ICH GCP - EU Clinical trials Registry - 2014-004641-27 (BE)

Klinische Studien Nct Page

Summary
EudraCT Number:	2014-004641-27
Sponsor's Protocol Code Number:	UZLeuven
National Competent Authority:	Belgium - FPS Health-DGM
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2015-04-21
Trial 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

A. Protocol Information

A.1

Member State Concerned

Belgium - FPS Health-DGM

A.2

EudraCT number

2014-004641-27

A.3

Full title of the trial

BOTOX in the treatment of urinary incontinence due to neurogenic detrusor overactivity in patients 3 months - 17 years

BOTOX in de behandeling van neurogene blaasdysfunctie bij kinderen tussen 3 maanden en 17 jaar

A.3.1

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

Treatment with Botox in patients with urinary incontinence (accidental loss of urine) due to neurogenic detrusor overactivity (bladder problems resulting from disease or damage to the nervous system

Botoxbehandeling bij patiënten met urine-incontinentie (accidenteelverlies) door neurogene overactiviteit van de detrusor (blaasproblemen ten gevolge van ziekte of schade aan het centraal zenuwstelsel)

A.3.2

Name or abbreviated title of the trial where available

Botox treatment

Botoxbehandeling

A.4.1

Sponsor's protocol code number

UZLeuven

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	UZLeuven
B.1.3.4	Country	Belgium
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	UZLeuven
B.4.2	Country	Belgium
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	UZLeuven
B.5.2	Functional name of contact point	Urology trial
B.5.3	Address:
B.5.3.1	Street Address	Herestraat 49
B.5.3.2	Town/ city	Leuven
B.5.3.3	Post code	3000
B.5.3.4	Country	Belgium
B.5.4	Telephone number	3216346930
B.5.5	Fax number	3216348349
B.5.6	E-mail	guy.bogaert@uzleuven.be

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Comparator
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	Yes
D.2.1.1.1	Trade name	Botox
D.2.1.1.2	Name of the Marketing Authorisation holder	Allergan
D.2.1.2	Country which granted the Marketing Authorisation	Belgium
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.3.4	Pharmaceutical form	Intravesical solution/solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravesical use
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	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	Yes
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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Urinary incontinence due to neurogenic detrusor overactivity

Urinaire incontinentie door neurogene detrusor overactiviteit

E.1.1.1

Medical condition in easily understood language

Accidental loss of urine due to bladder problems resulting from disease or damage to the nervous system

Accidenteel urineverlies door blaasproblemen ten gevolge van ziekte of schade aan het zenuwstelsel

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	18.0
E.1.2	Level	PT
E.1.2	Classification code	10029279
E.1.2	Term	Neurogenic bladder
E.1.2	System Organ Class	10038359 - Renal and urinary disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

Children who are still incontinent but have no shown detrusor contraction during urodynamic investigation

Kinderen die nog incontinent zijn, maar geen aantoonbare detrusorcontractie hebben tijdens het urodynamisch onderzoek

E.2.2

Secondary objectives of the trial

Not applicable

Niet van toepassing

E.2.3

Trial contains a sub-study

E.2.3.1

Full title, date and version of each sub-study and their related objectives

Not applicable

Niet van toepassing

E.3

Principal inclusion criteria

- Male or female, ≥ 0 and ≤ 17 years of age at the moment of signing the informed consent
- Patient with neurogenic bladder dysfunction shown during urodynamic investigation with or without overactive detrusor contraction
- Neurogenic detrusor overactivity caused by;
* spinal dysrafie, more specifically spina bifida (e.g. myelomeningocele, meningocele) and all forms of tethered cord
* Acquired neurogenic detrusor overactivity due to spinal cord injury
- Regular intermittent sondage to empty the bladder
- more than 4 episodes of urine incontinence over two days in the miction diary
- the neurogenic bladder dysfunction is not sufficiently controlled with anticholinergics only

- Mannelijk of vrouwelijk, ≥ 0 jaar tot ≤ 17 jaar op moment van ondertekenen van geïnformeerde toestemming
- Patiënt heeft neurogene blaasdysfunctie, bewezen tijdens urodynamisch onderzoek. Hierbij kan al dan niet een overactieve detrusorcontractie worden opgemerkt of niet
- Neurogene detrusoractiviteit veroorzaakt door:
* Spinale dysrafie, hetgeen spina bifida (bv. myelomeningocele, meningocele) en alle vormen van tethered cord
* Verworven neurogene detrusoroveractiviteit door een ruggenmerglaesie
- Patiënt moet regelmatig intermittente sondage gebruiken om de blaas te ledigen
- Patiënt moet ≥ 4 episodes van urine-incontinentie hebben over twee dagen in het mictiedagboek
- De neurogene blaasdysfunctie is onvoldoende onder controle met enkel anticholinergica

E.4

Principal exclusion criteria

- occurence of uncontrolled systemic diseases
- occurence of malignancies
- spinal cord surgery during the past 6 months
- additional pelvic or urologic abnormalities (open bladder neck, urethra stricture/urethra valve
- predominance of stress incontinence

- aanwezigheid van ongecontroleerde systeemziekten
- aanwezigheid van maligniteiten
- ruggenmergchirurgie in de afgelopen 6 maanden
- bijkomende pelvische of urologische abnormaliteiten (open blaashals, urethrastrictuur/urethraklep
- predominantie van stress incontinentie

E.5 End points

E.5.1

Primary end point(s)

- efficacy: The data of the miction diaries are studied during use of intradetrusor injections with Botox
- Safety: To confirm the safety of Botox in children on the basis of medical history, clinical investigation and labo

- efficiëntie: De gegevens van de mictiedagboeken worden bestudeerd gedurende gebruik van intradetrusorinjecties met Botox
- veiligheid: De veiligheid van Botox bij kinderen confirmeren aan de hand van anamnese, klinisch onderzoek, labo

E.5.1.1

Timepoint(s) of evaluation of this end point

- Screening
- Treatment
- Visits: week 2, 6, 12, 24, 36 and 48
- Calls: week 18, 30 and 42

- Screening
- Behandeling
- Visites: week 2, 6, 12, 24, 36 en 48
- telefonisch contact: week 18, 30 en 42

E.5.2

Secondary end point(s)

Not applicable

Niet van toepassing

E.5.2.1

Timepoint(s) of evaluation of this end point

Not applicable

Niet van toepassing

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)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

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

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.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

E.8.5.1

Number of sites anticipated in the EEA

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

Yes

F.1.1

Number of subjects for this age range:

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)

Yes

F.1.1.4.1

Number of subjects for this age range:

F.1.1.5

Children (2-11years)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

F.1.2

Adults (18-64 years)

F.1.3

Elderly (>=65 years)

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

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Patient population: inclusion possible as from the age of 3 months

patiëntenpopulatie: inclusie mogelijk vanaf 3 maanden

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

Geen

G. Investigator Networks to be involved in the Trial
G.4 Investigator Network to be involved in the Trial: 1

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

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2015-05-21

Ethics Committee Opinion of the trial application

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2018-06-08

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