Trial Investigating the Long Term Safety and Tolerability of Desmopressin Orally Disintegrating Tablets (ODT) for Nocturia Due to Nocturnal Polyuria in Japanese Subjects

A Multi-Centre Trial Investigating the Long Term Safety and Tolerability of Desmopressin (FE 992026) Orally Disintegrating Tablets for the Treatment of Nocturia Due to Nocturnal Polyuria in Japanese Subjects

Demonstrate the safety and tolerability of desmopressin ODT during long-term treatment of subjects with nocturia due to nocturnal polyuria, for up to 1 year

Study Overview

• Status: Completed
• Conditions: Nocturia
• Intervention / Treatment: Drug: Desmopressin ODT 25 μg; Drug: Desmopressin ODT 50 μg

• Study Type: Interventional
• Enrollment (Actual): 503
• Phase: Phase 3

Contacts and Locations

Study Locations

• Japan
  • Tokyo, Japan
    • Investigational site (there may be other sites in this country)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria (for subjects who participated in trials 000129 or 000130):

• Written informed consent prior to performance of any trial-related activity for the 000131 trial
• Has completed participation in trial 000129 or 000130

Exclusion Criteria (for subjects who participated in trials 000129 or 000130):

• Hyponatraemia (serum sodium level <135 mmol/L) at Visit 7 in trial 000129 or 000130
• Withdrawal from clinical trial 000129 or 000130

Inclusion Criteria (for subjects who did not participate in trials 000129 or 000130):

• Written informed consent prior to performance of any trial-related activity
• Adult ≥20 years of age
• Nocturia symptoms present for ≥6 months prior to trial entry at Visit 1a
• Nocturnal polyuria at the end of screening period prior to Visit 1b
• Bothered by nocturia on the Hsu 5-point Likert bother scale at Visit 1a and Visit 1b
• Has given agreement about contraception during the trial

Exclusion Criteria (for subjects who did not participate in trials 000129 or 000130):

• Early withdrawal from clinical trial 000129 or 000130
• Evidence of any significant voiding dysfunction resulting in abnormally low bladder capacity at the end of the screening period prior to Visit 1b
• History or evidence of significant obstructive sleep apnoea

• History or diagnosis of any of the following urological diseases:

  • Interstitial cystitis or bladder pain disorder

  • In males, suspicion of moderate or severe benign prostate hyperplasia (BPH), defined as international prostate symptom score (IPSS) ≥8 points and:

    • Urinary flow <5 mL/s or
    • Post-void residual volume >150 mL
  • Stress urinary incontinence or mixed incontinence, where stress incontinence is the predominant component based on prior history
  • Chronic prostatitis/chronic pelvic pain syndrome
• Surgical treatment, including transurethral resection, for BOO or BPH within the past 6 months prior to Visit 1a

• Symptoms of severe over-active bladder (OAB):

  • Defined as an over-active bladder symptom score (OABSS) ≥12 at Visit 1a
  • Defined as a mean of >8 voids and a mean of ≥1 urgency episode per 24 hours at the end of the screening period prior to Visit 1b
• Genito-urinary tract pathology that can in the investigator's opinion be responsible for urgency or urinary incontinence at Visit 1a
• Complication of cancer or a history of cancer which has not been in remission for the last 5 years at Visit 1a
• Current or a history of urologic malignancies, any lower urinary tract surgery, previous pelvic irradiation, or neoplasia at Visit 1a
• History of any neurological disease affecting bladder function or muscle strength at Visit 1a
• Habitual or psychogenic polydipsia based on medical history at Visit 1a or 24-hour urine output of >2.8 L based on the voiding diary at Visit 1b
• Central or nephrogenic diabetes insipidus at Visit 1a
• Syndrome of inappropriate antidiuretic hormone secretion at Visit 1a
• Suspicion or evidence of cardiac failure at Visit 1a
• Uncontrolled hypertension at Visit 1a and Visit 1b
• Hyponatraemia (serum sodium level <135 mmol/L) at Visit 1a Renal insufficiency at Visit 1a and Visit 1b
• Renal insufficiency at Visit 1a and Visit 1b
• Hepatic and/or biliary diseases at Visit 1a and Visit 1b
• Known or suspected hypersensitivity to desmopressin ODTs or previous desmopressin treatment for nocturia at Visit 1a
• In females, pregnancy, breastfeeding, or a plan to become pregnant during the period of the clinical trial.
• Known alcohol or substance abuse at Visit 1a
• Work or lifestyle that may interfere with regular night-time sleep at Visit 1a, e.g., shift workers
• Any other medical condition, laboratory abnormality, psychiatric condition, mental incapacity, or language barrier that, in the judgment of the investigator, would impair participation in the trial at Visit 1a
• Use of any prohibited therapy during the trial period

