Efficacy and Safety of Desmopressin Melt for the Treatmen... | NCT00477490 | Trialant
NCT00477490
Sponsor
Ferring Pharmaceuticals
Status
Completed
Last Update Posted
Nov 2, 2015Estimated
Enrollment
799Actual
Phase
Phase 3
Conditions
Nocturia
Interventions
desmopressin acetate
Placebo
Countries
United States
Canada
Protocol Section
Identification Module
NCT ID
NCT00477490
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
FE992026 CS29
Secondary IDs
Not provided
Brief Title
Efficacy and Safety of Desmopressin Melt for the Treatment of Nocturia
Official Title
A Randomized, Double Blind, Placebo Controlled, Parallel Group, Multi-Center Study With a Double Blind Extension Investigating the Efficacy and Safety of a Fast- Dissolving ("Melt") Formulation of Desmopressin for the Treatment of Nocturia in Adults
Acronym
Not provided
Organization
Ferring PharmaceuticalsINDUSTRY
Status Module
Record Verification Date
Sep 2015
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
May 2007
Primary Completion Date
Feb 2008Actual
Completion Date
Feb 2008Actual
First Submitted Date
May 22, 2007
First Submission Date that Met QC Criteria
May 22, 2007
First Posted Date
May 23, 2007Estimated
Results Waived
Not provided
Results First Submitted Date
Jun 16, 2015
Results First Submitted that Met QC Criteria
Sep 29, 2015
Results First Posted Date
Nov 2, 2015Estimated
Certification/Extension (aka Delayed Results) First Submitted Date
Jun 2, 2009
Certification/Extension First Submitted that Passed QC Review
Jun 5, 2009
Certification/Extension First Posted Date
Sep 30, 2009Estimated
Last Update Submitted Date
Sep 29, 2015
Last Update Posted Date
Nov 2, 2015Estimated
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Ferring PharmaceuticalsINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Not provided
Is FDA Regulated Device
Not provided
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
The purpose of this study is to investigate the efficacy and safety of several doses of the melt formulation of desmopressin in a broad population of adult patients with nocturia.
Detailed Description
Not provided
Conditions Module
Conditions
Nocturia
Keywords
Not provided
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 3
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
799Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Placebo
Placebo Comparator
Participants took a placebo 'melt' for 28 days to complete part 1 of the study. In part 2, placebo patients were randomized to one of the other 4 treatment arms based on assignments predetermined at the initial randomization, to receive active desmopressin melt for between 1 and 6 months (until the database for part 1 was locked and treatment was unblinded).
Drug: Placebo
desmopressin melt 10 μg
Experimental
Participants took desmopressin melt 10 μg for 28 days to complete part 1 of the study. Participants continued on this dose in part 2 of the study for between 1 and 6 months (until the database for part 1 was locked and treatment was unblinded).
Drug: desmopressin acetate
desmopressin melt 25 μg
Experimental
Participants took desmopressin melt 25 μg for 28 days to complete part 1 of the study. Participants continued on this dose in part 2 of the study for between 1 and 6 months (until the database for part 1 was locked and treatment was unblinded).
Drug: desmopressin acetate
desmopressin melt 50 μg
Experimental
Participants took desmopressin melt 50 μg for 28 days to complete part 1 of the study. Participants continued on this dose in part 2 of the study for between 1 and 6 months (until the database for part 1 was locked and treatment was unblinded).
Drug: desmopressin acetate
Interventions
Name
Type
Description
Arm Group Labels
Other Names
desmopressin acetate
Drug
Oral lyophilisate of desmopressin acetate placed under the participant's tongue, without water, once daily approximately 1 hour before bedtime in the assigned dosage: 10, 25, 50 or 100 μg
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Part I: Change From Baseline in Mean Number of Nocturnal Voids at Week 4
The number of nocturnal voids was the average over 3 consecutive 24-hours periods prior to Day 1 and prior to the week 4 visit as recorded in participant diaries.
This was the first co-primary outcome.
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
Part I: Percentage of Participants With Greater Than 33 Percent Reduction From Baseline in Mean Number of Nocturnal Voids at Week 4
Percentage of participants in each treatment arm that had a greater than 33% reduction from baseline to the end of Part I (week 4) in mean number of nocturnal voids. Nocturnal void data were recorded in participant diaries.
This was the second co-primary outcome.
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
Secondary Outcomes
Measure
Description
Time Frame
Part II: Change From Baseline in Mean Number of Nocturnal Voids to Days 29, 57, 113 and 169
Part II outcomes tested the durability of the effect observed in Part I. The number of nocturnal voids was the average over 3 consecutive 24-hours periods prior to Part I baseline and prior to the Part II visit as recorded in participant diaries.
- Week 3 to Day 1 (Baseline), Days 29, 57, 113 and 169
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Inclusion Criteria
Written informed consent prior to the performance of any study-related activity.
Patients 18 years and older with an average of ≥ 2 nocturnal voids per night as determined by a 3 day frequency-volume chart during the screening period.
Exclusion Criteria:
Males:
Clinical suspicion of bladder outlet obstruction and/or urine flow < 5 ml/s. If medical history and/or physical examination suggest bladder outlet obstruction, uroflowmetry should be performed to confirm the diagnosis
Surgical treatment for bladder outlet obstruction/benign prostatic hyperplasia performed within the past 6 months
Females:
Pregnancy. Females of reproductive age must have documentation of a reliable method of contraception.
Use of pessary for pelvic prolapse.
Unexplained pelvic mass.
Males and Females:
Clinical suspicion of urinary retention and/or post void residual volume > 150 ml. If medical history and/or physical examination suggest urinary retention, bladder ultrasound or catheterization should be performed to confirm the diagnosis.
Current or past urologic malignancy (e.g., bladder cancer, prostate cancer).
Clinical evidence of current genitourinary tract pathology that could interfere with voiding.
History of neurogenic detrusor activity (previously known as detrusor hyperreflexia).
Suspicion or evidence of cardiac failure.
Uncontrolled hypertension.
Uncontrolled diabetes mellitus.
Renal insufficiency. Serum creatinine must be within normal limits and estimated glomerular filtration rate (eGFR) >=60 mL/min.
Active hepatic and/or biliary disease. Aspartate transaminase (AST) or alanine transaminase (ALT) should not be >2 times the upper limit of normal. Total bilirubin should not be > 1.5 mg/dL.
Hyponatremia. Serum sodium level must be within normal limits
Syndrome of Inappropriate antidiuretic hormone secretion (SIADH).
Diabetes insipidus (urine output > 40 ml/kg over 24 hours) as determined by the 3-day voiding diary.
