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

• ClinicalTrials.gov Identifier: NCT03051009
• Recruitment Status: Completed
• First Posted: February 13, 2017
• Last Update Posted: September 17, 2018
• Sponsor: Ferring Pharmaceuticals
• Information provided by (Responsible Party): Ferring Pharmaceuticals

Study Description

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

Condition or disease	Intervention/treatment	Phase
Nocturia	Drug: Desmopressin ODT 25 μg; Drug: Desmopressin ODT 50 μg	Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 503 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• 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.
• Masking: Double (Participant, Investigator)

Masking Description

One dose level (25 μg desmopressin OCD) is included for female subjects and will be open-labelled for all female subjects.

Two different dose levels (25 μg desmopressin and 50 μg desmopressin ODT are included for male subjects in a double-blinded manner. Male subjects continuing from trial 000130 will continue their randomised treatment and subjects who received placebo will be randomised to one of the 2 dose levels (25 μg desmopressin or 50 μg desmopressin ODT).

New male subjects will be allocated to receive 50 μg desmopressin ODT in an open-labelled manner.

• Primary Purpose: Treatment
• 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
• Actual Study Start Date: January 11, 2017
• Actual Primary Completion Date: September 11, 2018
• Actual Study Completion Date: September 11, 2018

Arms and Interventions

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 Name: FE 992026
Experimental: Desmopressin ODT 25 μg (female previously on placebo); Subjects received placebo in trial 000129	Drug: Desmopressin ODT 25 μg; Other Name: FE 992026
Experimental: Desmopressin ODT 25 μg (female); New female subjects	Drug: Desmopressin ODT 25 μg; Other Name: 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 Name: 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 Name: FE 992026
Experimental: Desmopressin ODT 50 μg (male previous on placebo); Subjects received placebo in trial 000130	Drug: Desmopressin ODT 50 μg; Other Name: FE 992026
Experimental: Desmopressin ODT 25 μg (male); Subjects received placebo in trial 000130	Drug: Desmopressin ODT 25 μg; Other Name: FE 992026
Experimental: Desmopressin ODT 50 μg (male); New male subjects	Drug: Desmopressin ODT 50 μg; Other Name: FE 992026

Outcome Measures

Primary Outcome Measures :

1. The frequency and severity of adverse events [ Time Frame: Up to 1 year ]
   During long-term treatment
2. Clinically significant changes in laboratory values and vital signs [ Time Frame: Up to 1 year ]
   During long-term treatment
3. The incidence and severity of hyponatraemia [ Time Frame: Up to 1 year ]
   Measured by serum sodium levels during long-term treatment

Secondary Outcome Measures :

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

Eligibility Criteria

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

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

Contacts and Locations

Locations

Japan

Investigational site (there may be other sites in this country)

Tokyo, Japan

Sponsors and Collaborators

Ferring Pharmaceuticals

Investigators

• Study Director: Clinical Development Support; Ferring Pharmaceuticals

More Information

• Responsible Party: Ferring Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT03051009
• Other Study ID Numbers: 000131
• First Posted: February 13, 2017
• Last Update Posted: September 17, 2018
• Last Verified: September 2018

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Polyuria; Nocturia; Lower Urinary Tract Symptoms; Urological Manifestations; Signs and Symptoms; Urination Disorders; Urologic Diseases; Deamino Arginine Vasopressin; Hemostatics; Coagulants; Antidiuretic Agents; Natriuretic Agents; Physiological Effects of Drugs