A Study of Minirin Melt in Japanese Patients With Central Diabetes Insipidus (CDI).

Study Overview
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Brief Summary
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This is an open-label dose-titration study in Japanese Central Diabetes Insipidus (CDI) patients designed to demonstrate the efficacy and safety of orally-disintegrating tablet of desmopressin.

Official Title
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Peroral Administration of Different Doses of Desmopressin Administered as a New Orally-Disintegrating Tablet and Desmopressin for Nasal Administration in the Treatment of CDI in Japanese Patients

Conditions
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Central Diabetes Insipidus

Intervention / Treatment
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* Drug: Desmopressin Oral Melt
* Drug: Desmopressin intranasal


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participants must be documented to have Central Diabetes Insipidus (CDI) by at least two of the following four criteria (a-d): Failure to increase urine osmolality above 300 mOsm/kg during a period of fluid deprivation sufficient to raise plasma osmolality and sodium above the upper limit of normal level for the reference laboratory (usually 295 mOsm/kg and 148 mEq/l, respectively) Complete and continuous control of the DI by desmopressin therapy without breakthrough diuresis, hypernatremia, hyponatremia, or symptoms or signs of water intoxication. A deficient plasma vasopressin response to osmotic or non-osmotic stimulation. Absence of the posterior pituitary bright spot on T-1 weighted midsagittal magnetic resonance imaging (MRI) of the brain. Given written informed consent prior to any trial-related procedure is performed 24 hour urine volume (mL), urine osmolality (mOsm/kg), urine specific gravity, and serum sodium (mEq/L) maintained at a normal level by desmopressin nasal administration Outpatient The participant is, in the investigator's opinion, otherwise healthy Be willing and able to comply with the protocol requirements including restriction of water intake Exclusion Criteria: Presence or a history of nephrogenic diabetes insipidus or diabetes mellitus Presence of uncorrected hypothyroidism, hypoadrenalism or hypogonadism Abnormalities or disease of the oral cavity that might affect the release and absorption of drug Unable to be placed on water-intake restriction starting from two hours before bedtime Presence of a hypothalamus abnormality leading to thirst disorder Evidence of hepatic, renal, cardiac, or pulmonary dysfunction Uncontrolled hypertension Treatment with another investigational product within the past 3 months Concurrent treatment with diuretics, chlorpropamide, tricyclic antidepressants, indomethacin, carbamazepine Alcohol dependency or drug abuse Breastfeeding, pregnant, or likely to become pregnant A mental condition, the lack of decision-making ability, dementia or a speech handicap Any other reason that the Investigator believes inappropriate

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Desmopressin<br>Day 1 - participants continue desmopressin intranasal. Day 2 up to Day 4 - Desmopressin oral melt to optimum dose. Continue optimum dose for the four week treatment and one year follow-up periods. | Drug: Desmopressin Oral Melt<br>* Oral melt formulation starts on Day 2. The target initial dose of the orally disintegrating tablet is 180µg/day (60µg taken 3 times a day) and adjusted to optimally stabilise the participant's condition.<br>* Other names: FE992026;Drug: Desmopressin intranasal<br>* Self-administered intranasal desmopressin throughout the pre-study observation period (Days -30 to Day 0) and on study Day 1<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from Baseline in 24-hour Urine Volume |  | Day 0, Week 4 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 24-hour urine volume (mL) |  | Day 0, Week 4 | 
| Hourly diuresis rate (mL/hr) |  | Day 0, Week 4 | 
| Urine osmolality (mOsm/kg) |  | Day 0, Week 4 | 
| Urine specific gravity (g/mL) |  | Day 0, Week 4 | 
| Percentage of participants within normal range for urinary output, urinary osmolality and urine specific gravity |  | Day 0, Week 4 | 
| Serum sodium level |  | up to Month 13 | 
| Participants with Adverse Events Summarized by Incidence and Severity | Includes abnormal lab values and vital signs | up to Month 13 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Diabetes