Psychogenic or habitual polydipsia
Obstructive sleep apnea
Other
Known alcohol or substance abuse
Work or lifestyle potentially interfering with regular nighttime sleep (e.g., shift workers)
Previous desmopressin treatment for nocturia.
Any other medical condition, laboratory abnormality, psychiatric condition, mental incapacity or language barrier that, in the judgment of the investigator, could impair patient participation in the trial.
Use of loop diuretics (furosemide, torsemide, ethacrynic acid). Other classes of diuretics (thiazides, triamterene, chlorthalidone, amiloride, indapamide) were permitted, either as monotherapy or combination therapy. Subjects using a diuretic were to be encouraged to take it in the morning, if medically feasible.
Use of any other investigational drug within 30 days of screening.
Concomitant Medications
The following medications are permitted provided that the subject has been on a stable dose for the 3 months prior to the screening date (i.e. treatment has not been initiated or discontinued and there has been no change in dose):
Juul KV, Klein BM, Norgaard JP. Long-term durability of the response to desmopressin in female and male nocturia patients. Neurourol Urodyn. 2013 Apr;32(4):363-70. doi: 10.1002/nau.22306. Epub 2012 Sep 12.
Weiss JP, Zinner NR, Klein BM, Norgaard JP. Desmopressin orally disintegrating tablet effectively reduces nocturia: results of a randomized, double-blind, placebo-controlled trial. Neurourol Urodyn. 2012 Apr;31(4):441-7. doi: 10.1002/nau.22243. Epub 2012 Mar 22.
See Also Links
Not provided
Available IPD Information
Not provided
IPD Sharing Statement Module
No data available
No data is available for this block.
Results Section
Participant Flow Module
Pre-assignment Details
Potential subjects were given a sleep/voiding diary and urine collection device to record the time and volume of each void for 3 consecutive 24-hour periods.
Randomization was stratified by age (<65, ≥65 years) and by the absence/presence of nocturnal polyuria, defined as a ratio of nighttime urine volume/24-hour urine volume ≥33%.
Recruitment Details
Eighty-eight (88) sites were initiated in the United States and Canada, and 81 of these sites screened subjects; 78 sites enrolled and randomized subjects.
A total of 1412 subjects were screened for Part I of the study; 613 subjects were screening failures.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Placebo
Participants took a placebo 'melt' for 28 days to complete Part I of the study. In Part II, placebo patients were randomized to one of the other four treatment arms to receive active desmopressin melt for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
FG001
Periods
Title
Milestones
Reasons Not Completed
Part I: 4 Week Efficacy
Type
Comment
Milestone Data
STARTED
Baseline Characteristics Module
Baseline Analysis Population Description
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
5
More Info Module
Limitations and Caveats
Not provided
Annotation Section
No data available
No data is available for this block.
Document Section
No data available
No data is available for this block.
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Not provided
Submission Tracking
No data available
No data is available for this block.
Condition Browse Module
MeSH Terms
Intervention Browse Module
MeSH Terms
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Not provided
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
Triple
Masking Description
Not provided
Who Masked
ParticipantCare ProviderInvestigator
desmopressin melt 100 μg
Experimental
Participants will take desmopressin melt 100 μg for 28 days to complete part 1 of the study. Participants will continue on this dose in part 2 of the study for between 1-6 months (until the database for part 1 is locked and treatment is unblinded).
Drug: desmopressin acetate
desmopressin melt 10 μg
desmopressin melt 100 μg
desmopressin melt 25 μg
desmopressin melt 50 μg
Minirin® Melt
Nocturin®
FE992026
Placebo
Drug
Oral placebo placed under the participant's tongue, without water, once daily approximately 1 hour before bedtime.
Placebo
Part II: Percentage of Participants With Greater Than 33 Percent Reduction From Baseline in Mean Number of Nocturnal Voids to Days 29, 57, 113 and 169
Part II outcomes tested the durability of the effect observed in Part I. Percentage of participants in each treatment arm that had a greater than 33% reduction from baseline to Days 29, 57, 113 and 169 in mean number of nocturnal voids. Nocturnal void data were recorded in participant diaries.
- Week 3 to Day 1 (Baseline), Days 29, 57, 113 and 169
Part I: Change From Baseline in Total Reported Sleep Time at Week 4
Total sleep time was recorded by participants in study diaries.
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
Part I: Change From Baseline in Initial Period of Undisturbed Sleep at Week 4
Initial period of undisturbed sleep was the time elapsed from first falling asleep until either first void or morning arising. Data were captured in patient diaries.
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
Part I: Change From Baseline in Quality of Life Assessed by The International Consultation on Incontinence Modular Questionnaire - Nocturia (ICIQ-N) at Week 4
The ICIQ-N is a self-administered questionnaire designed to assess the frequency and bother of daytime and nighttime urination. Subjects were asked to rate the degree of bother of daytime urination and nighttime urination on a scale ranging from 0 (not at all) to 10 (a great deal). Higher numbers indicate lower quality of life.
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
Part I: Change From Baseline in the Two Domain Scores of the Nocturia Quality of Life (NQoL) Questionnaire at Week 4
The NQoL questionnaire is a self-administered questionnaire designed to assess the impact of nocturia on quality of life. It contains a sleep/energy domain (6 questions), a bother/concern domain (6 questions), and 1 global QoL question. The twelve core questions are scored on a 0 to 4 scale with higher numbers indicating a better quality of life. Domain summary scores were calculated by transforming the raw score into a 0-100 scale with higher numbers indicating a better quality of life.
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
Part I: Change From Baseline in Quality of Sleep as Assessed by the Global Score of the Pittsburgh Sleep Quality Index (PSQI) at Week 4
The PSQI is a self-administered 19-item questionnaire designed to assess sleep quality and disturbances. The global score ranges from 0 (better sleep quality) to 21 (worse sleep quality). Higher numbers indicate lower quality of life.
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
Part I: Change From Baseline in the Mental Health Summary and the Physical Health Summary of the Short Form-12 Version 2 (SF-12v2) at Week 4
The SF-12v2 was used to measure the impact of nocturia and lack of sleep on general quality of life. The SF-12 consists of 12 questions. Data were analyzed using norm-based scoring and summarized along 2 dimensions: Physical Health Summary and Mental Health Summary. Each summary has a range from 0 (poor health) to 100 (highest level of health). Higher numbers indicate better quality of life.
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
Part I: Participants With Treatment-Emergent Adverse Events (AEs) During Study Part I
A treatment-emergent adverse event (AE) was any AE occurring during the treatment period or a pretreatment AE that worsened in intensity during the treatment period. The treatment period was the period during which a subject received investigational medicinal product. If a subject discontinued the investigational medicinal product, the date of last dose was the last day of the treatment period.
