Clinical Trials Register

Summary
EudraCT Number:	2015-002969-27
Sponsor's Protocol Code Number:	PMF104PD1-2-3/2013
National Competent Authority:	Belgium - FPS Health-DGM
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2017-04-11
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

Belgium - FPS Health-DGM

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
(EMA Decision P/019/2017 on the acceptance of modification of Paediatric Investigation Plan)

Een gerandomiseerd, enkelblind, actief gecontroleerd, multicentrisch onderzoek ter evaluatie van de doeltreffendheid, veiligheid, verdraagbaarheid, aanvaardbaarheid en smakelijkheid van PMF104 in vergelijking met een conventionele PEG-elektrolytenoplossing bij kinderen van 2 tot 6 jaar, van 6 tot 12 jaar en adolescenten van 12 tot 18 jaar die een diagnostische procedure inzake de dikke darm vereisen

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

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/223/2018

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Alfasigma 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	Alfasigma 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	AML Clinical Services BVBA
B.5.2	Functional name of contact point	Clinical Operations
B.5.3	Address:
B.5.3.1	Street Address	Tempelzicht 8
B.5.3.2	Town/ city	Linden
B.5.3.3	Post code	B-3210
B.5.3.4	Country	Belgium
B.5.4	Telephone number	+32(0)16782170
B.5.5	Fax number	+32(0)16788672
B.5.6	E-mail	C125-AML@aml-research.be

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	Yes
D.2.1.1.1	Trade name	Clensia®
D.2.1.1.2	Name of the Marketing Authorisation holder	Alfasigma S.p.A. (Italy)
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.3.4	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
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.

E.1.1.1

Medical condition in easily understood language

Bowel cleansing before diagnostic or therapeutic procedures concerning colon.

E.1.1.2

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10066943
E.1.2	Term	Bowel preparation
E.1.2	System Organ Class	10042613 - Surgical and medical procedures

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

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

Het evalueren van de doeltreffendheid, veiligheid, verdraagbaarheid, aanvaardbaarheid en smakelijkheid van PMF104 bij pediatrische proefpersonen die een diagnostische procedure van de dikke darm nodig hebben

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) and agrees to abstain from intercourses until the colonoscopy is performed
3. Patients whose parents/legal representatives 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. Mannelijke en vrouwelijke patiënten van 2 tot 18 jaar oud tijdens de hele onderzoeksperiode en die electieve colonoscopie ondergaan
2. Vrouwelijke proefpersonen die momenteel ofwel:
niet vruchtbaar zijn (d.w.z. fysiologisch niet in staat zwanger te worden, waaronder alle vrouwen die chirurgisch gesteriliseerd werden via gedocumenteerde hysterectomie of bilaterale tubaligatie),
of
die vruchtbaar zijn: de proefpersoon komt in aanmerking om zich in dit onderzoek in te schrijven en eraan deel te nemen als zij geen borstvoeding geeft en een negatieve zwangerschapstest heeft (enkel vrouwen met menstruatie), en stemt ermee in zich te onthouden van geslachtsgemeenschap tot na de colonoscopie is uitgevoerd
3. Patiënten wiens ouders/wettelijke vertegenwoordigers grondig geïnformeerd werden over het doel van de onderzoeksprocedures en die een ondertekende en gedateerde schriftelijke geïnformeerde toestemming hebben gegeven
4. Kinderen van 6 tot 12 jaar die waar mogelijk geïnformeerde instemming hebben gegeven
5. Kinderen van 12 tot 18 jaar die geïnformeerde instemming hebben gegeven
6. Vermogen van de proefpersonen/of hun ouders/wettelijke vertegenwoordigers om de vereisten van het protocol, de instructies en de protocol-gerelateerde beperkingen te begrijpen en na te leven.

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), hepatic, renal or cardiac disease
12. Congestive heart failure (NYHA class III and IV)
13. Known pregnancy
14. Subject who have participated in another clinical trial or have taken an investigational drug within the last 3 months prior screening

1. Vereiste voor dringende colonoscopie
2. Gastro-intestinale blokkering of perforatie
3. Pseudo-obstructie van de darmen
4. Gastrische retentie
5. Toxische colitis
6. Toxische megacolon
7. Gekende of vermeende overgevoeligheid voor de actieve of andere bestanddelen van zowel het test- als referentieproduct
8. Klinisch significante ontregeling van de elektrolytbalans
9. Eerdere intestinale resectie
10. Structurele afwijking van het onderste gastro-intestinale (GI) kanaal
11. Gekende metabolische (in het bijzonder fenylketonurie), lever-, nier- of hartziekte
12. Congestief hartfalen (NYHA klasse III en IV)
13. Gekende zwangerschap
14. De proefpersoon heeft deelgenomen aan een ander klinisch onderzoek of heeft in de 3 maanden voorafgaand aan de screening een onderzoeksgeneesmiddel ingenomen.

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

E.5.1.1

Timepoint(s) of evaluation of this end point

Upon completion of the examination (Visit 3, Day 2)

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. Doeltreffendheid:
- Tijd tot het bereiken van heldere waterige stoelgang
- Aandeel patiënten bij wie intubatie van de blinde darm bereikt werd tijdens de colonoscopie
- Aandeel patiënten die een BBPS-score ≥ 5 behalen
2. Aandeel patiënten die een dosis reddingsmedicatie nodig hebben
3. Beoordelingen voor veiligheid
4. Beoordelingen voor verdraagbaarheid
5. Beoordelingen voor naleving
6. Beoordelingen voor aanvaardbaarheid
7. Beoordelingen voor smakelijkheid

E.5.2.1

Timepoint(s) of evaluation of this end point

During /after the treatment

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

Verdraagbaarheid, aanvaardbaarheid en smakelijkheid

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

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)

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

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.5

The trial involves multiple Member States

Yes

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

Last Visit Last Subject

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

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial 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:

396

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:

228

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 2017-04-11.

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

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

396

F.4.2.2

In the whole clinical trial

396

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

2017-05-04

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-06-21

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2021-03-05

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