Clinical Trials Register

Summary
EudraCT Number:	2012-004388-34
Sponsor's Protocol Code Number:	000088
National Competent Authority:	UK - MHRA
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2013-01-02
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

UK - MHRA

A.2

EudraCT number

2012-004388-34

A.3

Full title of the trial

A Double-blind, Randomised, Parallel-group Trial Investigating Sleep Behaviour and Daytime Performance in Nocturia Patients Treated with Desmopressin Orally Disintegrating Tablets as compared to Placebo

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

Clinical Trial Investigating Sleep Behaviour and Daytime Performance in Nocturia Patients Treated with Desmopressin Orally Disintegrating Tablets as compared to Placebo

A.3.2

Name or abbreviated title of the trial where available

DAISS

A.4.1

Sponsor's protocol code number

000088

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Ferring Pharmaceuticals A/S
B.1.3.4	Country	Denmark
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Ferring Pharmaceuticals A/S
B.4.2	Country	Denmark
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Ferring Pharmaceuticals A/S
B.5.2	Functional name of contact point	Clinical Development Support
B.5.3	Address:
B.5.3.1	Street Address	Kay Fiskers Plads 11
B.5.3.2	Town/ city	Copenhagen
B.5.3.3	Post code	2300
B.5.3.4	Country	Denmark
B.5.6	E-mail	DK0-Disclosure@ferring.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Desmopressin orally disintegrating tablet
D.3.4	Pharmaceutical form	Oral lyophilisate
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Sublingual use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	DESMOPRESSIN
D.3.9.1	CAS number	16679-58-6
D.3.9.2	Current sponsor code	FE 992026
D.3.9.4	EV Substance Code	SUB07001MIG
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	50
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Desmopressin orally disintegrating tablet
D.3.4	Pharmaceutical form	Oral lyophilisate
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Sublingual use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	DESMOPRESSIN
D.3.9.1	CAS number	16679-58-6
D.3.9.2	Current sponsor code	FE 992026
D.3.9.4	EV Substance Code	SUB07001MIG
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	25
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Oral lyophilisate
D.8.4	Route of administration of the placebo	Sublingual use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Nocturia

E.1.1.1

Medical condition in easily understood language

The complaint that the individual has to wake up at night one or more times to void

E.1.1.2

Therapeutic area

Diseases [C] - Symptoms and general pathology [C23]

MedDRA Classification

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

Efficacy Objectives
• To investigate the relationship between nocturia, sleep, and daytime performance
• To investigate the efficacy of desmopressin orally disintegrating tablets (50 µg in men and 25 µg in women) versus placebo for treatment of patients with nocturia with respect to nocturia, sleep, and daytime performance.

Safety Objective
• To confirm the safety of desmopressin orally disintegrating tablets (50 µg in men and 25 µg in women) in the treatment of nocturia.

E.2.2

Secondary objectives of the trial

Not applicable

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Written informed consent has been given prior to performance of any trial-related activity

2. Male or female patients, aged 25-65 years at the time of written consent, who currently have a regular sleep/wake schedule with minimal daytime napping

3. Nocturia symptoms which have been present for ≥6 months prior to trial entry (patient-reported)

4. At least 2 nocturnal voids per night as judged by the Investigator during both 3-day diary periods
5. A nocturia index >1 (nocturnal urine production exceeding maximum voided volume)

6. A time-in-bed period of between 6.0 and 9.5 hours per night (based on screening diary and actigraphy)

7. The patient is able to read and understand the informed consent form and understand the trial procedures

8. Pre- or peri-menopausal women must have a negative pregnancy test and be willing and able to use a reliable method of contraception throughout the trial. In the context of this trial, an effective method is defined as those which result in low failure rate (i.e. less than 1% per year) when used consistently and correctly such as: implants, injectables, combined or progesterone only oral contraceptives, sexual abstinence or vasectomised (male patients or partners). Amenorrhea of more than 12 months duration based on the reported date of the last menstrual period is sufficient documentation of post-menopausal status and does not require a pregnancy test