Day 1 up to Week 4 (end of Part I)
Part II: Participants With Treatment-Emergent Adverse Events (AEs) During Study Part II
A treatment-emergent adverse event (AE) was any AE occurring during the treatment period or a pretreatment AE that worsened in intensity during the treatment period. The treatment period was the period during which a subject received investigational medicinal product. If a subject discontinued the investigational medicinal product, the date of last dose was the last day of the treatment period.
Juul KV, Malmberg A, van der Meulen E, Walle JV, Norgaard JP. Low-dose desmopressin combined with serum sodium monitoring can prevent clinically significant hyponatraemia in patients treated for nocturia. BJU Int. 2017 May;119(5):776-784. doi: 10.1111/bju.13718. Epub 2016 Dec 10.
Bliwise DL, Holm-Larsen T, Goble S. Increases in duration of first uninterrupted sleep period are associated with improvements in PSQI-measured sleep quality. Sleep Med. 2014 Oct;15(10):1276-8. doi: 10.1016/j.sleep.2014.05.013. Epub 2014 Jun 13.
Juul KV, Klein BM, Sandstrom R, Erichsen L, Norgaard JP. Gender difference in antidiuretic response to desmopressin. Am J Physiol Renal Physiol. 2011 May;300(5):F1116-22. doi: 10.1152/ajprenal.00741.2010. Epub 2011 Mar 2.
Desmopressin Melt 10 μg
Participants took desmopressin melt 10 μg for 28 days to complete Part I of the study. Participants continued this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
FG002
Desmopressin Melt 25 μg
Participants took desmopressin melt 25 μg for 28 days to complete Part I of the study. Participants continued this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
FG003
Desmopressin Melt 50 μg
Participants took desmopressin melt 50 μg for 28 days to complete Part I of the study. Participants continued this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
FG004
Desmopressin Melt 100 μg
Participants took desmopressin melt 100 μg for 28 days to complete Part I of the study. Participants continued this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
FG005
Placebo to 10 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 10 μg once daily about an hour before bedtime.
FG006
Placebo to 25 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 25 μg once daily about an hour before bedtime.
FG007
Placebo to 50 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 50 μg once daily about an hour before bedtime.
FG008
Placebo to 100 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 100 μg once daily about an hour before bedtime.
FG000160 subjects
FG001163 subjects
FG002158 subjects
FG003158 subjects
FG004160 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
ITT Population
FG000156 subjectsReceived at least one dose of study drug and provided at least one primary efficacy measure.
FG001155 subjectsReceived at least one dose of study drug and provided at least one primary efficacy measure.
FG002152 subjectsReceived at least one dose of study drug and provided at least one primary efficacy measure.
FG003148 subjectsReceived at least one dose of study drug and provided at least one primary efficacy measure.
FG004146 subjectsReceived at least one dose of study drug and provided at least one primary efficacy measure.
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
COMPLETED
FG000145 subjects
FG001144 subjects
FG002148 subjects
FG003138 subjects
FG004135 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
NOT COMPLETED
FG00015 subjects
FG00119 subjects
FG00210 subjects
FG00320 subjects
FG00425 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
Type
Comment
Reasons
Adverse Event
FG0001 subjects
FG0014 subjects
FG0020 subjects
FG0035 subjects
FG0046 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
Serum Sodium <= 125mmol/L
FG0001 subjects
FG0010 subjects
FG0020 subjects
FG0035 subjects
FG004
Protocol Violation
FG0002 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
Withdrawal by Subject
FG0006 subjects
FG0015 subjects
FG0026 subjects
FG0034 subjects
FG004
Lost to Follow-up
FG0002 subjects
FG0014 subjects
FG0021 subjects
FG0035 subjects
FG004
Other, not specified
FG0002 subjects
FG0014 subjects
FG0020 subjects
FG0031 subjects
FG004
Not Reported
FG0001 subjects
FG0012 subjects
FG0023 subjects
FG0030 subjects
FG004
Part II: Extension Week 5 up to Day 169
Type
Comment
Milestone Data
STARTED
FG0000 subjects
FG001135 subjects
FG002135 subjects
FG003132 subjects
FG004127 subjects
FG00531 subjects
FG00637 subjects
FG00734 subjects
FG00834 subjects
COMPLETED
FG0000 subjects
FG001107 subjects
FG002120 subjects
FG003120 subjects
FG004
NOT COMPLETED
FG0000 subjects
FG00128 subjects
FG00215 subjects
FG00312 subjects
FG004
Type
Comment
Reasons
Withdrawal by Subject
FG0000 subjects
FG00115 subjects
FG0027 subjects
FG003
Demographic data offered from the Intent-to-Treat population
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Placebo
Participants took a placebo 'melt' for 28 days to complete Part I of the study. In Part II, placebo patients were randomized to one of the other four treatment arms to receive active desmopressin melt for between 1-6 months (until the database for part 1 was locked and treatment was unblinded).
BG001
Desmopressin Melt 10 μg
Participants took desmopressin melt 10 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
BG002
Desmopressin Melt 25 μg
Participants took desmopressin melt 25 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
BG003
Desmopressin Melt 50 μg
Participants took desmopressin melt 50 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
BG004
Desmopressin Melt 100 μg
Participants took desmopressin melt 100 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
BG005
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG000156
BG001155
BG002152
BG003148
BG004146
BG005757
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Standard Deviation
years
Title
Denominators
Categories
Title
Measurements
BG00062.1± 13.39
BG00161.7± 14.41
BG00262.4± 13.22
BG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG00066
BG00173
BG002
Race/Ethnicity, Customized
Number
participants
Title
Denominators
Categories
Caucasian
Title
Measurements
BG000136
BG001123
BG002
Ethnic Origin
Number
participants
Title
Denominators
Categories
Hispanic
Title
Measurements
BG00013
BG00110
BG002
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
Part I: Change From Baseline in Mean Number of Nocturnal Voids at Week 4
The number of nocturnal voids was the average over 3 consecutive 24-hours periods prior to Day 1 and prior to the week 4 visit as recorded in participant diaries.
This was the first co-primary outcome.
Intent to treat (ITT) population --All randomized participants who received at least one dose of study drug and provided at least one primary efficacy measure (i.e., nocturnal voids) during Part I were included in the ITT analysis dataset
Posted
Mean
Standard Deviation
nocturnal voids
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
ID
Title
Description
OG000
Placebo
Participants took a placebo 'melt' for 28 days to complete Part I of the study. In Part II, placebo patients were randomized to one of the other four treatment arms to receive active desmopressin melt for between 1-6 months (until the database for part 1 was locked and treatment was unblinded).