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

8

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Desmopressin ODT 25 μg (female previous on 25 μg); Subjects received 25 μg in trial 000129.	Drug: Desmopressin ODT 25 μg; Other Names: FE 992026
Experimental: Desmopressin ODT 25 μg (female previously on placebo); Subjects received placebo in trial 000129	Drug: Desmopressin ODT 25 μg; Other Names: FE 992026
Experimental: Desmopressin ODT 25 μg (female); New female subjects	Drug: Desmopressin ODT 25 μg; Other Names: FE 992026
Experimental: Desmopressin ODT 25 μg (male previous on 25 μg); Subjects received 25 μg in trial 000130	Drug: Desmopressin ODT 25 μg; Other Names: FE 992026
Experimental: Desmopressin ODT 50 μg (male previous on 50 μg); Subjects received 50 μg in trial 000130	Drug: Desmopressin ODT 50 μg; Other Names: FE 992026
Experimental: Desmopressin ODT 50 μg (male previous on placebo); Subjects received placebo in trial 000130	Drug: Desmopressin ODT 50 μg; Other Names: FE 992026
Experimental: Desmopressin ODT 25 μg (male); Subjects received placebo in trial 000130	Drug: Desmopressin ODT 25 μg; Other Names: FE 992026
Experimental: Desmopressin ODT 50 μg (male); New male subjects	Drug: Desmopressin ODT 50 μg; Other Names: FE 992026

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The frequency and severity of adverse events	During long-term treatment	Up to 1 year
Clinically significant changes in laboratory values and vital signs	During long-term treatment	Up to 1 year
The incidence and severity of hyponatraemia	Measured by serum sodium levels during long-term treatment	Up to 1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in mean number of nocturnal voids		Week 12, 24, 40 and 52
Change from baseline in mean time to first awakening to void		Week 12, 24, 40 and 52
Change from baseline in mean nocturnal urin volume		Week 12, 24, 40 and 52
Change from baseline in mean Nocturnal Polyuria Index (NPI)		Week 12, 24, 40 and 52
Change from baseline in Nocturia-Specific Quality-of-Life Questionnaire (N-QoL)		Week 12, 24, 40 and 52
Change from baseline in Insomnia Severity Index (ISI)		Week 12, 24, 40 and 52
Change from baseline in bother score	Assessed by the Hsu 5-point Likert bother scale	Week 12, 24, 40 and 52

Collaborators and Investigators

• Sponsor: Ferring Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): January 11, 2017
• Primary Completion (Actual): September 11, 2018
• Study Completion (Actual): September 11, 2018

Study Registration Dates

• First Submitted: February 9, 2017
• First Submitted That Met QC Criteria: February 9, 2017
• First Posted (Actual): February 13, 2017

Study Record Updates

• Last Update Posted (Actual): September 17, 2018
• Last Update Submitted That Met QC Criteria: September 14, 2018
• Last Verified: September 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urologic Diseases; Lower Urinary Tract Symptoms; Urological Manifestations; Urination Disorders; Nocturia; Polyuria; Physiological Effects of Drugs; Natriuretic Agents; Hemostatics; Coagulants; Antidiuretic Agents; Deamino Arginine Vasopressin
• Other Study ID Numbers: 000131

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No