9. Be willing and able to stay at the sleep centre as required by the protocol.

E.4

Principal exclusion criteria

1. Evidence of severe voiding dysfunction defined as >10 nocturnal voids as documented on any given night in the voiding diary during the screening period

2. History of, or evidence of obstructive sleep apnoea (AHI >10) and PLMS (≥10 arousals) as measured by PSG at Visit 1b

3. Evidence of other sleep disorders which are independent of nocturia

4. Signs or symptoms of any of the following diseases:
• severe bladder outlet obstruction with a Q max <5 ml/sec as confirmed by uroflowmetry; urinary retention or a post-void residual volume in excess of 150 mL (in women) or 250 mL (in men) as confirmed by bladder ultrasound
• severe lower urinary tract symptoms (LUTS) as defined by International Prostate Symptom Score (IPSS) (20 to 35 represents severe symptoms)
• interstitial cystitis
• moderate to severe overactive bladder (OAB) defined as ≥8 daytime voids per day/night time cycle, and/or any urge urinary incontinence (UUI) episode as documented in the 3-day voiding diary period just before Visit 2 patients with UUI are to be excluded.
• moderate to severe, as judged by the Investigator, stress urinary incontinence or mixed incontinence, where stress incontinence is the predominant component based on previous history

5. Genito-urinary tract pathology which in the opinion of the Investigator may be responsible for urgency or urinary incontinence e.g., symptomatic or recurrent urinary tract infections, interstitial cystitis, bladder related pain, or stone in the bladder and urethra causing symptoms

6. Urological malignancies

7. Neurogenic detrusor overactivity (e.g. parkinson, spinal cord damage, etc.)

8. Central or nephrogenic diabetes insipidus

9. Habitual or psychogenic polydipsia (fluid intake resulting in a urine production exceeding >40 mL/kg/24 hours)

10. Syndrome of inappropriate antidiuretic hormone (SIADH)

11. Cardiac failure, evidence based on physical examination, cardiac medical history, and electrocardiogram (ECG) output

12. Uncontrolled hypertension

13. Uncontrolled diabetes mellitus

14. Hyponatraemia with a serum sodium level <135 mmol/L or history of hyponatraemia

15. Renal insufficiency: Serum creatinine must be within normal laboratory reference intervals and estimated glomerular filtration rate must be ≥50 mL/min

16. Known or suspected clinically significant hepatic and/or biliary diseases:
•aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) levels must not be more than twice the upper limit of normal range (≤2 X ULN) and total bilirubin level must not be more than 1.5 mg/dL
•if AST and/or ALT ≥2 but ≤3 X ULN, total bilirubin level must be normal
•if total bilirubin level 1.5-2.0 mg/dL, AST and/or ALT must be normal

17. Women who are pregnant, breastfeeding, or who plan to become pregnant during the period of the trial

E.5 End points

E.5.1

Primary end point(s)

Efficacy Endpoints

Nocturia Endpoints:
• Mean number of nocturnal voids at Month 1 and Month 3
• Mean time to first void at Month 1 and Month 3.

Sleep Endpoints:
• Objective Measures of Sleep – polysomnography (PSG)
• Objective Measures of Sleep - Actigraphy
• Subjective Measures of Voiding and Sleep

Daytime Endpoints:
• Subjective Measures of Daytime Performance

Safety Endpoints:
• Incidence, type and severity of adverse events (AEs)
• Clinically significant changes in laboratory values, vital signs, and physical examinations.

E.5.1.1

Timepoint(s) of evaluation of this end point

Continually throughout the trial

E.5.2

Secondary end point(s)

Not applicable

E.5.2.1

Timepoint(s) of evaluation of this end point

Not applicable

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.5

The trial involves multiple Member States

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

None

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2013-02-13

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2013-03-06

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2014-06-20

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