Clinical trial NCT03051009: Trial Investigating the Long Term Safety and Tolerability of Desmopressin Orally Disintegrating Tablets (ODT) for Nocturia Due to Nocturnal Polyuria in Japanese Subjects
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Nocturia
Lead sponsor: Ferring Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2017-02-13
Enrollment: 503
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: The trial is designed as partly an extension trial for subjects completing trial 000129 (female subjects) and 000130 (male subjects) and partly as a trial for new female and male subjects.
Primary purpose: TREATMENT

Brief summary: 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


Eligibility criteria: 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

Primary outcomes:
measure: The frequency and severity of adverse events
timeFrame: Up to 1 year
measure: Clinically significant changes in laboratory values and vital signs
timeFrame: Up to 1 year
measure: The incidence and severity of hyponatraemia
timeFrame: Up to 1 year
Secondary outcomes:
measure: Change from baseline in mean number of nocturnal voids
timeFrame: Week 12, 24, 40 and 52
measure: Change from baseline in mean time to first awakening to void
timeFrame: Week 12, 24, 40 and 52
measure: Change from baseline in mean nocturnal urin volume
timeFrame: Week 12, 24, 40 and 52
measure: Change from baseline in mean Nocturnal Polyuria Index (NPI)
timeFrame: Week 12, 24, 40 and 52
measure: Change from baseline in Nocturia-Specific Quality-of-Life Questionnaire (N-QoL)
timeFrame: Week 12, 24, 40 and 52
measure: Change from baseline in Insomnia Severity Index (ISI)
timeFrame: Week 12, 24, 40 and 52
measure: Change from baseline in bother score
timeFrame: Week 12, 24, 40 and 52
Other outcomes:
Locations:
city: Tokyo
country: Japan
facility: Investigational site (there may be other sites in this country)
Study officials:
affiliation: Ferring Pharmaceuticals
name: Clinical Development Support
role: STUDY_DIRECTOR
References: