Efficacy and Safety of Desmopressin Melt for the Treatment of Nocturia
- Registration Number
- NCT00477490
- Lead Sponsor
- Ferring Pharmaceuticals
- Brief Summary
The purpose of this study is to investigate the efficacy and safety of several doses of the melt formulation of desmopressin in a broad population of adult patients with nocturia.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 799
Not provided
Males:
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Clinical suspicion of bladder outlet obstruction and/or urine flow < 5 ml/s. If medical history and/or physical examination suggest bladder outlet obstruction, uroflowmetry should be performed to confirm the diagnosis
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Surgical treatment for bladder outlet obstruction/benign prostatic hyperplasia performed within the past 6 months
Females:
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Pregnancy. Females of reproductive age must have documentation of a reliable method of contraception.
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Use of pessary for pelvic prolapse.
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Unexplained pelvic mass.
Males and Females:
-
Clinical suspicion of urinary retention and/or post void residual volume > 150 ml. If medical history and/or physical examination suggest urinary retention, bladder ultrasound or catheterization should be performed to confirm the diagnosis.
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Current or past urologic malignancy (e.g., bladder cancer, prostate cancer).
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Clinical evidence of current genitourinary tract pathology that could interfere with voiding.
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History of neurogenic detrusor activity (previously known as detrusor hyperreflexia).
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Suspicion or evidence of cardiac failure.
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Uncontrolled hypertension.
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Uncontrolled diabetes mellitus.
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Renal insufficiency. Serum creatinine must be within normal limits and estimated glomerular filtration rate (eGFR) >=60 mL/min.
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Active hepatic and/or biliary disease. Aspartate transaminase (AST) or alanine transaminase (ALT) should not be >2 times the upper limit of normal. Total bilirubin should not be > 1.5 mg/dL.
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Hyponatremia. Serum sodium level must be within normal limits
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Syndrome of Inappropriate antidiuretic hormone secretion (SIADH).
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Diabetes insipidus (urine output > 40 ml/kg over 24 hours) as determined by the 3-day voiding diary.
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Psychogenic or habitual polydipsia
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Obstructive sleep apnea
Other
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Known alcohol or substance abuse
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Work or lifestyle potentially interfering with regular nighttime sleep (e.g., shift workers)
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Previous desmopressin treatment for nocturia.
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Any other medical condition, laboratory abnormality, psychiatric condition, mental incapacity or language barrier that, in the judgment of the investigator, could impair patient participation in the trial.
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Use of loop diuretics (furosemide, torsemide, ethacrynic acid). Other classes of diuretics (thiazides, triamterene, chlorthalidone, amiloride, indapamide) were permitted, either as monotherapy or combination therapy. Subjects using a diuretic were to be encouraged to take it in the morning, if medically feasible.
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Use of any other investigational drug within 30 days of screening.
Concomitant Medications
The following medications are permitted provided that the subject has been on a stable dose for the 3 months prior to the screening date (i.e. treatment has not been initiated or discontinued and there has been no change in dose):
- Alpha-blockers: Cardura (doxazosin); Flomax (tamsulosin); Hytrin (terazosin); Uroxatral (alfuzosin)
- 5 alpha-reductase inhibitors: Avodart (dutasteride); Proscar (finasteride)
- Antispasmodic, anticholinergic, antimuscarinic therapy for overactive bladder: Detrol, Detrol LA (tolterodine); Ditropan, Ditropan XL (oxybutynin); Enablex (darifenacin); Levsin(hyoscyamine); Oxytrol transdermal (oxybutynin); Sanctura (trospium); Vesicare (solifenacin)
