Study Investigating the Impact Burden of Nocturia Using t... | NCT01552343

NCT01552343

Protocol Section

Identification Module

Results Section

Participant Flow Module

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

Status Module

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Female - Desmopressin 25 μg	Experimental	Female participants took 1 tablet of 25 μg every night, approximately 1 hour prior to bedtime (with the intention to sleep), for a period of 1 month.	Drug: Desmopressin
Female - Placebo	Placebo Comparator	Female participants took 1 tablet of placebo every night, approximately 1 hour prior to bedtime (with the intention to sleep), for a period of 1 month.	Drug: Placebo
Male - Desmopressin 75 μg	Experimental	Male participants took 1 tablet 75 μg every night, approximately 1 hour prior to bedtime (with the intention to sleep), for a period of 1 month.	Drug: Desmopressin
Male - Placebo	Placebo Comparator	Male participants took 1 tablet of placebo every night, approximately 1 hour prior to bedtime (with the intention to sleep), for a period of 1 month.	Drug: Placebo

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Desmopressin	Drug	Desmopressin orally disintegrating tablets. Female participants took a 25 μg tablet and male participants took a 75 μg tablet one hour prior to bedtime for one month.

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
The Pearson Correlation Coefficient Between Change From Baseline to Month 1 in Number of Nocturnal Voids and Change From Baseline to Month 1 in Nocturia Impact (NI) Diary Total Score	This outcome is a measure of sensitivity of the NI Diary to change in nocturia. The NI Diary is a 12-item instrument consisting of 11 core items and an overall impact question (Q12). Responses are scored from 0 (no impact) to 4 (highest impact); a lowering of score equals a decrease in impact caused by nocturia. The NI total score is the sum of the 11 core items scores. The NI total score was analyzable only if all 11 items (Q1-Q11) had non-missing responses. Otherwise, it was defined as missing. Missing values were not imputed. The average over the 3-day diary period prior to baseline (Day 1) and Month 1 was used for the overall impact score. The correlation was estimated using Fisher's z transformation, i.e. the NI total score was based on a standardized scale from 0 (lowest impact) to 100 (highest impact). Corresponding adjusted partial correlation coefficients were based on adjustments for mean number of Baseline voids, Baseline NI total score, age, and gender.	Day 1 (Baseline), Month 1
Difference in Mean Change From Baseline to Month 1 in Nocturia Impact (NI) Total Scores and Overall Impact Question for Responders and Non-Responders	This outcome is a measure of sensitivity of the NI Diary to change in nocturia. The NI Diary is a 12-item instrument consisting of 11 core items and an overall impact question (Q12). The NI total score is defined as the sum of the 11 core items scores. The overall impact question (Q12) and the NI total score were transformed using Fisher's z transformation, i.e. the scores were based on a standardized scale from 0 (lowest impact) to 100 (highest impact). The difference in mean change in NI total score for subjects who experienced a reduction from baseline of <33% in nocturnal voids at the Month 1 visit (non-responders) versus those with a reduction in nocturnal voids from Baseline of ≥33% (responders) was estimated.	Day 1 (Baseline), Month 1
Cohen's D Effect Size in Responsiveness in the Nocturia Impact (NI) Total Scores and Overall Impact Question as Measured From Baseline (Day 1) to Month 1	The responsiveness of the NI Diary was measured with Cohen's D effect size. The effect size was calculated for active treatment versus placebo, based on change from Baseline to Month 1. The effect size was evaluated as "small," "medium," or "large" if D was <=0.35, >0.35 - 0.65, or >0.65, respectively. Mean values are the Cohen's D effect size. Standard deviation is the pooled standard deviation.

Secondary Outcomes

Measure	Description	Time Frame
Internal Consistency of the Nocturia Impact (NI) Total Score for Each Day NI Diaries Were Completed Assessed as Cronbach's Alpha Values	Cronbach's alpha (CA) is a measure of the internal consistency of the Nocturia Impact (NI) Total scores. Higher scores indicate a more reliable (precise) instrument. A value of 0.70 set as the benchmark for declaring the scale as internally consistent. Cronbach's alpha was assessed for each of the three consecutive days NI diaries were completed during screening (Day -20 to Day -18), baseline (Day -2 to Day 1) and Month 1 (Day 28 to Day 30).

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Written informed consent prior to performance of any study-related activity
18 years of age (at the time of written consent) or older
Previous participation in FE992026 CS40 or FE992026 CS41 with a completion ≥ 30 days prior to Screening. The subject should have responded to active treatment during FE992026 CS40 or FE992026 CS41 or if he/she received placebo during these two studies he/she should have been a non-responder.
At least two nocturnal voids every night in two consecutive 3-day periods during the screening period (as determined by the two night-time voiding diaries dispensed at Visit 1 and collected at Visit 2)

Exclusion Criteria:

