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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Ferring Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00615836
First received: January 18, 2008
Last updated: November 11, 2015
Last verified: November 2015
  Purpose
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.

Condition Intervention Phase
Nocturia
Drug: Desmopressin Melt
Phase 3

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

Resource links provided by NLM:


Further study details as provided by Ferring Pharmaceuticals:

Primary Outcome Measures:
  • Change From Baseline in Mean Number of Nocturnal Voids [ Time Frame: Baseline of Study CS29 and Weeks 8, 12, 20, 28, 52-56, 72-76, and 92-96. ] [ Designated as safety issue: No ]

    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 [ Time Frame: Baseline of Study CS29 and Weeks 8, 12, 20, 28, 52-56, 72-76, and 92-96. ] [ Designated as safety issue: No ]

    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 [ Time Frame: Baseline of Study CS29 and Weeks 8, 12, 20, 28, 52-56, 72-76, and 92-96. ] [ Designated as safety issue: No ]

    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 [ Time Frame: Baseline of Study CS29 and Weeks 8, 12, 20, 28, 52-56, 72-76, and 92-96. ] [ Designated as safety issue: No ]

    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 [ Time Frame: 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) ] [ Designated as safety issue: No ]

    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 [ Time Frame: 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) ] [ Designated as safety issue: No ]

    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 [ Time Frame: 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) ] [ Designated as safety issue: No ]

    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 [ Time Frame: 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) ] [ Designated as safety issue: No ]

    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 [ Time Frame: 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) ] [ Designated as safety issue: No ]

    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 [ Time Frame: 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) ] [ Designated as safety issue: No ]

    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 [ Time Frame: 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) ] [ Designated as safety issue: No ]

    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 [ Time Frame: 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) ] [ Designated as safety issue: No ]

    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) [ Time Frame: From first dose of study drug in Study CS29 until the end of study CS31 (up to 35 months). ] [ Designated as safety issue: No ]

    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.



Enrollment: 554
Study Start Date: December 2007
Study Completion Date: May 2010
Primary Completion Date: May 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Desmopressin Melt 10 μg
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).
Drug: Desmopressin Melt
An orally disintegrating tablet of desmopressin administered under the tongue (sublingually), without water.
Other Names:
  • Minirin® Melt
  • Nocturin®
  • FE992026
Experimental: Desmopressin Melt 25 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.
Drug: Desmopressin Melt
An orally disintegrating tablet of desmopressin administered under the tongue (sublingually), without water.
Other Names:
  • Minirin® Melt
  • Nocturin®
  • FE992026
Experimental: Desmopressin Melt 50 μg
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.
Drug: Desmopressin Melt
An orally disintegrating tablet of desmopressin administered under the tongue (sublingually), without water.
Other Names:
  • Minirin® Melt
  • Nocturin®
  • FE992026
Experimental: Desmopressin Melt 100 μg
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.
Drug: Desmopressin Melt
An orally disintegrating tablet of desmopressin administered under the tongue (sublingually), without water.
Other Names:
  • Minirin® Melt
  • Nocturin®
  • FE992026

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.

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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).
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00615836

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Locations
United States, Arizona
Radiant Research
Scottsdale, Arizona, United States, 85251
United States, Arkansas
Arkansas Primary Care Clinic, PA
Little Rock, Arkansas, United States, 72204
United States, California
Advanced Urology Medical Center
Anaheim, California, United States, 92801
Impact Clinical Trials
Beverly Hills, California, United States, 90211
California Professional Research
Newport Beach, California, United States, 92660
San Diego Uro-Reseach
San Diego, California, United States, 92103
Radiant Research
Santa Rosa, California, United States, 95405
West Coast Clinical Research
Tarzana, California, United States, 91356
Western Clinical Research
Torrance, California, United States, 90505
United States, Colorado
Urology Associates PC
Denver, Colorado, United States, 80210
Downtown Women's Health Care
Denver, Colorado, United States, 80218
Genitourinary Surgical Consultants
Denver, Colorado, United States, 80220
United States, Connecticut
Connecticut Clinical Research Center, LLC
Middlebury, Connecticut, United States, 06762
United States, Florida
South Florida Medical Research
Aventura, Florida, United States, 33180
Women's Health Research Group, LLC
Clearwater, Florida, United States, 33759
Radiant Research - St. Petersburg
Pinellas Park, Florida, United States, 33781
Sunrise Medical Research
Plantation, Florida, United States, 33324
Radiant Research
Stuart, Florida, United States, 34996
Tampa Bay Urology
Tampa, Florida, United States, 33607
Radiant Research
West Palm Beach, Florida, United States, 33407
United States, Georgia
Southeastern Medical Research Institute
Columbus, Georgia, United States, 31904
Investigational site
Dunwoody, Georgia, United States, 30338
United States, Illinois
Radiant Research, Inc
Chicago, Illinois, United States, 60654
Accelovance
Peoria, Illinois, United States, 61602
United States, Kansas
Radiant Research, Kansas City
Overland Park, Kansas, United States, 66202
United States, Louisiana
Benchmark Research
Metairie, Louisiana, United States, 70006
Regional Urology, LLC
Shreveport, Louisiana, United States, 71106
United States, Massachusetts
FutureCare Studies, Inc.
Springfield, Massachusetts, United States, 01103
United States, Minnesota
Radiant Research, Minneapolis
Edina, Minnesota, United States, 55435
United States, Missouri
Radiant Research, Inc.
St. Louis, Missouri, United States, 63141
United States, Nebraska
Women's Clinic of Lincoln, P.C.
Lincoln, Nebraska, United States, 68510
United States, Nevada
Sheldon J Freedman Ltd
Las Vegas, Nevada, United States
United States, New Jersey
Central Jersey Medical Research Center
Elizabeth, New Jersey, United States, 07202
Lawrenceville Urology, P.A. DBA
Lawrenceville, New Jersey, United States, 08648
United States, New Mexico
Urology Group of New Mexico, PC
Albuquerque, New Mexico, United States, 87109
United States, New York
Investigational site - Adult & Pediatric Urology
Carmel, New York, United States, 10512
AccuMed Research Associates
Garden City, New York, United States, 11530
University Urology Associates
New York, New York, United States, 10016
Upstate Urology
NY, New York, United States, 12206-1092
Hudson Valley Urology, PC
Poughkeepsie, New York, United States, 12601
United States, North Carolina
PharmQuest
Greensboro, North Carolina, United States, 27408
New Hanover Medical Research
Wilmington, North Carolina, United States, 28401
Piedmont Medical Research Associates
Winston-Salem, North Carolina, United States, 27103
United States, Ohio
Radiant Research Inc.
Cincinnati, Ohio, United States, 45249
Radiant Research - Akron
Mogadore, Ohio, United States, 44260
United States, Pennsylvania
Urologic Consultants of SE PA
Bala Cynwyd, Pennsylvania, United States, 19004
Philadelphia Clinical Research, LLC
Philadelphia, Pennsylvania, United States, 19114
Advanced Clinical Concepts
West Readings, Pennsylvania, United States, 19611
United States, South Carolina
Radiant Research, Greer
Greer, South Carolina, United States, 29651
Palmetto Medical Research
Mt. Pleasant, South Carolina, United States, 29464
Carolina Urologic Research Center
Myrtle Beach, South Carolina, United States, 29572
United States, Tennessee
Holston Medical Group
Kingsport, Tennessee, United States, 37660
United States, Texas
Advanced Research Associates
Corpus Christi, Texas, United States, 78414
Investigational site - NationsMed Clinical Research
Houston, Texas, United States, 77004
Accelovance
Houston, Texas, United States, 77024
Regional Medical Center and Diagnostic
Humble, Texas, United States, 77338
Innovative Clinical Trials
San Antonio, Texas, United States, 78229
Radiant Research San Antonio
San Antonio, Texas, United States, 78229
IMED Research, P.A.
San Antonio, Texas, United States, 78258
NationsMed
Stafford, Texas, United States
United States, Virginia
Virginia Urology
Richmond, Virginia, United States, 23235
Urology of Virginia PC
Virginia Beach, Virginia, United States, 23454
United States, Washington
Seattle Urology Research Center
Seattle, Washington, United States, 98166
Women's Clinical Research Center
Seattle, Washington, United States, 98105
Canada, British Columbia
Southern Interior Medical Research Inc.
Kelowna, British Columbia, Canada, V1Y-2H4
Investigational site - Clinical Research
Victoria, British Columbia, Canada, V8V 3N1
Can-Med Clinical Research Inc.
Victoria, British Columbia, Canada, V8T 5G1
Canada, New Brunswick
Investigational site - Professional Corporation
Fredericton, New Brunswick, Canada, E3B 5B8
Canada, Ontario
The Male/Female Health and Research
Barrie, Ontario, Canada, L4M 7G1
Brantford Urology Research
Brantford, Ontario, Canada, N3R 4N3
Guelph Urology Associates
Guelph, Ontario, Canada, N1H 5J1
Investigational site
North Bay, Ontario, Canada, P1B 7K8
The Fe/Male Health Centres
Oakville, Ontario, Canada, L6H 3P1
Sponsors and Collaborators
Ferring Pharmaceuticals
Investigators
Study Director: Clinical Development Support Ferring Pharmaceuticals
  More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Ferring Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00615836     History of Changes
Other Study ID Numbers: FE992026 CS31 
Study First Received: January 18, 2008
Results First Received: June 23, 2015
Last Updated: November 11, 2015
Health Authority: United States: Food and Drug Administration
Canada: Health Canada

Additional relevant MeSH terms:
Nocturia
Lower Urinary Tract Symptoms
Urological Manifestations
Signs and Symptoms
Deamino Arginine Vasopressin
Hemostatics
Coagulants
Antidiuretic Agents
Natriuretic Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on July 28, 2016