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An Extension Study Investigating the Efficacy and Safety of a Fast-Dissolving ("Melt") Formulation of Desmopressin for the Treatment of Nocturia in Adults

Primary Purpose

Nocturia

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Desmopressin Melt

About this trial

This is an interventional treatment trial for Nocturia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Written informed consent prior to the performance of any study-related activity.
Was randomized into Part II of Protocol FE992026 CS29 (NCT00477490), entitled "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" and have completed at least Visit 3E in Part II (Day 15).

Exclusion Criteria:

Patients using loop diuretics (furosemide, torsemide, ethacrynic acid).

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Arm Label

Desmopressin Melt 10 μg

Desmopressin Melt 25 μg

Desmopressin Melt 50 μg

Desmopressin Melt 100 μg

Arm Description

Participants received desmopressin melt 10 μg once a day, placed under the tongue one hour before bedtime until they were re-randomized to one of the other doses of desmopressin Melt (25 μg, 50 μg, or 100 μg).

Participants received desmopressin melt 25 μg once a day, placed under the tongue one hour before bedtime for up to 2 years and 2.5 months.

Participants received desmopressin melt 50 μg once a day, placed under the tongue one hour before bedtime for up to 2 years and 2.5 months.

Participants received desmopressin melt 100 μg once a day, placed under the tongue one hour before bedtime for up to 2 years and 2.5 months.

Outcomes

Primary Outcome Measures

Change From Baseline in Mean Number of Nocturnal Voids

Participants completed a voiding diary for 3 consecutive 24-hour periods prior to the study visit in which they recorded each nocturnal urination (void). The mean number of voids per night was the average number of voids from the 3-day diary. Baseline refers to Baseline of Study CS29 and the number of weeks represents the total exposure to study drug. Participants in the 10μg arm are included only until the time of dose escalation.

Percentage of Participants With a Greater Than 33% Reduction in the Mean Number of Nocturnal Voids

Percentage of participants with >33% reduction from Baseline in the mean number of nocturnal urinations per night, calculated from the 3-day voiding diary completed prior to each study visit. Participants in the 10μg arm are included only until the time of dose escalation.

Change From Baseline in Initial Period of Undisturbed Sleep

Participants completed a sleep diary on 3 consecutive mornings prior to each study visit, from which the initial period of undisturbed sleep was calculated and averaged for the 3 days. The Initial Period of Undisturbed Sleep is the time elapsed from bedtime to either first void or morning arising minus the minutes it took to fall asleep. Baseline refers to Baseline of Study CS29 and the number of weeks represents the total exposure to study drug. Participants in the 10μg arm are included only until the time of dose escalation.

Secondary Outcome Measures

Change From Baseline in Total Sleep Time

Participants completed a sleep diary on 3 consecutive mornings prior to each study visit, from which the total sleep time was calculated and averaged for the 3 days. Baseline refers to Baseline of Study CS29 and the number of weeks represents the total exposure to study drug. Participants in the 10μg arm are included only until the time of dose escalation.

Change From Baseline in International Consultation on Incontinence Modular Questionnaire - Nocturia (ICIQ-N) Nighttime Urination Bother Score

The ICIQ-N is a self-administered 4-item questionnaire designed to assess the frequency and bother of daytime and nighttime urination. To assess nighttime urination bother, participants were asked to rate the degree of bother of nighttime urination by answering the question "Night time urination: How much does this bother you?" on a scale ranging from 0 (not at all) to 10 (a great deal). Higher numbers indicate greater bother. Participants in the 10μg arm are included only until the time of dose escalation.

Change From Baseline in Nocturia Quality of Life (NQoL) Overall Score

The NQoL is a self-administered 13-item 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 (which is not included in the overall score). The 12 core items are scored on a 0 to 4 scale, and the overall score is calculated by transforming the raw score into a 0-100 scale with higher numbers indicating better impact on quality of life. Participants in the 10μg arm are included only until the time of dose escalation.

Change From Baseline in NQoL Bother/Concern Domain Score

The NQoL is a self-administered 13-item 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 12 core items are scored on a 0 to 4 scale with higher numbers indicating a better quality of life. The bother/concern domain summary score is calculated by transforming the raw score into a 0-100 scale with higher numbers indicating a better impact on quality of life. Participants in the 10μg arm are included only until the time of dose escalation.

Change From Baseline in Nocturia Quality of Life (NQoL) Sleep/Energy Domain Score

The NQoL is a self-administered 13-item 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 12 core items are scored on a 0 to 4 scale with higher numbers indicating a better quality of life. The sleep/energy domain summary score is calculated by transforming the raw score into a 0-100 scale with higher numbers indicating a better impact on quality of life. Participants in the 10μg arm are included only until the time of dose escalation.

Change From Baseline in the Nocturia Quality of Life (NQoL) Global Quality of Life Score

The NQoL is a self-administered 13-item 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 global QoL question is scored on a scale ranging from 0 (not at all) to 10 (a great deal). Higher numbers indicate better impact on quality of life. Participants in the 10μg arm are included only until the time of dose escalation.

Change From Baseline in Pittsburgh Sleep Quality Index (PSQI) Global Score

The PSQI is a self-administered 19-item questionnaire designed to assess sleep quality and disturbances. The 19 individual items are scored on an evenly weighted 0 to 3 scale and generate 7 component scores: subjective sleep quality, sleep latency, sleep duration, habitual sleep efficiency, sleep disturbances, use of sleeping medication, and daytime dysfunction. The sum of scores for these 7 components yields 1 global score ranging from 0 to 21. Higher numbers indicate greater sleep disturbance. Participants in the 10μg arm are included only until the time of dose escalation.