OG001
Desmopressin Melt 10 μg
Participants took desmopressin melt 10 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG002
Desmopressin Melt 25 μg
Participants took desmopressin melt 25 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG003
Desmopressin Melt 50 μg
Participants took desmopressin melt 50 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG004
Desmopressin Melt 100 μg
Participants took desmopressin melt 100 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
Units
Counts
Participants
OG000156
OG001155
OG002152
OG003
Title
Denominators
Categories
Title
Measurements
OG000-0.86± 1.054
OG001-0.83± 1.069
OG002-1.00± 1.125
OG003
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
ANCOVA
Treatment difference and 95% CI were adjusted for age, absence/presence of nocturnal polyuria, and baseline number of nocturnal voids.
0.9303
The a priori statistically significant difference versus placebo is p≤0.05.
Mean Difference (Final Values)
0.010
2-Sided
95
-0.221
0.242
No
Superiority or Other
Primary
Part I: Percentage of Participants With Greater Than 33 Percent Reduction From Baseline in Mean Number of Nocturnal Voids at Week 4
Percentage of participants in each treatment arm that had a greater than 33% reduction from baseline to the end of Part I (week 4) in mean number of nocturnal voids. Nocturnal void data were recorded in participant diaries.
This was the second co-primary outcome.
Intent to treat (ITT) population --All randomized participants who received at least one dose of study drug and provided at least one primary efficacy measure (i.e., nocturnal voids) during Part I were included in the ITT analysis dataset
Posted
Number
percentage of participants
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
ID
Title
Description
OG000
Placebo
Participants took a placebo 'melt' for 28 days to complete Part I of the study. In Part II, placebo patients were randomized to one of the other four treatment arms to receive active desmopressin melt for between 1-6 months (until the database for part 1 was locked and treatment was unblinded).
OG001
Desmopressin Melt 10 μg
Participants took desmopressin melt 10 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG002
Secondary
Part II: Change From Baseline in Mean Number of Nocturnal Voids to Days 29, 57, 113 and 169
Part II outcomes tested the durability of the effect observed in Part I. The number of nocturnal voids was the average over 3 consecutive 24-hours periods prior to Part I baseline and prior to the Part II visit as recorded in participant diaries.
Observed Cases (OC) Analysis Set which consisted of participants who had information on mean number of nocturnal voids for both the screening visit and the final visit in Part I (Day 28) and did not have any major protocol deviations.
Posted
Mean
Standard Deviation
nocturnal voids
- Week 3 to Day 1 (Baseline), Days 29, 57, 113 and 169
ID
Title
Description
OG000
Desmopressin Melt 10 μg
Participants took desmopressin melt 10 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG001
Desmopressin Melt 25 μg
Participants took desmopressin melt 25 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG002
Desmopressin Melt 50 μg
Secondary
Part II: Percentage of Participants With Greater Than 33 Percent Reduction From Baseline in Mean Number of Nocturnal Voids to Days 29, 57, 113 and 169
Part II outcomes tested the durability of the effect observed in Part I. Percentage of participants in each treatment arm that had a greater than 33% reduction from baseline to Days 29, 57, 113 and 169 in mean number of nocturnal voids. Nocturnal void data were recorded in participant diaries.
Observed Cases (OC) Analysis Set which consisted of participants who had information on mean number of nocturnal voids for both the screening visit and the final visit in Part I (Day 28) and did not have any major protocol deviations. Participants had data representing the visit.
Posted
Number
percentage of participants
- Week 3 to Day 1 (Baseline), Days 29, 57, 113 and 169
ID
Title
Description
OG000
Desmopressin Melt 10 μg
Participants took desmopressin melt 10 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG001
Desmopressin Melt 25 μg
Participants took desmopressin melt 25 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
Secondary
Part I: Change From Baseline in Total Reported Sleep Time at Week 4
Total sleep time was recorded by participants in study diaries.
Intent to treat (ITT) population --All randomized subjects who received at least one dose of study drug and provided at least one primary efficacy measure (i.e., number of nocturnal voids) during Part I were included in the ITT analysis dataset. Participants analyzed had baseline and Week 4/Day 28/End of Part 1 measurements.
Posted
Mean
Standard Deviation
minutes
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
ID
Title
Description
OG000
Placebo
Participants took a placebo 'melt' for 28 days to complete Part I of the study. In Part II, placebo patients were randomized to one of the other four treatment arms to receive active desmopressin melt for between 1-6 months (until the database for part 1 was locked and treatment was unblinded).
OG001
Desmopressin Melt 10 μg
Participants took desmopressin melt 10 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG002
Desmopressin Melt 25 μg
Participants took desmopressin melt 25 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
Secondary
Part I: Change From Baseline in Initial Period of Undisturbed Sleep at Week 4
Initial period of undisturbed sleep was the time elapsed from first falling asleep until either first void or morning arising. Data were captured in patient diaries.
ITT population --All randomized subjects who received at least one dose of study drug and provided at least one primary efficacy measure (i.e., number of nocturnal voids) during Part I were included in the ITT analysis dataset. Participants with both baseline and Week 4/Day 28/End of Part I data are included.
Posted
Mean
Standard Deviation
minutes
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
ID
Title
Description
OG000
Placebo
Participants took a placebo 'melt' for 28 days to complete Part I of the study. In Part II, placebo patients were randomized to one of the other four treatment arms to receive active desmopressin melt for between 1-6 months (until the database for part 1 was locked and treatment was unblinded).
OG001
Desmopressin Melt 10 μg
Participants took desmopressin melt 10 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG002
Desmopressin Melt 25 μg
Secondary
Part I: Change From Baseline in Quality of Life Assessed by The International Consultation on Incontinence Modular Questionnaire - Nocturia (ICIQ-N) at Week 4
The ICIQ-N is a self-administered questionnaire designed to assess the frequency and bother of daytime and nighttime urination. Subjects were asked to rate the degree of bother of daytime urination and nighttime urination on a scale ranging from 0 (not at all) to 10 (a great deal). Higher numbers indicate lower quality of life.
Intent to treat population of participants who completed the questionnaire at both baseline and end of Part 1.
Posted
Mean
Standard Deviation
units on a scale
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
ID
Title
Description
OG000
Placebo
Participants took a placebo 'melt' for 28 days to complete Part I of the study. In Part II, placebo patients were randomized to one of the other four treatment arms to receive active desmopressin melt for between 1-6 months (until the database for part 1 was locked and treatment was unblinded).
