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

Nocturia Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00615836
• Other study ID #: FE992026 CS31
• Status: Completed
• Phase: Phase 3
• First received: January 18, 2008
• Last updated: November 11, 2015
• Start date: December 2007
• Est. completion date: May 2010

Study information

• Verified date: November 2015
• Source: Ferring Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationCanada: Health Canada
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 554
• Est. completion date: May 2010
• Est. primary completion date: May 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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).

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Nocturia

Intervention

Drug:
• Desmopressin Melt
An orally disintegrating tablet of desmopressin administered under the tongue (sublingually), without water.

Locations

Country	Name	City	State
Canada	The Male/Female Health and Research	Barrie	Ontario
Canada	Brantford Urology Research	Brantford	Ontario
Canada	Investigational site - Professional Corporation	Fredericton	New Brunswick
Canada	Guelph Urology Associates	Guelph	Ontario
Canada	Southern Interior Medical Research Inc.	Kelowna	British Columbia
Canada	Investigational site	North Bay	Ontario
Canada	The Fe/Male Health Centres	Oakville	Ontario
Canada	Can-Med Clinical Research Inc.	Victoria	British Columbia
Canada	Investigational site - Clinical Research	Victoria	British Columbia
United States	Urology Group of New Mexico, PC	Albuquerque	New Mexico
United States	Advanced Urology Medical Center	Anaheim	California
United States	South Florida Medical Research	Aventura	Florida
United States	Urologic Consultants of SE PA	Bala Cynwyd	Pennsylvania
United States	Impact Clinical Trials	Beverly Hills	California
United States	Investigational site - Adult & Pediatric Urology	Carmel	New York
United States	Radiant Research, Inc	Chicago	Illinois
United States	Radiant Research Inc.	Cincinnati	Ohio
United States	Women's Health Research Group, LLC	Clearwater	Florida
United States	Southeastern Medical Research Institute	Columbus	Georgia
United States	Advanced Research Associates	Corpus Christi	Texas
United States	Downtown Women's Health Care	Denver	Colorado
United States	Genitourinary Surgical Consultants	Denver	Colorado
United States	Urology Associates PC	Denver	Colorado
United States	Investigational site	Dunwoody	Georgia
United States	Radiant Research, Minneapolis	Edina	Minnesota
United States	Central Jersey Medical Research Center	Elizabeth	New Jersey
United States	AccuMed Research Associates	Garden City	New York
United States	PharmQuest	Greensboro	North Carolina
United States	Radiant Research, Greer	Greer	South Carolina
United States	Accelovance	Houston	Texas
United States	Investigational site - NationsMed Clinical Research	Houston	Texas
United States	Regional Medical Center and Diagnostic	Humble	Texas
United States	Holston Medical Group	Kingsport	Tennessee
United States	Sheldon J Freedman Ltd	Las Vegas	Nevada
United States	Lawrenceville Urology, P.A. DBA	Lawrenceville	New Jersey
United States	Women's Clinic of Lincoln, P.C.	Lincoln	Nebraska
United States	Arkansas Primary Care Clinic, PA	Little Rock	Arkansas
United States	Benchmark Research	Metairie	Louisiana
United States	Connecticut Clinical Research Center, LLC	Middlebury	Connecticut
United States	Radiant Research - Akron	Mogadore	Ohio
United States	Palmetto Medical Research	Mt. Pleasant	South Carolina
United States	Carolina Urologic Research Center	Myrtle Beach	South Carolina
United States	University Urology Associates	New York	New York
United States	California Professional Research	Newport Beach	California
United States	Upstate Urology	NY	New York
United States	Radiant Research, Kansas City	Overland Park	Kansas
United States	Accelovance	Peoria	Illinois
United States	Philadelphia Clinical Research, LLC	Philadelphia	Pennsylvania
United States	Radiant Research - St. Petersburg	Pinellas Park	Florida
United States	Sunrise Medical Research	Plantation	Florida
United States	Hudson Valley Urology, PC	Poughkeepsie	New York
United States	Virginia Urology	Richmond	Virginia
United States	IMED Research, P.A.	San Antonio	Texas
United States	Innovative Clinical Trials	San Antonio	Texas
United States	Radiant Research San Antonio	San Antonio	Texas
United States	San Diego Uro-Reseach	San Diego	California
United States	Radiant Research	Santa Rosa	California
United States	Radiant Research	Scottsdale	Arizona
United States	Seattle Urology Research Center	Seattle	Washington
United States	Women's Clinical Research Center	Seattle	Washington
United States	Regional Urology, LLC	Shreveport	Louisiana
United States	FutureCare Studies, Inc.	Springfield	Massachusetts
United States	Radiant Research, Inc.	St. Louis	Missouri
United States	NationsMed	Stafford	Texas
United States	Radiant Research	Stuart	Florida
United States	Tampa Bay Urology	Tampa	Florida
United States	West Coast Clinical Research	Tarzana	California
United States	Western Clinical Research	Torrance	California
United States	Urology of Virginia PC	Virginia Beach	Virginia
United States	Radiant Research	West Palm Beach	Florida
United States	Advanced Clinical Concepts	West Readings	Pennsylvania
United States	New Hanover Medical Research	Wilmington	North Carolina
United States	Piedmont Medical Research Associates	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Ferring Pharmaceuticals

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Baseline of Study CS29 and Weeks 8, 12, 20, 28, 52-56, 72-76, and 92-96.	No
Primary	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.	Baseline of Study CS29 and Weeks 8, 12, 20, 28, 52-56, 72-76, and 92-96.	No
Primary	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.	Baseline of Study CS29 and Weeks 8, 12, 20, 28, 52-56, 72-76, and 92-96.	No
Secondary	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.	Baseline of Study CS29 and Weeks 8, 12, 20, 28, 52-56, 72-76, and 92-96.	No
Secondary	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.	Baseline of Study CS29, Week 16, Visit 12 (approximately 56-78 weeks total study time) and End of Study (up to a maximum of 35 months)	No
Secondary	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.	Baseline of Study CS29, Week 16, Visit 12 (approximately 56-78 weeks total study time) and End of Study (up to a maximum of 35 months)	No
Secondary	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.	Baseline of Study CS29, Week 16, Visit 12 (approximately 56-78 weeks total study time) and End of Study (up to a maximum of 35 months)	No
Secondary	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.	Baseline of Study CS29, Week 16, Visit 12 (approximately 56-78 weeks total study time) and End of Study (up to a maximum of 35 months)	No
Secondary	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.	Baseline of Study CS29, Week 16, Visit 12 (approximately 56-78 weeks total study time) and End of Study (up to a maximum of 35 months)	No
Secondary	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.	Baseline of Study CS29, Week 16, Visit 12 (approximately 56-78 weeks total study time) and End of Study (up to a maximum of 35 months)	No
Secondary	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.	Baseline of Study CS29, Week 16, Visit 12 (approximately 56-78 weeks total study time) and End of Study (up to a maximum of 35 months)	No
Secondary	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.	Baseline of Study CS29, Week 16, Visit 12 (approximately 56-78 weeks total study time) and End of Study (up to a maximum of 35 months)	No
Secondary	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.	From first dose of study drug in Study CS29 until the end of study CS31 (up to 35 months).	No