Change From Baseline in the Short Form-12, Version 2 (SF-12v2) Mental Component Summary Score

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 spanning 8 domains: physical functioning, role function-physical, role function-emotional, bodily pain, general health, vitality, social functioning, and mental health. These scales are combined to create 2 summary measures: the Physical Health Summary and Mental Health Summary. The Mental Health Summary score ranges from 0 to 100, where higher numbers indicate better quality of life. Participants in the 10μg arm are included only until the time of dose escalation.

Change From Baseline in the Short Form-12, Version 2 (SF-12v2) Physical Component Summary Score

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 spanning 8 domains: physical functioning, role function-physical, role function-emotional, bodily pain, general health, vitality, social functioning, and mental health. These scales are combined to create 2 summary measures: the Physical Health Summary and Mental Health Summary. The Physical Health Summary score ranges from 0 to 100, where higher numbers indicate better quality of life. Participants in the 10μg arm are included only until the time of dose escalation.

Participants With Treatment-Emergent Adverse Events (AEs)

An AE was any untoward medical occurrence that did not necessarily have a causal relationship with the study drug. An adverse drug reaction (ADR) was an AE evaluated by the Investigator as being probably or possibly causally related to treatment with the study drug. A serious AE (SAE) was any event that resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity or congenital anomaly/birth defect or was an important medical event that could have jeopardized the patient's safety or required medical or surgical intervention to prevent 1 of the outcomes listed above. The intensity of an AE was defined as severe if it resulted in the inability to work or perform usual activities.

Full Information

NCT ID

NCT00615836

First Posted

January 18, 2008

Last Updated

November 11, 2015

Sponsor

Ferring Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT00615836

Brief Title

An Extension Study Investigating the Efficacy and Safety of a Fast-Dissolving ("Melt") Formulation of Desmopressin for the Treatment of Nocturia in Adults

Official Title

A Multi-Center Extension Study Investigating the Long Term Efficacy and Safety of a Fast-Dissolving ("Melt") Formulation of Desmopressin for the Treatment of Nocturia in Adults

Study Type

Interventional

2. Study Status

Record Verification Date

November 2015

Overall Recruitment Status

Completed

Study Start Date

December 2007 (undefined)

Primary Completion Date

May 2010 (Actual)

Study Completion Date

May 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Ferring Pharmaceuticals

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

The purpose of this study was to investigate the long term efficacy and safety of several doses of the Melt formulation of desmopressin in a broad population of adult patients with nocturia.

Detailed Description

FE992026 CS31 was a multicenter open-label extension study for patients who were enrolled in Study FE992026 CS29 (NCT00477490) and had completed at least Visit 3E in Part II of that study. The CS29 study was structured into 2 double-blind parts (Part I and Part II). In Part I, the initial 28-day treatment period, participants were randomly assigned to 1 of 5 treatment groups: placebo or desmopressin Melt 10 μg, 25 μg, 50 μg, or 100 μg. Immediately upon completion of Part I of the study, all participants on active treatment continued into Part II on the same treatment for approximately 1 to 6 months. Participants assigned to placebo in Part I were randomly assigned to 1 of the 4 active treatments in Part II, based on re-randomization predetermined at the initial randomization (to maintain the blind). Part II began at the final visit for Part I and continued until the database for Part I was locked. Therefore, treatment duration for Part II varied between 1 and 6 months, depending upon when the participant entered. Upon completion of Part II of CS29, participants were given the option to participate in the open-label extension study (CS31). During CS31, each participant assigned to the 10 μg dose was switched to a higher dose in an open-label manner among the remaining 3 higher doses.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Nocturia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

554 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Desmopressin Melt 10 μg

Arm Type

Experimental

Arm Description

Arm Title

Desmopressin Melt 25 μg

Arm Type

Experimental

Arm Description

Participants received desmopressin melt 25 μg once a day, placed under the tongue one hour before bedtime for up to 2 years and 2.5 months.

Arm Title

Desmopressin Melt 50 μg

Arm Type

Experimental

Arm Description

Participants received desmopressin melt 50 μg once a day, placed under the tongue one hour before bedtime for up to 2 years and 2.5 months.

Arm Title

Desmopressin Melt 100 μg

Arm Type

Experimental

Arm Description

Participants received desmopressin melt 100 μg once a day, placed under the tongue one hour before bedtime for up to 2 years and 2.5 months.

Intervention Type

Drug

Intervention Name(s)

Desmopressin Melt

Other Intervention Name(s)

Minirin® Melt, Nocturin®, FE992026

Intervention Description

An orally disintegrating tablet of desmopressin administered under the tongue (sublingually), without water.

Primary Outcome Measure Information:

Title

Change From Baseline in Mean Number of Nocturnal Voids

Description

Time Frame

Baseline of Study CS29 and Weeks 8, 12, 20, 28, 52-56, 72-76, and 92-96.

Title

Percentage of Participants With a Greater Than 33% Reduction in the Mean Number of Nocturnal Voids

Description

Time Frame

Baseline of Study CS29 and Weeks 8, 12, 20, 28, 52-56, 72-76, and 92-96.

Title

Change From Baseline in Initial Period of Undisturbed Sleep

Description

Time Frame

Baseline of Study CS29 and Weeks 8, 12, 20, 28, 52-56, 72-76, and 92-96.

Secondary Outcome Measure Information:

Title

Change From Baseline in Total Sleep Time

Description

Time Frame

Baseline of Study CS29 and Weeks 8, 12, 20, 28, 52-56, 72-76, and 92-96.

Title

Change From Baseline in International Consultation on Incontinence Modular Questionnaire - Nocturia (ICIQ-N) Nighttime Urination Bother Score

Description

Time Frame