A Trial to Evaluate the Efficacy and Safety of PMF104 Compared to a Conventional PEG-electrolyte Solution in Children

NCT ID: NCT03106922

Last Updated: 2018-09-28

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE3
• Total Enrollment: 430 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-04-30
• Study Completion Date: 2020-07-31

Brief Summary

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 paediatric subjects requiring a diagnostic procedure concerning the colon.

Detailed Description

The aim of the study is 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.

Patients will be stratified according the following age strata:

• Children from 2 to less than 6 years of age (PMF104 PD1/13)
• Children from 6 to less than 12 years of age (PMF104 PD2/13)
• Children from 12 to less than 18 years of age (PMF104 PD3/13) The study will consist of a Screening Visit (V1), a Randomisation Visit (V2), a Drug Administration (Day 1), a Diagnostic Procedure (i.e. colonoscopy; V3) and a Follow-up Telephone Contact (FU).

At Screening Visit (V1) children will be evaluated for study eligibility, up to 11 days before the colonoscopy.

At Randomisation Visit (V2), if eligible, they will be randomised according to a single blind randomisation schedule in a 1:1 ratio to PMF104 or Klean-prep®; they will also receive the instructions to be followed for bowel preparation, and the questionnaire to be answered on the day of the bowel preparation. In case the Investigator will consider it as appropriate, the child will be hospitalised in the clinical facility the day before the colonoscopy in order to perform bowel preparation.

The day before the colonoscopy study drug will be administered orally starting in the mid-late afternoon (4-6 p.m.), as single dose.

The day of colonoscopy the diagnostic procedure will be performed blindly by an endoscopist unaware of the treatment administered to the subject, and both primary and secondary endpoints will be assessed.

On day 31 ± 2 (i.e. 30 ± 2 days after the day of the bowel preparation intake), the subjects will be contacted by a telephone call, to inquire about possible adverse events occurred after hospital discharge.

Conditions

Bowel Preparation Before Colonoscopy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: SINGLE

Study Groups

PMF104

The day before the colonoscopy, starting in the mid-late afternoon (4-6 p.m.), by oral route:

2<=Age<6

• 500 ml in 1-1.5 hours <= to 18 kg
• 625 ml in 1-1.5 hours >18 kg 6<=Age<12:
• 750 ml in 1-2 hours <=25 kg
• 1000 ml in 1-2 hours 25-35 kg
• 1250 ml in 1-2 hours >35 kg 12>=Age<18 :
• 1500 ml in 2-3 hours <= 45 kg
• 1750 ml in 2-3 hours>45 kg.

Rescue dose (if no clear watery stools 3 hours after the entire solution):

• 250 ml 2 Age <=6;
• 500 ml 6 <=Age<12; up to a cumulative maximum volume of 2000 ml 12<=Age<18.

Group Type: EXPERIMENTAL

PMF104

Intervention Type: DRUG

Powder for oral solution:

• Sachet A: macrogol 4000 52.500 g, anhydrous sodium sulphate 3.750 g, simeticone 0.080 g;
• Sachet B: sodium citrate 1.863 g, anhydrous citric acid 0.813 g, sodium chloride 0.730 g, potassium chloride 0.370 g;

One sachet A + 1 sachet B in 500 ml of water

Klean- prep

The day before the colonoscopy, starting in the mid-late afternoon (4-6 p.m.), by oral route:

• 2<=Age<6:

• 90 ml/kg in 1-1.5 hours 2<=Age<6
• 80 ml/kg in 1-1.5 hours 5<=Age<6
• 2<=Age<6:

• 80 ml/kg in 1-2 hours 6<=Age<10
• 70 ml/kg in 1-2 hours 10<=Age<12
• 12<=Age<18:

• 70 ml/kg in 2-3 hours. Rescue dose (if no clear watery stools 3 hours after the entire Klean-Prep solution): 50% of the initial dose.

Group Type: ACTIVE_COMPARATOR

Klean-prep

Intervention Type: DRUG

Powder for oral solution:

macrogol 3350 59.000 g, anhydrous sodium sulphate 5.685 g, sodium bicarbonate 1.685 g, sodium chloride 1.465 g, potassium chloride 0.7425 g.

One sachet to be dissolved in 1 L of water

Interventions

PMF104

Powder for oral solution:

• Sachet A: macrogol 4000 52.500 g, anhydrous sodium sulphate 3.750 g, simeticone 0.080 g;
• Sachet B: sodium citrate 1.863 g, anhydrous citric acid 0.813 g, sodium chloride 0.730 g, potassium chloride 0.370 g;

One sachet A + 1 sachet B in 500 ml of water

Intervention Type: DRUG

Klean-prep

Powder for oral solution:

macrogol 3350 59.000 g, anhydrous sodium sulphate 5.685 g, sodium bicarbonate 1.685 g, sodium chloride 1.465 g, potassium chloride 0.7425 g.

One sachet to be dissolved in 1 L of water

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Male and female patients aged from 2 to less than 18 years during the entire study period 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
• Childbearing potential: the subject is eligible to enter and participate in this study if she is not lactating and has a negative pregnancy test, and agrees to abstain from intercourses until the colonoscopy is performed (only menstruated females);

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.

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.

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Alfasigma S.p.A.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Salvatore Cucchiara, MD

Role: PRINCIPAL_INVESTIGATOR

Azienda Policlinico Umberto I

Locations

Pediatric Gastroenterology, Hepatology and Nutrition Children's University Hospital

Brussels, , Belgium

Site Status: NOT_YET_RECRUITING

Hôpital Jeanne de Flandre

Lille, , France

Site Status: NOT_YET_RECRUITING

University and Pediatric Hospital of Lyon

Lyon, , France

Site Status: NOT_YET_RECRUITING

Hôpital Armand-Trousseau

Paris, , France

Site Status: NOT_YET_RECRUITING

Ospedale Maggiore "C.A. Pizzardi" AUSL Bologna

Bologna, , Italy

Site Status: ACTIVE_NOT_RECRUITING

Azienda Ospedaliero-Universitaria Meyer

Florence, , Italy

Site Status: ACTIVE_NOT_RECRUITING

Azienda Ospedaliera Universitaria "G. Martino"

Messina, , Italy

Site Status: NOT_YET_RECRUITING

Azienda Policlinico Umberto I

Rome, , Italy

Site Status: RECRUITING

Ospedale Pediatrico Bambino Gesù

Rome, , Italy

Site Status: RECRUITING

Countries

Belgium; France; Italy

Central Contacts

Raffaella Tacchi, MD

Role: CONTACT

rtacchi@alfawassermann.it

+39 051 6489867

Michela Padovani, MSc

Role: CONTACT

mpadovani@alfawassermann.it

+39 051 6489599

Facility Contacts

Genevieve Veereman

Role: primary

gveereman@mail.com

Laurent Michaud, MD

Role: primary

Laurent.MICHAUD@CHRU-LILLE.FR

Alain Lachaux, MD

Role: primary

alain.lachaux@chu-lyon.fr

Julie Lemale, MD

Role: primary

julie.lemale@aphp.fr

Claudio Romano, MD

Role: primary

romanoc@unime.it

Salvatore Cucchiara, MD

Role: primary

salvatore.cucchiara@uniroma1.it

06 49979326

Luigi Dall'Oglio, MD

Role: primary

luigi.dalloglio@opbg.net

+39 0668592841

Other Identifiers

PMF104 PD1-2-3/2013

Identifier Type: -

Identifier Source: org_study_id