- Sedative/hypnotic medications for sleep disorders
- Selective serotonin and mixed norepinephrine/serotonin reuptake inhibitors: Celexa (citalopram); Cymbalta (duloxetine); Effexor (venlafaxine); Lexapro (escitalopram); Paxil(paroxetine); Prozac (fluoxetine); Zoloft (sertraline)
- Chronic use of nonsteroidal anti-inflammatory agents
- Diabinese (chlorpropamide)
- Carbamazepine (carbatrol/tegretol)
- Amiodarone
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo Participants took a placebo 'melt' for 28 days to complete part 1 of the study. In part 2, placebo patients were randomized to one of the other 4 treatment arms based on assignments predetermined at the initial randomization, to receive active desmopressin melt for between 1 and 6 months (until the database for part 1 was locked and treatment was unblinded). desmopressin melt 10 μg desmopressin acetate Participants took desmopressin melt 10 μg for 28 days to complete part 1 of the study. Participants continued on this dose in part 2 of the study for between 1 and 6 months (until the database for part 1 was locked and treatment was unblinded). desmopressin melt 100 μg desmopressin acetate Participants will take desmopressin melt 100 μg for 28 days to complete part 1 of the study. Participants will continue on this dose in part 2 of the study for between 1-6 months (until the database for part 1 is locked and treatment is unblinded). desmopressin melt 50 μg desmopressin acetate Participants took desmopressin melt 50 μg for 28 days to complete part 1 of the study. Participants continued on this dose in part 2 of the study for between 1 and 6 months (until the database for part 1 was locked and treatment was unblinded). desmopressin melt 25 μg desmopressin acetate Participants took desmopressin melt 25 μg for 28 days to complete part 1 of the study. Participants continued on this dose in part 2 of the study for between 1 and 6 months (until the database for part 1 was locked and treatment was unblinded).
- Primary Outcome Measures
Name Time Method Part I: Percentage of Participants With Greater Than 33 Percent Reduction From Baseline in Mean Number of Nocturnal Voids at Week 4 - Week 3 to Day 1 (Baseline), Week 4 (end of Part I) Percentage of participants in each treatment arm that had a greater than 33% reduction from baseline to the end of Part I (week 4) in mean number of nocturnal voids. Nocturnal void data were recorded in participant diaries.
This was the second co-primary outcome.Part I: Change From Baseline in Mean Number of Nocturnal Voids at Week 4 - Week 3 to Day 1 (Baseline), Week 4 (end of Part I) The number of nocturnal voids was the average over 3 consecutive 24-hours periods prior to Day 1 and prior to the week 4 visit as recorded in participant diaries.
This was the first co-primary outcome.
- Secondary Outcome Measures
Name Time Method Part I: Change From Baseline in Total Reported Sleep Time at Week 4 - Week 3 to Day 1 (Baseline), Week 4 (end of Part I) Total sleep time was recorded by participants in study diaries.
Part II: Change From Baseline in Mean Number of Nocturnal Voids to Days 29, 57, 113 and 169 - Week 3 to Day 1 (Baseline), Days 29, 57, 113 and 169 Part II outcomes tested the durability of the effect observed in Part I. The number of nocturnal voids was the average over 3 consecutive 24-hours periods prior to Part I baseline and prior to the Part II visit as recorded in participant diaries.
Part II: Percentage of Participants With Greater Than 33 Percent Reduction From Baseline in Mean Number of Nocturnal Voids to Days 29, 57, 113 and 169 - Week 3 to Day 1 (Baseline), Days 29, 57, 113 and 169 Part II outcomes tested the durability of the effect observed in Part I. Percentage of participants in each treatment arm that had a greater than 33% reduction from baseline to Days 29, 57, 113 and 169 in mean number of nocturnal voids. Nocturnal void data were recorded in participant diaries.
Part I: Change From Baseline in Initial Period of Undisturbed Sleep at Week 4 - Week 3 to Day 1 (Baseline), Week 4 (end of Part I) Initial period of undisturbed sleep was the time elapsed from first falling asleep until either first void or morning arising. Data were captured in patient diaries.
Part I: Change From Baseline in Quality of Life Assessed by The International Consultation on Incontinence Modular Questionnaire - Nocturia (ICIQ-N) at Week 4 - Week 3 to Day 1 (Baseline), Week 4 (end of Part I) The ICIQ-N is a self-administered questionnaire designed to assess the frequency and bother of daytime and nighttime urination. Subjects were asked to rate the degree of bother of daytime urination and nighttime urination on a scale ranging from 0 (not at all) to 10 (a great deal). Higher numbers indicate lower quality of life.
