Clinical Trials Register

Summary
EudraCT Number:	2015-002969-27
Sponsor's Protocol Code Number:	PMF104PD1-2-3/2013
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2015-09-18
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

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2015-002969-27

A.3

Full title of the trial

A RANDOMISED, SINGLE-BLIND, ACTIVE CONTROLLED, MULTI-CENTRE TRIAL TO EVALUATE THE EFFICACY, SAFETY, TOLERABILITY, ACCEPTABILITY AND PALATABILITY OF PMF104 COMPARED TO A CONVENTIONAL PEG-ELECTROLYTE SOLUTION IN CHILDREN AGED FROM 2 TO LESS THAN 6, FROM 6 TO LESS THAN 12 AND ADOLESCENTS FROM 12 TO LESS THAN 18 YEARS OF AGE REQUIRING A DIAGNOSTIC PROCEDURE CONCERNING THE COLON

Studio randomizzato, in singolo-cieco, controllato e multicentrico per valutare l’efficacia, la sicurezza, la tollerabilità, l’accettabilità e la palatabilità di PMF104 rispetto ad una convenzionale soluzione elettrolitica di polietilenglicole, somministrate a bambini da 2 a meno di 6 anni, da 6 a meno di 12 anni e ad adolescenti da 12 a meno di 18 anni che necessitano di una procedura diagnostica a livello del colon

A.3.1

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

Clinical trial aimed at evaluating the efficacy, safety, tolerability, acceptability and palatability of PMF104 in children from 2 to less than 18 years old

Studio clinico che ha l'obiettivo di valutare l'efficacia, la sicurezza, la tollerabilità, l'accettabilità da parte del paziente e la palatabilità del farmaco PMF104 in bambini di età compresa tra i 2 e meno di 18 anni.

A.4.1

Sponsor's protocol code number

PMF104PD1-2-3/2013

A.5.4

Other Identifiers

Name:

CRO-15-125

Number:

CRO code

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/051/2014

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Alfa Wassermann S.p.A.
B.1.3.4	Country	Italy
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	Alfa Wassermann S.p.A.
B.4.2	Country	Italy
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	CROSS S.A. and associated companies CROSS research S.A. and CROSS Metrics S.A.
B.5.2	Functional name of contact point	Ottavia Annoni
B.5.3	Address:
B.5.3.1	Street Address	Via F.A. Giorgioli 14
B.5.3.2	Town/ city	Arzo
B.5.3.3	Post code	CH-6864
B.5.3.4	Country	Switzerland
B.5.4	Telephone number	0041916300510
B.5.5	Fax number	0041916300511
B.5.6	E-mail	ottavia.annoni@croalliance.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	EASYGOL
D.3.2	Product code	PMF104
D.3.4	Pharmaceutical form	Powder for oral solution in sachet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Nasogastric use (Noncurrent) Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Macrogol 4000
D.3.9.1	CAS number	25322-68-3
D.3.9.3	Other descriptive name	MACROGOL 4000 PH. EUR.
D.3.9.4	EV Substance Code	SUB172298
D.3.10	Strength
D.3.10.1	Concentration unit	g gram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	52.500
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Sodium sulfate, anhydrous
D.3.9.1	CAS number	7757-82-6
D.3.9.3	Other descriptive name	SODIUM SULPHATE
D.3.9.4	EV Substance Code	SUB20969
D.3.10	Strength
D.3.10.1	Concentration unit	g gram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	3.750
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Simeticone
D.3.9.1	CAS number	8050-81-5
D.3.9.3	Other descriptive name	SIMETICONE
D.3.9.4	EV Substance Code	SUB04403MIG
D.3.10	Strength
D.3.10.1	Concentration unit	g gram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	0.080
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Sodium citrate
D.3.9.1	CAS number	68-04-2
D.3.9.3	Other descriptive name	SODIUM CITRATE ANHYDROUS
D.3.9.4	EV Substance Code	SUB34328
D.3.10	Strength
D.3.10.1	Concentration unit	g gram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	1.863
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Citric acid anhydrous
D.3.9.1	CAS number	77-92-9
D.3.9.3	Other descriptive name	CITRIC ACID ANHYDROUS
D.3.9.4	EV Substance Code	SUB29050
D.3.10	Strength
D.3.10.1	Concentration unit	g gram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	0.813
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Sodium chloride
D.3.9.1	CAS number	7647-14-5
D.3.9.3	Other descriptive name	SODIUM CHLORIDE
D.3.9.4	EV Substance Code	SUB12581MIG
D.3.10	Strength
D.3.10.1	Concentration unit	g gram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	0.730
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Potassium chloride
D.3.9.1	CAS number	7447-40-7
D.3.9.3	Other descriptive name	POTASSIUM CHLORIDE
D.3.9.4	EV Substance Code	SUB12559MIG
D.3.10	Strength
D.3.10.1	Concentration unit	g gram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	0.370
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	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	KLEAN-PREP
D.2.1.1.2	Name of the Marketing Authorisation holder	Norgine Italia S.r.l.
D.2.1.2	Country which granted the Marketing Authorisation	Italy
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	KLEAN-PREP
D.3.4	Pharmaceutical form	Powder for oral solution in sachet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Nasogastric use (Noncurrent) Oral use
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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Need of bowel cleansing in order to perform a colonoscopy or any other diagnostic or therapeutic procedure concerning the colon.