OG001
Desmopressin Melt 10 μg
Participants took desmopressin melt 10 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG002
Secondary
Part I: Change From Baseline in the Two Domain Scores of the Nocturia Quality of Life (NQoL) Questionnaire at Week 4
The NQoL questionnaire is a self-administered questionnaire designed to assess the impact of nocturia on quality of life. It contains a sleep/energy domain (6 questions), a bother/concern domain (6 questions), and 1 global QoL question. The twelve core questions are scored on a 0 to 4 scale with higher numbers indicating a better quality of life. Domain summary scores were calculated by transforming the raw score into a 0-100 scale with higher numbers indicating a better quality of life.
Intent to treat population of participants who completed the questionnaire at both baseline and end of Part 1.
Posted
Mean
Standard Deviation
units on a scale
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
ID
Title
Description
OG000
Placebo
Participants took a placebo 'melt' for 28 days to complete Part I of the study. In Part II, placebo patients were randomized to one of the other four treatment arms to receive active desmopressin melt for between 1-6 months (until the database for part 1 was locked and treatment was unblinded).
OG001
Desmopressin Melt 10 μg
Participants took desmopressin melt 10 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
Secondary
Part I: Change From Baseline in Quality of Sleep as Assessed by the Global Score of the Pittsburgh Sleep Quality Index (PSQI) at Week 4
The PSQI is a self-administered 19-item questionnaire designed to assess sleep quality and disturbances. The global score ranges from 0 (better sleep quality) to 21 (worse sleep quality). Higher numbers indicate lower quality of life.
Intent to treat population of participants who completed the questionnaire at both baseline and end of Part 1.
Posted
Mean
Standard Deviation
units on a scale
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
ID
Title
Description
OG000
Placebo
Participants took a placebo 'melt' for 28 days to complete Part I of the study. In Part II, placebo patients were randomized to one of the other four treatment arms to receive active desmopressin melt for between 1-6 months (until the database for part 1 was locked and treatment was unblinded).
OG001
Desmopressin Melt 10 μg
Participants took desmopressin melt 10 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG002
Desmopressin Melt 25 μg
Secondary
Part I: Change From Baseline in the Mental Health Summary and the Physical Health Summary of the Short Form-12 Version 2 (SF-12v2) at Week 4
The SF-12v2 was used to measure the impact of nocturia and lack of sleep on general quality of life. The SF-12 consists of 12 questions. Data were analyzed using norm-based scoring and summarized along 2 dimensions: Physical Health Summary and Mental Health Summary. Each summary has a range from 0 (poor health) to 100 (highest level of health). Higher numbers indicate better quality of life.
Intent to treat population of participants who completed the questionnaire at both baseline and end of Part 1.
Posted
Mean
Standard Deviation
units on a scale
- Week 3 to Day 1 (Baseline), Week 4 (end of Part I)
ID
Title
Description
OG000
Placebo
Participants took a placebo 'melt' for 28 days to complete Part I of the study. In Part II, placebo patients were randomized to one of the other four treatment arms to receive active desmopressin melt for between 1-6 months (until the database for part 1 was locked and treatment was unblinded).
OG001
Desmopressin Melt 10 μg
Participants took desmopressin melt 10 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG002
Secondary
Part I: Participants With Treatment-Emergent Adverse Events (AEs) During Study Part I
A treatment-emergent adverse event (AE) was any AE occurring during the treatment period or a pretreatment AE that worsened in intensity during the treatment period. The treatment period was the period during which a subject received investigational medicinal product. If a subject discontinued the investigational medicinal product, the date of last dose was the last day of the treatment period.
Part I Safety Population includes study participants who received study intervention and had at least one safety assessment during study Part I.
Posted
Number
participants
Day 1 up to Week 4 (end of Part I)
ID
Title
Description
OG000
Placebo
Participants took a placebo 'melt' for 28 days to complete Part I of the study. In Part II, placebo patients were randomized to one of the other four treatment arms to receive active desmopressin melt for between 1-6 months (until the database for part 1 was locked and treatment was unblinded).
OG001
Desmopressin Melt 10 μg
Participants took desmopressin melt 10 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG002
Desmopressin Melt 25 μg
Secondary
Part II: Participants With Treatment-Emergent Adverse Events (AEs) During Study Part II
A treatment-emergent adverse event (AE) was any AE occurring during the treatment period or a pretreatment AE that worsened in intensity during the treatment period. The treatment period was the period during which a subject received investigational medicinal product. If a subject discontinued the investigational medicinal product, the date of last dose was the last day of the treatment period.
Part II Safety Population includes study participants who received study intervention and had at least one safety assessment during study Part II.
Posted
Number
participants
Week 5 up to Day 169
ID
Title
Description
OG000
Desmopressin Melt 10 μg
Participants took desmopressin melt 10 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG001
Desmopressin Melt 25 μg
Participants took desmopressin melt 25 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG002
Desmopressin Melt 50 μg
Time Frame
Part I covers Day 1 to Week 4. Part II covers Week 5 up to Day169.
Description
Not provided
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
Part I: Placebo
Participants took a placebo 'melt' for 28 days to complete Part I of the study.
1
160
64
160
EG001
Part I: Desmopressin Melt 10 μg
Participants took desmopressin melt 10 μg for 28 days to complete Part I of the study.
2
163
67
163
EG002
Part I: Desmopressin Melt 25 μg
Participants took desmopressin melt 25 μg for 28 days to complete Part I of the study.
1
158
62
158
EG003
Part I: Desmopressin Melt 50 μg
Participants took desmopressin melt 50 μg for 28 days to complete Part I of the study.
2
158
76
158
EG004
Part I: Desmopressin Melt 100 μg
Participants took desmopressin melt 100 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
0
160
79
160
EG005
Part II: Desmopressin Melt 10 μg
Participants who took desmopressin melt 10 μg in Part I of the study continued on this dose in Part II which ran between Months 1-6 (until the database for Part I was locked and treatment was unblinded).
3
135
71
135
EG006
Part II: Desmopressin Melt 25 μg
Participants who took desmopressin melt 25 μg in Part I of the study continued on this dose in Part II which ran between Months 1-6 (until the database for Part I was locked and treatment was unblinded).
1
135
69
135
EG007
Part II: Desmopressin Melt 50 μg
Participants who took desmopressin melt 50 μg in Part I of the study continued on this dose in Part II which ran between Months 1-6 (until the database for Part I was locked and treatment was unblinded).
0
132
66
132
EG008
Part II: Desmopressin Melt 100 μg
Participants who took desmopressin melt 100 μg in Part I of the study continued on this dose in Part II which ran between Months 1-6 (until the database for Part I was locked and treatment was unblinded).
