the 14-day Baseline Period minus the number of wet nights during the 14-day Treatment Period II.
Time Frame Baseline (14-day period prior to starting study treatment) and Treatment Period II (weeks 3-4 after treatment initiation).
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The Full Analysis Set (FAS) included all participants who received at least one dose of study treatment, satisfied all the major eligibility criteria and for whom efficacy data was obtained.
Arm/Group Title Desmopressin Placebo
Hide Arm/Group Description:
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.
Participants received matching placebo tablets during treatment periods I and II according to the same efficacy criteria as participants in the Desmopressin treatment group.
Overall Number of Participants Analyzed 44 44
Least Squares Mean (95% Confidence Interval)
Unit of Measure: wet nights
3.264
(2.126 to 4.402)
1.451
(0.333 to 2.570)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Desmopressin, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.009
Comments Desmopressin was considered to be superior to Placebo if the p-value for the comparison was < 0.05 and the reduction from Baseline in number of wet nights was larger in the FE992026 group than in the Placebo group.
Method ANCOVA
Comments Factors in the analysis were Gender (male, female), age (6≤x≤9, 10≤x≤15), number of wet nights at Baseline (10≤x≤13, x=14), and treatment group.
Method of Estimation Estimation Parameter Least Squares Mean Difference
Estimated Value 1.813
Confidence Interval (2-Sided) 95%
0.462 to 3.163
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Change in Number of Wet Nights Between Baseline and Treatment Period I
Hide Description The number of wet nights was recorded by participants (or their caregivers) in a daily diary. The difference was calculated from the number of wet nights during the 14-day Baseline Period minus the number of wet nights during the 14-day Treatment Period I.
Time Frame Baseline (14-day period prior to starting study treatment) and Treatment Period I (weeks 1-2 after treatment initiation).
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The Full analysis set.
Arm/Group Title Desmopressin Placebo
Hide Arm/Group Description:
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.
Participants received matching placebo tablets during treatment periods I and II according to the same efficacy criteria as participants in the Desmopressin treatment group.
Overall Number of Participants Analyzed 44 44
Least Squares Mean (95% Confidence Interval)
Unit of Measure: wet nights
2.946
(1.815 to 4.078)
1.317
(0.205 to 2.428)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Desmopressin, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.018
Comments [Not Specified]
Method ANCOVA
Comments Factors in the analysis were Gender (male, female), age (6≤x≤9, 10≤x≤15), number of wet nights at Baseline (10≤x≤13, x=14), and treatment group.
Method of Estimation Estimation Parameter Least Squares mean Difference
Estimated Value 1.629
Confidence Interval (2-Sided) 95%
0.287 to 2.972
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Change in Number of Wet Nights Between Treatment Periods I and II
Hide Description The number of wet nights was recorded by participants (or their caregivers) in a daily diary. The difference was calculated from the number of wet nights during the 14-day Treatment Period I minus the number of wet nights during the 14-day Treatment Period II.
Time Frame Treatment Period I (weeks 1-2) and Treatment Period II (weeks 3-4)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The full analysis set.
Arm/Group Title Desmopressin Placebo
Hide Arm/Group Description:
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.
Participants received matching placebo tablets during treatment periods I and II according to the same efficacy criteria as participants in the Desmopressin treatment group.
Overall Number of Participants Analyzed 44 44
Least Squares Mean (95% Confidence Interval)
Unit of Measure: wet nights
0.318
(-0.653 to 1.289)
0.135
(-0.819 to 1.088)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Desmopressin, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.752
Comments [Not Specified]
Method ANCOVA
Comments Factors in the analysis included Gender (male, female), age (6≤x≤9, 10≤x≤15), number of wet nights at Baseline (10≤x≤13, x=14), and treatment group.
Method of Estimation Estimation Parameter Least Squares Mean Difference
Estimated Value 0.183
Confidence Interval (2-Sided) 95%
-0.968 to 1.335
Estimation Comments [Not Specified]
Time Frame 4 weeks (periods I + II)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Desmopressin Placebo
Hide Arm/Group Description 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. Participants received matching placebo tablets during treatment periods I and II according to the same efficacy criteria as participants in the Desmopressin treatment group.
All-Cause Mortality
Desmopressin Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Desmopressin Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/45 (0.00%)   0/44 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Desmopressin Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   18/45 (40.00%)   14/44 (31.82%) 
Eye disorders     
Blepharitis  1  1/45 (2.22%)  0/44 (0.00%) 
Gastrointestinal disorders     
Abdominal pain  1  1/45 (2.22%)  0/44 (0.00%) 
Dental caries  1  0/45 (0.00%)  1/44 (2.27%) 
Diarrhoea  1  0/45 (0.00%)  1/44 (2.27%) 
Enterocolitis  1  1/45 (2.22%)  0/44 (0.00%) 
Lip ulceration  1  0/45 (0.00%)  1/44 (2.27%) 
Stomatitis  1  2/45 (4.44%)  1/44 (2.27%) 
General disorders     
Asthenia  1  2/45 (4.44%)  0/44 (0.00%) 
Feeling hot  1  0/45 (0.00%)  1/44 (2.27%) 
Malaise  1  1/45 (2.22%)  0/44 (0.00%) 
Infections and infestations     
Empyema  1  0/45 (0.00%)  1/44 (2.27%) 
Nasopharyngitis  1  6/45 (13.33%)  6/44 (13.64%) 
Tonsillitis  1  1/45 (2.22%)  0/44 (0.00%) 
Upper respiratory tract infection  1  1/45 (2.22%)  0/44 (0.00%) 
Injury, poisoning and procedural complications     
Arthropod bite  1  1/45 (2.22%)  0/44 (0.00%) 
Wound  1  0/45 (0.00%)  1/44 (2.27%) 
Investigations     
Blood calcium decreased  1  0/45 (0.00%)  1/44 (2.27%) 
Nervous system disorders     
Headache  1  1/45 (2.22%)  0/44 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Rhinitis allergic  1  0/45 (0.00%)  1/44 (2.27%) 
Tonsillar hypertrophy  1  1/45 (2.22%)  0/44 (0.00%) 
Skin and subcutaneous tissue disorders     
Dermatitis atopic  1  1/45 (2.22%)  0/44 (0.00%) 
Dyshidrosis  1  1/45 (2.22%)  0/44 (0.00%) 
Skin papilloma  1  1/45 (2.22%)  0/44 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA version 13.1
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 only disclosure restriction on the PI is that the sponsor can review the draft manuscript prior to publication and can request delay of publication where any contents are deemed patentable by the sponsor or confidential to the sponsor. Comments will be given within four weeks from receipt of the draft manuscript. Additional time may be required to allow Ferring to seek patent protection of the invention
Results Point of Contact
Name/Title: Clinical Development Support
Organization: Ferring Pharmaceuticals
Responsible Party: Ferring Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01078753     History of Changes
Other Study ID Numbers: FE992026 CS35
First Submitted: March 1, 2010
First Posted: March 2, 2010
Results First Submitted: September 27, 2011
Results First Posted: November 2, 2011
Last Update Posted: November 2, 2011