Chronic prostatitis (males)/chronic pelvic pain syndrome (CPPS)
Suspicion of bladder outlet obstruction (BOO) or a urine flow of < 5 mL/s as confirmed by uroflowmetry performed after suspicion of BOO
Surgical treatment, including transurethral resection, for BOO or benign prostatic hyperplasia (males) within the past six months
Urinary retention or a post void residual volume > 150 mL for females and > 250 mL for males as confirmed by bladder ultrasound performed after suspicion of urinary retention
Central or nephrogenic diabetes insipidus
Syndrome of inappropriate antidiuretic hormone
Current or a history of urologic malignancies e.g. bladder cancer
Genito-urinary tract pathology e.g. infection or stone in the bladder and urethra causing symptoms
Neurogenic detrusor activity (detrusor overactivity)
Suspicion or evidence of cardiac failure
Chronic prostatitis (males)/chronic pelvic pain syndrome (CPPS)
Uncontrolled hypertension
Uncontrolled diabetes mellitus
Hyponatraemia: serum sodium level must be within normal limits
Renal insufficiency: Serum creatinine must be within normal limits and estimated glomerular filtration rate must be ≥ 50 mL/min
Hepatic and/or biliary diseases: Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) levels must not be more than twice the upper limit of normal range. Total bilirubin level must not be > 1.5 mg/dL
History of obstructive sleep apnea
Treatment with another investigational product (except desmopressin) within three months prior to screening and throughout the study
Concomitant treatment with loop diuretics (furosemide, torsemide, ethacrynic acid)
Pregnancy, breastfeeding, or an intention of becoming pregnant during the period of the clinical study. Female subjects of reproductive age must have documentation of a reliable method of contraception. All pre-and perimenopausal female subjects have to perform pregnancy tests. Amenorrhea of > 12 months duration based on the reported date of the last menstrual period is sufficient documentation of post-menopausal status and does not require a pregnancy test
Known alcohol or substance abuse
Work or lifestyle that may interfere with regular night-time sleep e.g. shiftworkers 23. Any other medical condition, laboratory abnormality, psychiatric condition, mental incapacity, or language barrier which, in the judgment of the Investigator, would impair participation in the study

Name	Affiliation	Role
Clinical Development Support	Ferring Pharmaceuticals	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
South Florida Medical Research		Aventura	Florida		United States
Avail Clinical Research, LLC

PubMed Identifier	Type	Citation	Retractions
25236993	Result	Holm-Larsen T, Andersson F, van der Meulen E, Yankov V, Rosen RC, Norgaard JP. The Nocturia Impact Diary: a self-reported impact measure to complement the voiding diary. Value Health. 2014 Sep;17(6):696-706. doi: 10.1016/j.jval.2014.06.007. Epub 2014 Aug 20.

ID	Title	Description
FG000	Placebo	Female and male participants took 1 tablet of placebo every night, approximately 1 hour prior to bedtime (with the intention to sleep), for a period of 1 month.
FG001	Desmopressin	Female participants took 1 desmopressin 25 μg tablet and male participants took 1 tablet 75 μg every night, approximately 1 hour prior to bedtime (with the intention to sleep), for a period of 1 month.

ID	Title	Description
BG000	Placebo	Female and male participants took 1 tablet of placebo every night, approximately 1 hour prior to bedtime (with the intention to sleep), for a period of 1 month.
BG001	Desmopressin

Units	Counts
Participants	BG000

Title	Description	Population Description	Parameter Type	Dispersion Type	Unit of Measure	Calculate Percentage	Denominator Units Selected	Denominators	Classes
Age, Customized			Number

Outcome Measures Module

Outcome Measures

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	The Pearson Correlation Coefficient Between Change From Baseline to Month 1 in Number of Nocturnal Voids and Change From Baseline to Month 1 in Nocturia Impact (NI) Diary Total Score	This outcome is a measure of sensitivity of the NI Diary to change in nocturia. The NI Diary is a 12-item instrument consisting of 11 core items and an overall impact question (Q12). Responses are scored from 0 (no impact) to 4 (highest impact); a lowering of score equals a decrease in impact caused by nocturia. The NI total score is the sum of the 11 core items scores. The NI total score was analyzable only if all 11 items (Q1-Q11) had non-missing responses. Otherwise, it was defined as missing. Missing values were not imputed. The average over the 3-day diary period prior to baseline (Day 1) and Month 1 was used for the overall impact score. The correlation was estimated using Fisher's z transformation, i.e. the NI total score was based on a standardized scale from 0 (lowest impact) to 100 (highest impact). Corresponding adjusted partial correlation coefficients were based on adjustments for mean number of Baseline voids, Baseline NI total score, age, and gender.	Full analysis set. Study results are reported as per the pre-planned statistical analysis plan on combined treatment groups. The study is a psychometric evaluation of a new PRO tool and is not designed to show treatment difference between active and placebo.	Posted		Number	95% Confidence Interval	correlation coefficient		Day 1 (Baseline), Month 1

Adverse Events Module

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	Placebo	Female and male participants took 1 tablet of placebo every night, approximately 1 hour prior to bedtime (with the intention to sleep), for a period of 1 month.

ID	Term
D059411	Lower Urinary Tract Symptoms
D020924	Urological Manifestations
D012816	Signs and Symptoms
D013568	Pathological Conditions, Signs and Symptoms

ID	Term
D001127	Arginine Vasopressin
D014667	Vasopressins
D010909	Pituitary Hormones, Posterior
D010907	Pituitary Hormones