2
127
73
127
EG009
Part II: Placebo to Desmopressin Melt 10 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 10 μg once daily about an hour before bedtime
0
31
17
31
EG010
Part II: Placebo to Desmopressin Melt 25 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 25 μg once daily about an hour before bedtime.
0
37
23
37
EG011
Part II: Placebo to Desmopressin Melt 50 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 50 μg once daily about an hour before bedtime.
2
34
19
34
EG012
Part II: Placebo to Desmopressin Melt 100 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 100 μg once daily about an hour before bedtime.
2
34
20
34
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Atrial fibrillation
Cardiac disorders
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0011 affected163 at risk
EG0020 affected158 at risk
EG0030 affected158 at risk
EG0040 affected160 at risk
EG0050 affected135 at risk
EG0060 affected135 at risk
EG0070 affected132 at risk
EG0080 affected127 at risk
EG0090 affected31 at risk
EG0100 affected37 at risk
EG0110 affected34 at risk
EG0120 affected34 at risk
Cardiac failure congestive
Cardiac disorders
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Myocardial infarction
Cardiac disorders
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Diverticulitis
Gastrointestinal disorders
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0011 affected163 at risk
EG0020 affected158 at risk
EG003
Duodenal ulcer haemorrhage
Gastrointestinal disorders
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Appendicitis
Infections and infestations
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Cellulitis
Infections and infestations
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Pneumonia
Infections and infestations
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Myalgia
Musculoskeletal and connective tissue disorders
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0021 affected158 at risk
EG003
Adenosquamous cell lung cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Bladder cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Metastasis
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Prostate cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (10.0)
Systematic Assessment
EG0001 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Asthma
Respiratory, thoracic and mediastinal disorders
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Knee arthroplasty
Surgical and medical procedures
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Dry mouth
Gastrointestinal disorders
MedDRA (10.0)
Systematic Assessment
EG00053 affected160 at risk
EG00153 affected163 at risk
EG00250 affected158 at risk
EG00354 affected158 at risk
EG00449 affected160 at risk
EG00556 affected135 at risk
EG00647 affected135 at risk
EG00746 affected132 at risk
EG00847 affected127 at risk
EG00913 affected31 at risk
EG01014 affected37 at risk
EG01113 affected34 at risk
EG01213 affected34 at risk
Nausea
Gastrointestinal disorders
MedDRA (10.0)
Systematic Assessment
EG0001 affected160 at risk
EG0014 affected163 at risk
EG0024 affected158 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA (10.0)
Systematic Assessment
EG0003 affected160 at risk
EG0015 affected163 at risk
EG0026 affected158 at risk
EG003
Abdominal pain upper
Gastrointestinal disorders
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Dyspepsia
Gastrointestinal disorders
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Pain
General disorders
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Upper respiratory tract infection
Infections and infestations
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Urinary tract infection
Infections and infestations
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Blood glucose increased
Investigations
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Blood sodium decreased
Investigations
MedDRA (10.0)
Systematic Assessment
EG0001 affected160 at risk
EG0012 affected163 at risk
EG0022 affected158 at risk
EG003
Hyponatraemia
Metabolism and nutrition disorders
MedDRA (10.0)
Systematic Assessment
EG0003 affected160 at risk
EG0011 affected163 at risk
EG0021 affected158 at risk
EG003
Headache
Nervous system disorders
MedDRA (10.0)
Systematic Assessment
EG00011 affected160 at risk
EG00113 affected163 at risk
EG0026 affected158 at risk
EG003
Dizziness
Nervous system disorders
MedDRA (10.0)
Systematic Assessment
EG0001 affected160 at risk
EG0017 affected163 at risk
EG0023 affected158 at risk
EG003
Depression
Psychiatric disorders
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Pollakiuria
Renal and urinary disorders
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Hypertension
Vascular disorders
MedDRA (10.0)
Systematic Assessment
EG0000 affected160 at risk
EG0010 affected163 at risk
EG0020 affected158 at risk
EG003
Certain Agreements
Are all PI(s) employees of the sponsor?
No
Restriction Type
OTHER
Results Disclosure Restriction on PI(s)?
Yes
Other Details
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
Point of Contact
Title
Organization
Phone
Extension
Email
Clinical Development Support
Ferring Pharmaceuticals
DK0-Disclosure@ferring.com
ID
Term
D053158
Nocturia
Ancestor Terms
ID
Term
D059411
Lower Urinary Tract Symptoms
D020924
Urological Manifestations
D012816
Signs and Symptoms
D013568
Pathological Conditions, Signs and Symptoms
Browse Leaves
Not provided
Browse Branches
Not provided
ID
Term
D003894
Deamino Arginine Vasopressin
Ancestor Terms
ID
Term
D001127
Arginine Vasopressin
D014667
Vasopressins
D010909
Pituitary Hormones, Posterior
D010907
Pituitary Hormones
D036361
Peptide Hormones
D006728
Hormones
D006730
Hormones, Hormone Substitutes, and Hormone Antagonists
D009479
Neuropeptides
D010455
Peptides
D000602
Amino Acids, Peptides, and Proteins
D009842
Oligopeptides
D009419
Nerve Tissue Proteins
D011506
Proteins
Browse Leaves
Not provided
Browse Branches
Not provided
4 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
1 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
9 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
3 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
1 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
1 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
107 subjects
FG00525 subjects
FG00632 subjects
FG00729 subjects
FG00826 subjects
20 subjects
FG0056 subjects
FG0065 subjects
FG0075 subjects
FG0088 subjects
7 subjects
FG00410 subjects
FG0054 subjects
FG0062 subjects
FG0073 subjects
FG0082 subjects
Adverse Event
FG0000 subjects
FG0012 subjects
FG0023 subjects
FG0031 subjects
FG0041 subjects
FG0051 subjects
FG0061 subjects
FG0071 subjects
FG0084 subjects
Lost to Follow-up
FG0000 subjects
FG0013 subjects
FG0022 subjects
FG0030 subjects
FG0045 subjects
FG0050 subjects
FG0061 subjects
FG0070 subjects
FG0080 subjects
Decreased sodium
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG0041 subjects
FG0050 subjects
FG0060 subjects
FG0071 subjects
FG0081 subjects
Excluded medication
FG0000 subjects
FG0011 subjects
FG0020 subjects
FG0030 subjects
FG0040 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
Other, not specified
FG0000 subjects
FG0014 subjects
FG0022 subjects
FG0034 subjects
FG0042 subjects
FG0051 subjects
FG0061 subjects
FG0070 subjects
FG0081 subjects
Not reported
FG0000 subjects
FG0013 subjects
FG0021 subjects
FG0030 subjects
FG0041 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
61.6
± 11.80
BG00462.1± 12.34
BG00562.0± 13.05
65
BG00371
BG00466
BG005341
Male
BG00090
BG00182
BG00287
BG00377
BG00480
BG005416
120
BG003119
BG004111
BG005609
Black/African American
Title
Measurements
BG00016
BG00121
BG00228
BG00324
BG00427
BG005116
Asian
Title
Measurements
BG0003
BG0012
BG0022
BG0033
BG0046
BG00516
American Indian/Alaskan Native
Title
Measurements
BG0000
BG0012
BG0020
BG0030
BG0040
BG0052
Native Hawaiian/other Pacific Islander
Title
Measurements
BG0000
BG0011
BG0021
BG0030
BG0040
BG0052
Other
Title
Measurements
BG0001
BG0016
BG0021
BG0032
BG0042
BG00512
10
BG00313
BG0046
BG00552
Not Hispanic
Title
Measurements
BG000143
BG001145
BG002142
BG003135
BG004140
BG005705
148
OG004146
-1.18
± 1.187
OG004-1.43± 1.219
OG000
OG002
ANCOVA
Treatment difference and 95% CI were adjusted for age, absence/presence of nocturnal polyuria, and baseline number of nocturnal voids.
