Clinical trial NCT01280188: A Study of Minirin Melt in Japanese Patients With Central Diabetes Insipidus (CDI).
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Central Diabetes Insipidus
Keywords: Diabetes
Lead sponsor: Ferring Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2011-01-20
Enrollment: 20
Sex: ALL
Minimum age (years): 6
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Change from Baseline in 24-hour Urine Volume
timeFrame: Day 0, Week 4
Secondary outcomes:
measure: 24-hour urine volume (mL)
timeFrame: Day 0, Week 4
measure: Hourly diuresis rate (mL/hr)
timeFrame: Day 0, Week 4
measure: Urine osmolality (mOsm/kg)
timeFrame: Day 0, Week 4
measure: Urine specific gravity (g/mL)
timeFrame: Day 0, Week 4
measure: Percentage of participants within normal range for urinary output, urinary osmolality and urine specific gravity
timeFrame: Day 0, Week 4
measure: Serum sodium level
timeFrame: up to Month 13
measure: Participants with Adverse Events Summarized by Incidence and Severity
timeFrame: up to Month 13
Other outcomes:
Locations:
city: Nagakute, Aichi
country: Japan
facility: Aichi Medical University
state: Aichi
city: Nagoya
country: Japan
facility: Nagoya University Hospital
state: Aichi
city: Minato
country: Japan
facility: Toranomon Hospital
state: Tokyo
city: Osaka
country: Japan
facility: Osaka Saiseikai Nakatsu Hospital
city: Saitama
country: Japan
facility: Saitama Medical Center Jichi Medical University
Study officials:
affiliation: Ferring Pharmaceuticals
name: Clinical Development Support
role: STUDY_DIRECTOR
References:
citation: Arima H, Oiso Y, Juul KV, Norgaard JP. Efficacy and safety of desmopressin orally disintegrating tablet in patients with central diabetes insipidus: results of a multicenter open-label dose-titration study. Endocr J. 2013;60(9):1085-94. doi: 10.1507/endocrj.ej13-0165. Epub 2013 Jun 28.