Part I: Change From Baseline in the Two Domain Scores of the Nocturia Quality of Life (NQoL) Questionnaire at Week 4 - Week 3 to Day 1 (Baseline), Week 4 (end of Part I) The NQoL questionnaire is a self-administered 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 twelve core questions are scored on a 0 to 4 scale with higher numbers indicating a better quality of life. Domain summary scores were calculated by transforming the raw score into a 0-100 scale with higher numbers indicating a better quality of life.
Part II: Participants With Treatment-Emergent Adverse Events (AEs) During Study Part II Week 5 up to Day 169 A treatment-emergent adverse event (AE) was any AE occurring during the treatment period or a pretreatment AE that worsened in intensity during the treatment period. The treatment period was the period during which a subject received investigational medicinal product. If a subject discontinued the investigational medicinal product, the date of last dose was the last day of the treatment period.
Part I: Change From Baseline in Quality of Sleep as Assessed by the Global Score of the Pittsburgh Sleep Quality Index (PSQI) at Week 4 - Week 3 to Day 1 (Baseline), Week 4 (end of Part I) The PSQI is a self-administered 19-item questionnaire designed to assess sleep quality and disturbances. The global score ranges from 0 (better sleep quality) to 21 (worse sleep quality). Higher numbers indicate lower quality of life.
Part I: Change From Baseline in the Mental Health Summary and the Physical Health Summary of the Short Form-12 Version 2 (SF-12v2) at Week 4 - Week 3 to Day 1 (Baseline), Week 4 (end of Part I) 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. Data were analyzed using norm-based scoring and summarized along 2 dimensions: Physical Health Summary and Mental Health Summary. Each summary has a range from 0 (poor health) to 100 (highest level of health). Higher numbers indicate better quality of life.
Part I: Participants With Treatment-Emergent Adverse Events (AEs) During Study Part I Day 1 up to Week 4 (end of Part I) A treatment-emergent adverse event (AE) was any AE occurring during the treatment period or a pretreatment AE that worsened in intensity during the treatment period. The treatment period was the period during which a subject received investigational medicinal product. If a subject discontinued the investigational medicinal product, the date of last dose was the last day of the treatment period.
Trial Locations
- Locations (72)
PharmQuest
🇺🇸Greensboro, North Carolina, United States
Advanced Research Associates
🇺🇸Corpus Christi, Texas, United States
Accelovance
🇺🇸Houston, Texas, United States
Women's Clinical Research Center
🇺🇸Seattle, Washington, United States
Investigational site - Medical Professional
🇺🇸Seattle, Washington, United States
Western Clinical Research
🇺🇸Torrance, California, United States
Pierremont Women's Clinic
🇺🇸Shreveport, Louisiana, United States
Medsearch Professional Group
🇺🇸Miami, Florida, United States
San Diego Uro-Research
🇺🇸San Diego, California, United States
Arkansas Primary Care Clinic
🇺🇸Little Rock, Arkansas, United States
Impact Clinical Trials
🇺🇸Beverly Hills, California, United States
Advanced Urology Medical Center
🇺🇸Anaheim, California, United States
California Professional Research
🇺🇸Newport Beach, California, United States
Southeastern Research Group, Inc.
🇺🇸Tallahassee, Florida, United States
Investigational site - Clinical Research
🇨🇦Victoria, British Columbia, Canada
AccuMed Research Associates
🇺🇸Garden City, New York, United States
Philadelphia Clinical Research, LLC
🇺🇸Philadelphia, Pennsylvania, United States
Connecticut Clinical Research Center, LLC
🇺🇸Middlebury, Connecticut, United States
Urology Group of New Mexico, PC
🇺🇸Albuquerque, New Mexico, United States
Southern Interior Medical Center
🇨🇦Kelowna, British Columbia, Canada
Upstate Urology
🇺🇸Albany, New York, United States
Can-Med Clinical Research Inc.
🇨🇦Victoria, British Columbia, Canada
Seattle Urology Research Center
🇺🇸Seattle, Washington, United States
University Medical Group
🇺🇸Greenville, South Carolina, United States
The Fe/Male Health Centres
🇨🇦Oakville, Ontario, Canada
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
Urology Associates PC
🇺🇸Denver, Colorado, United States
Downtown Women's Health Care
🇺🇸Denver, Colorado, United States
Urology San Antonio Research, PA
🇺🇸San Antonio, Texas, United States
Radiant Research San Antonio
🇺🇸San Antonio, Texas, United States
West Coast Clinical Research
🇺🇸Tarzana, California, United States
Tampa Bay Urology
🇺🇸Tampa, Florida, United States
Virginia Urology Center
🇺🇸Richmond, Virginia, United States
Investigational site - PC
🇺🇸Dunwoody, Georgia, United States
Radiant Research, Kansas City
🇺🇸Overland park, Kansas, United States
University Urology Associates
🇺🇸New York, New York, United States
Urologic Consultants of SE PA
🇺🇸Bala Cynwyd, Pennsylvania, United States
Palmetto Medical Research
🇺🇸Mt. Pleasant, South Carolina, United States
Urology of Virginia PC
🇺🇸Virginia Beach, Virginia, United States
Investigational site - Professional Corporation
🇨🇦Fredericton, New Brunswick, Canada
Atlantic Urology Medical Group
🇺🇸Long Beach, California, United States
Women's Medical Research Group, LLC
🇺🇸Clearwater, Florida, United States
South Florida Medical Research
🇺🇸Aventura, Florida, United States
Genitourinary Surgical Consultants
🇺🇸Denver, Colorado, United States
Benchmark Research
🇺🇸Metairie, Louisiana, United States
FutureCare Studies, Inc.
🇺🇸Springfield, Massachusetts, United States
Regional Urology, LLC
🇺🇸Shreveport, Louisiana, United States
Sunrise Medical Research
🇺🇸Plantation, Florida, United States
New Hanover Medical Research
🇺🇸Wilmington, North Carolina, United States
Radiant Research, Greer
🇺🇸Greer, South Carolina, United States
Ferring Pharmaceutical Inc
🇺🇸Suffern, New York, United States
Holston Medical Group
🇺🇸Kingsport, Tennessee, United States
Lawrenceville Urology
🇺🇸Lawrenceville, New Jersey, United States
Guelph Urology Associates
🇨🇦Guelph, Ontario, Canada
AdvanceMed Research
🇺🇸Lawrenceville, New Jersey, United States
Southeastern Medical Research Institute
🇺🇸Columbus, Georgia, United States
Investigational site
🇨🇦North Bay, Ontario, Canada
Radiant Research Inc.
🇺🇸St. Louis, Missouri, United States
Women's Clinic of Lincoln, P.C
🇺🇸Lincoln, Nebraska, United States
Morristown Urology
🇺🇸Morristown, New Jersey, United States
Northeast Urology Research
🇺🇸Concord, North Carolina, United States
Carolina Urologic Research Center
🇺🇸Myrtle Beach, South Carolina, United States
Radiant Research - Akron
🇺🇸Mogadore, Ohio, United States
Investigational site - Adult & Pediatric Urology
🇺🇸Carmel, New York, United States
Brantford Urology Research
🇨🇦Brantford, Ontario, Canada
Advanced Clinical Concepts
🇺🇸Reading, Pennsylvania, United States
The Male/Female Health and Reserach
🇨🇦Barrie, Ontario, Canada
Health Central Women's Care
🇺🇸Dallas, Texas, United States
Regional Medical Center and Diagnostic
🇺🇸Humble, Texas, United States
Sunnybrook Health Sciences Centre
🇨🇦Toronto, Ontario, Canada
Piedmont Medical Research Associates
🇺🇸Winston-Salem, North Carolina, United States
Radiant Research
🇺🇸Philadelphia, Pennsylvania, United States