0.3104
The a priori statistically significant difference versus placebo is p≤0.05.
Mean Difference (Final Values)
-0.120
2-Sided
95
-0.353
0.112
No
Superiority or Other
OG000
OG003
The trial was to be declared positive only if in the 100 μg group, a statistically significant positive effect as compared to placebo on both co-primaries was demonstrated. And only then, the next higher dose group (i.e., 50 μg) could be claimed superior to placebo, if it showed a statistically significant improvement over placebo on both co-primaries.
ANCOVA
Treatment difference and 95% CI were adjusted for age, absence/presence of nocturnal polyuria, and baseline number of nocturnal voids.
0.0207
The a priori statistically significant difference versus placebo is p≤0.05.
Mean Difference (Final Values)
-0.277
2-Sided
95
-0.511
-0.042
No
Superiority or Other
OG000
OG004
The trial was to be declared positive only if in the 100 μg group, a statistically significant positive effect as compared to placebo on both co-primaries was demonstrated. And only then, the next higher dose group (i.e., 50 μg) could be claimed superior to placebo, if it showed a statistically significant improvement over placebo on both co-primaries.
ANCOVA
Treatment difference and 95% CI were adjusted for age, absence/presence of nocturnal polyuria, and baseline number of nocturnal voids.
<0.0001
The a priori statistically significant difference versus placebo is p≤0.05.
Mean Difference (Final Values)
-0.613
2-Sided
95
-0.848
-0.378
No
Superiority or Other
Desmopressin Melt 25 μg
Participants took desmopressin melt 25 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG003
Desmopressin Melt 50 μg
Participants took desmopressin melt 50 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG004
Desmopressin Melt 100 μg
Participants took desmopressin melt 100 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
Units
Counts
Participants
OG000156
OG001155
OG002152
OG003148
OG004146
Title
Denominators
Categories
Title
Measurements
OG00047
OG00147
OG00250
OG00353
OG00471
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Regression, Logistic
Treatment difference and 95% CI were adjusted for age, absence/presence of nocturnal polyuria, and baseline number of nocturnal voids
0.9420
The a priori statistically significant difference versus placebo is p≤0.05.
Odds Ratio (OR)
1.017
2-Sided
95
0.647
1.598
No
Superiority or Other
OG000
OG002
Regression, Logistic
Treatment difference and 95% CI were adjusted for age, absence/presence of nocturnal polyuria, and baseline number of nocturnal voids.
0.5527
The a priori statistically significant difference versus placebo is p≤0.05.
Odds Ratio (OR)
1.147
2-Sided
95
0.729
1.807
No
Superiority or Other
OG000
OG003
The trial was to be declared positive only if in the 100 μg group, a statistically significant positive effect as compared to placebo on both co-primaries was demonstrated. And only then, the next higher dose group (i.e., 50 μg) could be claimed superior to placebo, if it showed a statistically significant improvement over placebo on both co-primaries.
Regression, Logistic
Treatment difference and 95% CI were adjusted for age, absence/presence of nocturnal polyuria, and baseline number of nocturnal voids.
0.2662
The a priori statistically significant difference versus placebo is p≤0.05.
Odds Ratio (OR)
1.296
2-Sided
95
0.821
2.050
No
Superiority or Other
OG000
OG004
The trial was to be declared positive only if in the 100 μg group, a statistically significant positive effect as compared to placebo on both co-primaries was demonstrated. And only then, the next higher dose group (i.e., 50 μg) could be claimed superior to placebo, if it showed a statistically significant improvement over placebo on both co-primaries.
Regression, Logistic
Treatment difference and 95% CI were adjusted for age, absence/presence of nocturnal polyuria, and baseline number of nocturnal voids.
<0.0001
The a priori statistically significant difference versus placebo is p≤0.05.
Odds Ratio (OR)
2.893
2-Sided
95
1.795
4.715
No
Superiority or Other
Participants took desmopressin melt 50 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG003
Desmopressin Melt 100 μg
Participants took desmopressin melt 100 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG004
Placebo to 10 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 10 μg once daily about an hour before bedtime.
OG005
Placebo to 25 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 25 μg once daily about an hour before bedtime.
OG006
Placebo to 50 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 50 μg once daily about an hour before bedtime.
OG007
Placebo to 100 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 100 μg once daily about an hour before bedtime.
Units
Counts
Participants
OG000104
OG001100
OG00293
OG003104
OG00422
OG00528
OG00627
OG00722
Title
Denominators
Categories
Day 29 (n=104,100,93,104,22,28,27,22)
Title
Measurements
OG000-1.12± 1.161
OG001-1.31± 1.163
OG002-1.43± 1.273
OG003-1.47± 1.080
OG004-1.20± 0.912
OG005-1.19± 1.215
OG006-1.00± 1.140
OG007-1.58± 1.169
Day 57 (n=86,85,78,81,17,24,21,16)
Title
Measurements
OG000-1.14± 1.147
OG001-1.42± 1.231
OG002-1.50± 1.156
OG003
Day 113 (n=53,56,49,47,11,15,13,11)
Title
Measurements
OG000-1.31± 1.195
OG001-1.56± 1.158
OG002-1.65± 1,279
OG003
Day 169 (n=17,15,17,15,3,3,4,2)
Title
Measurements
OG000-1.47± 1.537
OG001-1.71± 1.214
OG002-1.65± 0.916
OG003
OG002
Desmopressin Melt 50 μg
Participants took desmopressin melt 50 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG003
Desmopressin Melt 100 μg
Participants took desmopressin melt 100 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG004
Placebo to 10 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 10 μg once daily about an hour before bedtime.