Necessità di svuotamento intestinale prima di una colonscopia o di una qualsiasi altra procedure diagnostica o terapeutica concernente il colon.

E.1.1.1

Medical condition in easily understood language

Bowel cleansing before diagnostic or therapeutic procedures concerning colon.

Pulizia intestinale prima di una qualsiasi procedura diagnostica o terapeutica concernente il colon.

E.1.1.2

Therapeutic area

Not possible to specify

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	18.0
E.1.2	Level	LLT
E.1.2	Classification code	10071002
E.1.2	Term	Rectal cleansing
E.1.2	System Organ Class	100000004865

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To evaluate the efficacy of PMF104 compared to KLEAN-PREP in children aged from 2 to less than 18 years old requiring a diagnostic procedure of the colon

Valutare l'efficacia di PMF104 rispetto a KLEAN-PREP in bambini di età compresa tra 2 e meno di 18 anni con l'indicazione ad effettuare una procedura diagnostica a livello del colon

E.2.2

Secondary objectives of the trial

To evaluate the efficacy, safety, tolerability, acceptability and palatability of PMF104 compared to KLEAN-PREP in children aged from 2 to less than 18 years old requiring a diagnostic procedure of the colon

Valutare l'efficacia, la sicurezza, la tollerabilità, l'accettabilità e la palatabilità di PMF104 rispetto a KLEAN-PREP in bambini di età compresa tra 2 e meno di 18 anni con l'indicazione ad effettuare una procedura diagnostica a livello del colon

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Male and female patients aged from 2 to less than 18 years undergoing elective colonoscopy
2. Female subjects currently either of:
non-childbearing potential (i.e., physiologically incapable of becoming pregnant, including any female who is surgically sterilised via documented hysterectomy or bilateral tubal ligation),
or
child bearing potential: the subject is eligible to enter and participate in this study if she is not lactating and has a negative pregnancy test (only menstruated females)
3. Patients whose parent(s)/legal representative have been thoroughly informed of the aim of the study procedures and provided signed and dated written informed consent
4. Children aged from 6 to less than 12 years providing informed assent whenever possible
5. Adolescents aged from 12 to less than 18 years providing informed assent
6. Ability of the subjects and/or their parents/legal representatives to understand and comply with protocol requirements, instructions and protocol-stated restrictions

1. Maschi e femmine di età compresa fra 2 e meno di 18 anni con l'indicazione ad effettuare una colonscopia
2. Femmine non fertili (cioè fisiologicamente incapaci di procreare, incluse femmine sterilizzate chirurgicamente tramite isterectomia o legatura bilaterale delle tube) o, se fertili, non in allattamento e con un test di gravidanza negativo (solo per le pazienti mestruate)
3. Pazienti il/i cui genitore/i o legale rappresentante sia stato informato riguardo a tutte le procedure di studio e abbia fornito il consenso informato scritto
4. Bambini di età compresa tra 6 e meno di 12 anni che abbiano fornito l'assenso informato, quando possibile
5. Adolescenti da 12 a meno di 18 anni che abbiano fornito l'assenso informato
6. Capacità dei soggetti e/o dei loro genitori/legale rappresentante di comprendere ed attenersi alle richieste, istruzioni e restrizioni previste dal protocollo

