Clinical Trial: Efficacy and Safety of Desmopressin Melt for the Treatment of Nocturia

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: 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") Formulat

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 Summary:
Sponsor: Ferring Pharmaceuticals

Current Primary Outcome:

• Part I: Change From Baseline in Mean Number of Nocturnal Voids at Week 4 [ Time Frame: - 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.

• Part I: Percentage of Participants With Greater Than 33 Percent Reduction From Baseline in Mean Number of Nocturnal Voids at Week 4 [ Time Frame: - 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.

Original Primary Outcome: 1) Change in mean number of nocturnal voids 2) Proportion of subjects with > 33% reduction from baseline in mean number of voids. [ Time Frame: 4 weeks ]

Current Secondary Outcome:

• Part II: Change From Baseline in Mean Number of Nocturnal Voids to Days 29, 57, 113 and 169 [ Time Frame: - 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 [ Time Frame: - 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 Total Reported Sleep Time at Week 4 [ Time Frame: - Week 3 to Day 1 (Baseline), Week 4 (end of Part I) ]
  Total sleep time was recorded by participants in study diaries.
• Part I: Change From Baseline in Initial Period of Undisturbed Sleep at Week 4 [ Time Frame: - 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 [ Time Frame: - 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 [ Time Frame: - 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 I: Change From Baseline in Quality of Sleep as Assessed by the Global Score of the Pittsburgh Sleep Quality Index (PSQI) at Week 4 [ Time Frame: - 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 [ Time Frame: - 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 [ Time Frame: 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.
• Part II: Participants With Treatment-Emergent Adverse Events (AEs) During Study Part II [ Time Frame: 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.

Original Secondary Outcome: 1) Change in total sleep time 2) Quality of life 3) Treatment safety. [ Time Frame: 1-6 months ]

Information By: Ferring Pharmaceuticals

Dates:
Date Received: May 22, 2007
Date Started: May 2007
Date Completion:
Last Updated: September 29, 2015
Last Verified: September 2015