Trial · Interventional

CompletedPhase 3Results postedRegistry data

Study Investigating the Impact Burden of Nocturia Using the Nocturia Impact Diary

Short name IMPACT

NCTNCT01552343Other id000034

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Overview

Status: Completed
Phase: Phase 3
Enrollment: 56 participants (actual)
Sponsor: Ferring Pharmaceuticals · Industry
Design: Randomized · Triple masking
Countries: 1 country
United States
Start: Mar 2012
Primary completion: May 2012
Completion: Jun 2012
First posted: 13 Mar 2012 (estimated)
Last update posted: 11 Jul 2017
Conditions: Nocturia

Timeline

1 trial · 1 completed · 1 with posted results

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

20122013

Ph 3
IMPACT
NCT01552343
Mar 2012 to Jun 2012

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

The purpose of this study is to assess psychometric properties (reliability and validity) of the Nocturia Impact (NI) diary. To assess the association between reduction of number of nocturnal voids and the mean changes in NI scores (sensitivity of the NI total score to change in nocturia). To assess which NI diary items account for the main difference in change in total NI score in treatment versus placebo.

Interventions

DesmopressinDrug
PlaceboDrug

Arms4

Experimental
Female - Desmopressin 25 μg
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.
Placebo comparator
Female - Placebo
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.
Experimental
Male - Desmopressin 75 μg
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.
Placebo comparator
Male - Placebo
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.

Primary outcomes3

Outcomes the sponsor listed as 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
Time frame · Day 1 (Baseline), Month 1
Registry description
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.
Difference in Mean Change From Baseline to Month 1 in Nocturia Impact (NI) Total Scores and Overall Impact Question for Responders and Non-Responders
Time frame · Day 1 (Baseline), Month 1
Registry description
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.
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
Time frame · Day 1 (Baseline), Month 1
Registry description
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(5)

Internal Consistency of the Nocturia Impact (NI) Total Score for Each Day NI Diaries Were Completed Assessed as Cronbach's Alpha Values
Time frame · Screening (Day -20 to Day -18), Baseline (Day -2 to Day 1) and Treatment (Day 28 to Day 30)
Registry description
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).
Construct Validity For the Nocturia Impact (NI) Total Scores and Overall Impact Question (Q12) for Participants With High/Low Number of Nocturnal Voids
Time frame · Screening (Day -20), Baseline (Day 1)
Registry description
The known group validity was assessed by comparing participants who experienced ≥3 nocturnal voids to those who experienced \<3 nocturnal voids, using the average over 3 days for the Screening and Baseline diaries. Results are reported for the NI Total Scores and the Overall Impact Question (Q12). 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).
Change From Baseline to Month 1 on Nocturia Impact (NI) Total Score
Time frame · Baseline (Day -2 to Day 1), Treatment (Day 28-30)
Registry description
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); the NI total score is the sum of the 11 core items scores (range of 0-44) which is then transformed to a 0-100 scale (high score indicates high impact). 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. Negative change from baseline scores indicate a decrease in impact caused by nocturia.
Minimum Post-Treatment Serum Sodium Levels
Time frame · Day 1 up to 1 month
Registry description
Serum sodium levels were monitored since hyponatremia is a potential serious adverse event associated with daily doses of desmopressin. A participant was to be withdrawn from the trial if the serum sodium level was \<=125 mmol/L at any time.
Summary of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame · Day 1 up to 1 month
Registry description
A TEAE was any adverse event occurring after start of treatment and within the time of residual drug effect, i.e. within one day of the last dose of desmopressin.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeNumber· Day 1 (Baseline), Month 1

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

Group	Value (correlation coefficient)
All Participants	0.31

Primary outcomeMean· 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

Group	Value (units on a scale)	Spread
Non-Responders	-2.6	6.8
Responders	-13.3	17.9

Primary outcomeMean· 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

Group	Value (units on a scale)	Spread
All Participants	0.73	14.7

Secondary and other outcome results(5)

Secondary outcomeNumber· Screening (Day -20 to Day -18), Baseline (Day -2 to Day 1) and Treatment (Day 28 to Day 30)

Internal Consistency of the Nocturia Impact (NI) Total Score for Each Day NI Diaries Were Completed Assessed as Cronbach's Alpha Values

Group	Value (ratio of variance)
All Participants	0.915

Secondary outcomeMean· Screening (Day -20), Baseline (Day 1)

Construct Validity For the Nocturia Impact (NI) Total Scores and Overall Impact Question (Q12) for Participants With High/Low Number of Nocturnal Voids

Group	Value (units on a scale)	Spread
< 3 Voids	28.1	20.4
>= 3 Voids	36.7	23.0

Secondary outcomeMean· Baseline (Day -2 to Day 1), Treatment (Day 28-30)

Change From Baseline to Month 1 on Nocturia Impact (NI) Total Score

Group	Value (units on a scale)	Spread
Desmopressin	-8.5	13.7
Placebo	-9.64	17.2

Secondary outcomeNumber· Day 1 up to 1 month

Minimum Post-Treatment Serum Sodium Levels

Group	Value (participants)
Female - Placebo	0
Male - Placebo	0
Female - Desmopressin 25 μg	0
Male - Desmopressin 75 μg	0

Secondary outcomeNumber· Day 1 up to 1 month

Summary of Participants With Treatment-Emergent Adverse Events (TEAEs)

Group	Value (participants)
Female - Placebo	2
Male - Placebo	1
Female - Desmopressin 25 μg	2
Male - Desmopressin 75 μg	0

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
Group	Deaths	Serious events	Other events
PlaceboFemale 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.	–	0 / 29	0 / 29
DesmopressinFemale 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.	–	0 / 27	0 / 27

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Other registry references

References listed at the registry that are not in PepBank. They link to PubMed.

ResultHolm-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. PMID 25236993

Eligibility

Who could join, as posted.

Sex: All
Age: from 18 Years
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

Inclusion Criteria: 1. Written informed consent prior to performance of any study-related activity 2. 18 years of age (at the time of written consent) or older 3. 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. 4. 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: 1. Chronic prostatitis (males)/chronic pelvic pain syndrome (CPPS) 2. Suspicion of bladder outlet obstruction (BOO) or a urine flow of \< 5 mL/s as confirmed by uroflowmetry performed after suspicion of BOO 3. Surgical treatment, including transurethral resection, for BOO or benign prostatic hyperplasia (males) within the past six months 4. 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 5. Central or nephrogenic diabetes insipidus 6. Syndrome of inappropriate antidiuretic hormone 7. Current or a history of urologic malignancies e.g. bladder cancer 8. Genito-urinary tract pathology e.g. infection or stone in the bladder and urethra causing symptoms 9. Neurogenic detrusor activity (detrusor overactivity) 10. Suspicion or evidence of cardiac failure 11. Chronic prostatitis (males)/chronic pelvic pain syndrome (CPPS) 12. Uncontrolled hypertension 13. Uncontrolled diabetes mellitus 14. Hyponatraemia: serum sodium level must be within normal limits 15. Renal insufficiency: Serum creatinine must be within normal limits and estimated glomerular filtration rate must be ≥ 50 mL/min 16. 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 17. History of obstructive sleep apnea 18. Treatment with another investigational product (except desmopressin) within three months prior to screening and throughout the study 19. Concomitant treatment with loop diuretics (furosemide, torsemide, ethacrynic acid) 20. 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 21. Known alcohol or substance abuse 22. 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

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