E.4

Principal exclusion criteria

1. Requirement for urgent colonoscopy
2. Gastrointestinal obstruction or perforation
3. Bowel pseudo-obstruction
4. Gastric retention
5. Toxic colitis
6. Toxic megacolon
7. Known or suspected hypersensitivity to the active or other ingredients of both test product and reference product
8. Clinically significant electrolyte imbalance
9. Prior intestinal resection
10. Structural abnormality of the lower gastrointestinal (GI) tract
11. Known metabolic (particularly phenylketonuria), renal or cardiac disease
12. Known pregnancy
13. Subject who have participated in another clinical trial or have taken an investigational drug within the last 3 months prior screening

1. Indicazione ad una colonscopia urgente
2. Ostruzione o perforazione gastrointestinale
3. Pseudo-ostruzione dell'intestino
4. Ritenzione gastrica
5. Colite tossica
6. Megacolon tossico
7. Ipersensibilità nota o sospetta al principio attivo o agli altri ingredienti del test e del reference
8. Squilibrio elettrolitico clinicamente significativo
9. Precedente resezione intestinale
10. Anomalia strutturale del basso tratto gastrointestinale
11. Patologie metaboliche (in particolare fenilchetonuria), renali o cardiache
12. Gravidanza nota
13. Soggetti che hanno partecipato ad un altro studio clinico o hanno assunto un altro trattamento sperimentale nei tre mesi precedenti lo screening

E.5 End points

E.5.1

Primary end point(s)

To compare the efficacy in terms of colon cleansing according the Boston Bowel Preparation Scale (BBPS) score on the day of the diagnostic procedure, to be determinated blindly by the endoscopist upon completion of the examination

Comparazione dell'efficacia in termini di pulizia del colon, in accordo alla scala di Boston per la preparazione dell'intestino, valutata dall'endoscopista (che effettua la colonscopia in cieco rispetto al trattamento) al termine della colonscopia stessa

E.5.1.1

Timepoint(s) of evaluation of this end point

Upon completion of the examination

Al completamento della colonscopia

E.5.2

Secondary end point(s)

1. Efficacy:
- Time to reach clear watery stools
- Proportion of patients in whom caecal intubation was achieved during colonoscopy
- Proportion of patients achieving a BBPS score grater than or equal to 5
2. Proportion of patients that needed a rescue dose
3. Safety assessment
4. Tolerability assessment
5. Compliance assessment
6. Acceptability assessment
7. Palatability assessment

1. Efficacia:
- Tempo necessario per ottenere feci chiare ed acquose
- Proporzione di pazienti in cui è stata ottenuta l'intubazione cecale
- Proporzione di pazienti con uno score BBPS maggiore o uguale a 5
2. Proporzione di pazienti che hanno necessitato di una rescue dose
3. Valutazione della sicurezza
4. Valutazione della tollerabilità
5. Valutazione della compliance
6. Valutazione dell'accettabilità
7. Valutazione della palatabilità

E.5.2.1

Timepoint(s) of evaluation of this end point

During /after the treatment

Durante/dopo il trattamento

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

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

Yes

E.6.13.1

Other scope of the trial description

Tolerability, acceptability and palatability

Tollerabilità, accettabilità e palatabilità

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

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

Yes

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)

Yes

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

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in 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

Ultima visita dell'ultimo paziente

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:

430

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)

Yes

F.1.1.5.1

Number of subjects for this age range:

262

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

168

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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2015-09-18.

Yes

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Preschool minors

Minori in età prescolare

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

430

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

Nessuno

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

2015-12-11

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2015-10-22

P. End of Trial
P.	End of Trial Status	Prematurely Ended

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
Clear advanced search filters

Austria
Belgium
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Estonia
Finland
France
Germany
Greece
Hungary
Iceland
Ireland
Italy
Latvia
Liechtenstein
Lithuania
Luxembourg
Malta
Netherlands
Norway
Poland
Portugal
Romania
Slovakia
Slovenia
Spain
Sweden
United Kingdom
Outside EU/EEA

Clinical trials