OG005
Placebo to 25 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 25 μg once daily about an hour before bedtime.
OG006
Placebo to 50 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 50 μg once daily about an hour before bedtime.
OG007
Placebo to 100 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 100 μg once daily about an hour before bedtime.
Units
Counts
Participants
OG000104
OG001100
OG00293
OG003104
OG00422
OG00528
OG00627
OG00722
Title
Denominators
Categories
Day 29 (n=104,100,93,104,22,28,27,22)
Title
Measurements
OG00057
OG00164
OG00268
OG00368
OG00473
OG00543
OG00648
OG00773
Day 57 (n=86,85,78,81,17,24,21,16)
Title
Measurements
OG00059
OG00168
OG00271
OG003
Day 113 (n=53,56,49,47,11,15,13,11)
Title
Measurements
OG00058
OG00179
OG00269
OG003
Day 169 (n=17,15,17,15,3,3,4,2)
Title
Measurements
OG00061
OG00180
OG00276
OG003
OG003
Desmopressin Melt 50 μg
Participants took desmopressin melt 50 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG004
Desmopressin Melt 100 μg
Participants took desmopressin melt 100 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
Units
Counts
Participants
OG000139
OG001137
OG002141
OG003138
OG004133
Title
Denominators
Categories
Title
Measurements
OG00024.6± 80.66
OG0017.8± 58.55
OG00215.9± 53.92
OG00324.9± 72.21
OG00419.0± 68.94
Participants took desmopressin melt 25 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG003
Desmopressin Melt 50 μg
Participants took desmopressin melt 50 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG004
Desmopressin Melt 100 μg
Participants took desmopressin melt 100 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
Units
Counts
Participants
OG000126
OG001126
OG002121
OG003123
OG004121
Title
Denominators
Categories
Title
Measurements
OG00038.7± 88.79
OG00150.9± 111.47
OG00282.7± 105.57
OG00385.1± 109.33
OG004106.7± 116.18
Desmopressin Melt 25 μg
Participants took desmopressin melt 25 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG003
Desmopressin Melt 50 μg
Participants took desmopressin melt 50 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG004
Desmopressin Melt 100 μg
Participants took desmopressin melt 100 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
Units
Counts
Participants
OG000147
OG001145
OG002143
OG003137
OG004137
Title
Denominators
Categories
Daytime urination: How much does it bother you?
Title
Measurements
OG000-0.7± 2.61
OG001-0.7± 2.59
OG002-0.9± 2.96
OG003-0.7± 2.86
OG004-1.0± 2.90
Nighttime urination: How much does it bother you?
Title
Measurements
OG000-1.4± 2.86
OG001-1.8± 2.96
OG002-2.1± 3.19
OG003
OG002
Desmopressin Melt 25 μg
Participants took desmopressin melt 25 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG003
Desmopressin Melt 50 μg
Participants took desmopressin melt 50 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG004
Desmopressin Melt 100 μg
Participants took desmopressin melt 100 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
Units
Counts
Participants
OG000147
OG001148
OG002145
OG003135
OG004138
Title
Denominators
Categories
Sleep/Energy Domain
Title
Measurements
OG00015.2± 19.30
OG00112.3± 23.54
OG00214.6± 18.71
OG00314.7± 20.92
OG00416.3± 21.09
Bother/Concern Domain
Title
Measurements
OG00012.7± 19.73
OG00114.7± 23.42
OG00215.7± 21.29
OG003
Participants took desmopressin melt 25 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG003
Desmopressin Melt 50 μg
Participants took desmopressin melt 50 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG004
Desmopressin Melt 100 μg
Participants took desmopressin melt 100 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
Units
Counts
Participants
OG000143
OG001138
OG002136
OG003129
OG004132
Title
Denominators
Categories
Title
Measurements
OG000-1.6± 2.8
OG001-1.6± 2.8
OG002-1.8± 3.0
OG003-2.0± 3.2
OG004-1.9± 3.0
Desmopressin Melt 25 μg
Participants took desmopressin melt 25 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG003
Desmopressin Melt 50 μg
Participants took desmopressin melt 50 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG004
Desmopressin Melt 100 μg
Participants took desmopressin melt 100 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
Units
Counts
Participants
OG000146
OG001146
OG002140
OG003137
OG004137
Title
Denominators
Categories
Mental Health Summary
Title
Measurements
OG0002.4± 7.98
OG0012.5± 8.06
OG0022.1± 8.16
OG0032.2± 8.39
OG0041.7± 7.73
Physical Health Summary
Title
Measurements
OG0001.0± 6.81
OG0010.5± 6.89
OG0021.2± 7.17
OG003
Participants took desmopressin melt 25 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG003
Desmopressin Melt 50 μg
Participants took desmopressin melt 50 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG004
Desmopressin Melt 100 μg
Participants took desmopressin melt 100 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
Units
Counts
Participants
OG000160
OG001163
OG002158
OG003158
OG004160
Title
Denominators
Categories
All AEs
Title
Measurements
OG00094
OG001100
OG00298
OG003103
OG004110
Deaths
Title
Measurements
OG0000
OG0010
OG0020
OG003
Serious AEs
Title
Measurements
OG0001
OG0012
OG0021
OG003
AEs leading to discontinuation
Title
Measurements
OG0008
OG0016
OG0022
OG003
Severe AEs
Title
Measurements
OG0004
OG0018
OG0025
OG003
Adverse drug reactions (ADRs)
Title
Measurements
OG00062
OG00162
OG00261
OG003
Participants took desmopressin melt 50 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG003
Desmopressin Melt 100 μg
Participants took desmopressin melt 100 μg for 28 days to complete Part I of the study. Participants continued on this dose in study Part II for between 1-6 months (until the database for Part I was locked and treatment was unblinded).
OG004
Placebo to 10 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 10 μg once daily about an hour before bedtime.
OG005
Placebo to 25 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 25 μg once daily about an hour before bedtime.
OG006
Placebo to 50 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 50 μg once daily about an hour before bedtime.
OG007
Placebo to 100 μg
Participants randomized to Placebo in study Part I were re-randomized to receive active desmopressin in Part II. These participants were randomized to receive desmopressin melt 100 μg once daily about an hour before bedtime.