Central Diabetes Insipidus
Conditions
Keywords
Diabetes
Brief summary
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.
Interventions
DRUGDesmopressin Oral Melt
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.
DRUGDesmopressin intranasal
Self-administered intranasal desmopressin throughout the pre-study observation period (Days -30 to Day 0) and on study Day 1
Sponsors
Ferring Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Participants must be documented to have Central Diabetes Insipidus (CDI) by at least two of the following four criteria (a-d): 1. 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) 2. Complete and continuous control of the DI by desmopressin therapy without breakthrough diuresis, hypernatremia, hyponatremia, or symptoms or signs of water intoxication. 3. A deficient plasma vasopressin response to osmotic or non-osmotic stimulation. 4. 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from Baseline in 24-hour Urine Volume | Day 0, Week 4 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | — |
| 24-hour urine volume (mL) | Day 0, Week 4 | — |
| Serum sodium level | up to Month 13 | — |
| Participants with Adverse Events Summarized by Incidence and Severity | up to Month 13 | Includes abnormal lab values and vital signs |
| Percentage of participants within normal range for urinary output, urinary osmolality and urine specific gravity | Day 0, Week 4 | — |
Countries
Japan
Outcome results
None listed