Trial Outcomes & Findings for Study With Two Different Doses of Desmopressin Orally Lyophilisate Tablet With Nocturnal Enuresis (NCT NCT01078753)
NCT ID: NCT01078753
Last Updated: 2011-11-02
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
89 participants
Primary outcome timeframe
Baseline (14-day period prior to starting study treatment) and Treatment Period II (weeks 3-4 after treatment initiation).
Results posted on
2011-11-02
Participant Flow
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.
Participant milestones
| Measure |
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.
|
|---|---|---|
|
Treatment Period I
STARTED
|
45
|
44
|
|
Treatment Period I
Full Analysis Set
|
44
|
44
|
|
Treatment Period I
COMPLETED
|
45
|
44
|
|
Treatment Period I
NOT COMPLETED
|
0
|
0
|
|
Treatment Period II
STARTED
|
45
|
44
|
|
Treatment Period II
Full Analysis Set
|
44
|
44
|
|
Treatment Period II
COMPLETED
|
45
|
44
|
|
Treatment Period II
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study With Two Different Doses of Desmopressin Orally Lyophilisate Tablet With Nocturnal Enuresis
Baseline characteristics by cohort
| Measure |
Desmopressin
n=45 Participants
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
n=44 Participants
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
n=89 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age Continuous
|
8.2 years
STANDARD_DEVIATION 1.6 • n=99 Participants
|
8.1 years
STANDARD_DEVIATION 1.4 • n=107 Participants
|
8.1 years
STANDARD_DEVIATION 1.5 • n=206 Participants
|
|
Age, Customized
6 to 9 years old
|
36 participants
n=99 Participants
|
36 participants
n=107 Participants
|
72 participants
n=206 Participants
|
|
Age, Customized
10 to15 years old
|
9 participants
n=99 Participants
|
8 participants
n=107 Participants
|
17 participants
n=206 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=99 Participants
|
21 Participants
n=107 Participants
|
38 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
28 Participants
n=99 Participants
|
23 Participants
n=107 Participants
|
51 Participants
n=206 Participants
|
|
Region of Enrollment
Japan
|
45 participants
n=99 Participants
|
44 participants
n=107 Participants
|
89 participants
n=206 Participants
|
|
Height
|
128.14 cm
STANDARD_DEVIATION 11.30 • n=99 Participants
|
127.68 cm
STANDARD_DEVIATION 8.61 • n=107 Participants
|
127.92 cm
STANDARD_DEVIATION 10.01 • n=206 Participants
|
|
Weight
|
27.59 kg
STANDARD_DEVIATION 8.40 • n=99 Participants
|
27.01 kg
STANDARD_DEVIATION 5.32 • n=107 Participants
|
27.30 kg
STANDARD_DEVIATION 7.01 • n=206 Participants
|
|
Body Mass Index (BMI)
|
16.46 kg/m˄2
STANDARD_DEVIATION 2.47 • n=99 Participants
|
16.43 kg/m˄2
STANDARD_DEVIATION 1.77 • n=107 Participants
|
16.44 kg/m˄2
STANDARD_DEVIATION 2.14 • n=206 Participants
|
|
Number of Wet Nights during Baseline
|
12.2 days
STANDARD_DEVIATION 1.5 • n=99 Participants
|
12.4 days
STANDARD_DEVIATION 1.5 • n=107 Participants
|
12.3 days
STANDARD_DEVIATION 1.5 • n=206 Participants
|
|
Number of Wet Nights during Baseline by stratification criteria
10 to 13 wet nights
|
32 participants
n=99 Participants
|
30 participants
n=107 Participants
|
62 participants
n=206 Participants
|
|
Number of Wet Nights during Baseline by stratification criteria
14 wet nights
|
13 participants
n=99 Participants
|
14 participants
n=107 Participants
|
27 participants
n=206 Participants
|
|
Prior treatment of Nocturnal Enuresis with Drugs
No prior treatment
|
45 participants
n=99 Participants
|
44 participants
n=107 Participants
|
89 participants
n=206 Participants
|
|
Prior treatment of Nocturnal Enuresis with Drugs
Prior treatment received
|
0 participants
n=99 Participants
|
0 participants
n=107 Participants
|
0 participants
n=206 Participants
|
|
Prior treatment of Nocturnal Enuresis with Therapy
No prior therapy
|
0 participants
n=99 Participants
|
0 participants
n=107 Participants
|
0 participants
n=206 Participants
|
|
Prior treatment of Nocturnal Enuresis with Therapy
Prior therapy
|
45 participants
n=99 Participants
|
44 participants
n=107 Participants
|
89 participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Baseline (14-day period prior to starting study treatment) and Treatment Period II (weeks 3-4 after treatment initiation).Population: 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.
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.
Outcome measures
| Measure |
Desmopressin
n=44 Participants
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
n=44 Participants
Participants received matching placebo tablets during treatment periods I and II according to the same efficacy criteria as participants in the Desmopressin treatment group.
|
|---|---|---|
|
Change in the Number of Wet Nights Between Baseline and Treatment Period II
|
3.264 wet nights
Interval 2.126 to 4.402
|
1.451 wet nights
Interval 0.333 to 2.57
|
SECONDARY outcome
Timeframe: Baseline (14-day period prior to starting study treatment) and Treatment Period I (weeks 1-2 after treatment initiation).Population: The Full analysis set.
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.
Outcome measures
| Measure |
Desmopressin
n=44 Participants
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
n=44 Participants
Participants received matching placebo tablets during treatment periods I and II according to the same efficacy criteria as participants in the Desmopressin treatment group.
|
|---|---|---|
|
Change in Number of Wet Nights Between Baseline and Treatment Period I
|
2.946 wet nights
Interval 1.815 to 4.078
|
1.317 wet nights
Interval 0.205 to 2.428
|
SECONDARY outcome
Timeframe: Treatment Period I (weeks 1-2) and Treatment Period II (weeks 3-4)Population: The full analysis set.
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.
Outcome measures
| Measure |
Desmopressin
n=44 Participants
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
n=44 Participants
Participants received matching placebo tablets during treatment periods I and II according to the same efficacy criteria as participants in the Desmopressin treatment group.
|
|---|---|---|
|
Change in Number of Wet Nights Between Treatment Periods I and II
|
0.318 wet nights
Interval -0.653 to 1.289
|
0.135 wet nights
Interval -0.819 to 1.088
|
Adverse Events
Desmopressin
Serious events: 0 serious events
Other events: 18 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 14 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Desmopressin
n=45 participants at risk
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
n=44 participants at risk
Participants received matching placebo tablets during treatment periods I and II according to the same efficacy criteria as participants in the Desmopressin treatment group.
|
|---|---|---|
|
General disorders
Asthenia
|
4.4%
2/45 • 4 weeks (periods I + II)
|
0.00%
0/44 • 4 weeks (periods I + II)
|
|
Infections and infestations
Empyema
|
0.00%
0/45 • 4 weeks (periods I + II)
|
2.3%
1/44 • 4 weeks (periods I + II)
|
|
Infections and infestations
Nasopharyngitis
|
13.3%
6/45 • 4 weeks (periods I + II)
|
13.6%
6/44 • 4 weeks (periods I + II)
|
|
Infections and infestations
Tonsillitis
|
2.2%
1/45 • 4 weeks (periods I + II)
|
0.00%
0/44 • 4 weeks (periods I + II)
|
|
Infections and infestations
Upper respiratory tract infection
|
2.2%
1/45 • 4 weeks (periods I + II)
|
0.00%
0/44 • 4 weeks (periods I + II)
|
|
Nervous system disorders
Headache
|
2.2%
1/45 • 4 weeks (periods I + II)
|
0.00%
0/44 • 4 weeks (periods I + II)
|
|
Eye disorders
Blepharitis
|
2.2%
1/45 • 4 weeks (periods I + II)
|
0.00%
0/44 • 4 weeks (periods I + II)
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
0.00%
0/45 • 4 weeks (periods I + II)
|
2.3%
1/44 • 4 weeks (periods I + II)
|
|
Respiratory, thoracic and mediastinal disorders
Tonsillar hypertrophy
|
2.2%
1/45 • 4 weeks (periods I + II)
|
0.00%
0/44 • 4 weeks (periods I + II)
|
|
Gastrointestinal disorders
Abdominal pain
|
2.2%
1/45 • 4 weeks (periods I + II)
|
0.00%
0/44 • 4 weeks (periods I + II)
|
|
Gastrointestinal disorders
Dental caries
|
0.00%
0/45 • 4 weeks (periods I + II)
|
2.3%
1/44 • 4 weeks (periods I + II)
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/45 • 4 weeks (periods I + II)
|
2.3%
1/44 • 4 weeks (periods I + II)
|
|
Gastrointestinal disorders
Enterocolitis
|
2.2%
1/45 • 4 weeks (periods I + II)
|
0.00%
0/44 • 4 weeks (periods I + II)
|
|
Gastrointestinal disorders
Lip ulceration
|
0.00%
0/45 • 4 weeks (periods I + II)
|
2.3%
1/44 • 4 weeks (periods I + II)
|
|
Gastrointestinal disorders
Stomatitis
|
4.4%
2/45 • 4 weeks (periods I + II)
|
2.3%
1/44 • 4 weeks (periods I + II)
|
|
Skin and subcutaneous tissue disorders
Dermatitis atopic
|
2.2%
1/45 • 4 weeks (periods I + II)
|
0.00%
0/44 • 4 weeks (periods I + II)
|
|
Skin and subcutaneous tissue disorders
Dyshidrosis
|
2.2%
1/45 • 4 weeks (periods I + II)
|
0.00%
0/44 • 4 weeks (periods I + II)
|
|
Skin and subcutaneous tissue disorders
Skin papilloma
|
2.2%
1/45 • 4 weeks (periods I + II)
|
0.00%
0/44 • 4 weeks (periods I + II)
|
|
General disorders
Feeling hot
|
0.00%
0/45 • 4 weeks (periods I + II)
|
2.3%
1/44 • 4 weeks (periods I + II)
|
|
General disorders
Malaise
|
2.2%
1/45 • 4 weeks (periods I + II)
|
0.00%
0/44 • 4 weeks (periods I + II)
|
|
Investigations
Blood calcium decreased
|
0.00%
0/45 • 4 weeks (periods I + II)
|
2.3%
1/44 • 4 weeks (periods I + II)
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
2.2%
1/45 • 4 weeks (periods I + II)
|
0.00%
0/44 • 4 weeks (periods I + II)
|
|
Injury, poisoning and procedural complications
Wound
|
0.00%
0/45 • 4 weeks (periods I + II)
|
2.3%
1/44 • 4 weeks (periods I + II)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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
- Publication restrictions are in place
Restriction type: OTHER