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

Bowel Preparation Before Colonoscopy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03106922
• Other study ID #: PMF104 PD1-2-3/2013
• Status: Recruiting
• Phase: Phase 3
• Start date: April 2016
• Est. completion date: July 2020

Study information

• Verified date: April 2017
• Source: Alfasigma S.p.A.
• Contact: Raffaella Tacchi, MD
• Phone: +39 051 6489867
• Email: rtacchi[@]alfawassermann.it
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 430
• Est. completion date: July 2020
• Est. primary completion date: July 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 17 Years

Eligibility

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.

Related Conditions & MeSH terms

• Bowel Preparation Before Colonoscopy

Intervention

Drug:
• 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
• 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

Locations

Country	Name	City	State
Belgium	Pediatric Gastroenterology, Hepatology and Nutrition Children's University Hospital	Brussel
France	Hôpital Jeanne de Flandre	Lille
France	University and Pediatric Hospital of Lyon	Lyon
France	Hôpital Armand-Trousseau	Paris
Italy	Ospedale Maggiore "C.A. Pizzardi" AUSL Bologna	Bologna
Italy	Azienda Ospedaliero-Universitaria Meyer	Florence
Italy	Azienda Ospedaliera Universitaria "G. Martino"	Messina
Italy	Azienda Policlinico Umberto I	Rome
Italy	Ospedale Pediatrico Bambino Gesù	Rome

Sponsors (1)

Lead Sponsor	Collaborator
Alfasigma S.p.A.

Countries where clinical trial is conducted

Belgium,  France,  Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Colon cleansing efficacy	Boston Bowel Preparation Scale score assessed blindly by the endoscopist	Visit 3 (Day 2)
Secondary	Time to reach clear watery stools	The time when clear watery stools are obtained after completing the bowel solution	Day 1
Secondary	Proportion of patients in whom caecal intubation was achieved during colonoscopy	Proportion of patients in whom caecal intubation was achieved during colonoscopy	Visit 3 (Day 2)
Secondary	Proportion of patients achieving a BBPS score greater than or equal to 5	Based on the BBPS score assigned during colonoscopy, each patient will be classified as being a "success" (patient achieving a BBPS score = 5) or a "failure" (patient achieving a BBPS score < 5).	Visit 3 (Day 2)
Secondary	Proportion of patients that needs a rescue dose	Additional amount of test or reference will be given if patient will not have clear watery stools 3 hours after the administration of the scheduled amount of bowel solution	Day 1
Secondary	Compliance	Poor: intake of < 75% of the solution; Good: intake of at least 75% of the solution but <100%; Optimal: intake of the whole solution.	Day 1
Secondary	Acceptability	Pre-determined answers to question:"How difficult was to drink the bowel preparation solution?" 1 =Very difficult; 2 =Moderately difficult; 3 = Slightly difficult; 4 =Not difficult at all	Day 1
Secondary	Palatability	Pre-determined answers to question:"How did the solution taste?" 1 =Very bad; 2 = Bad; 3 =Good; 4 = Very good	Day 1
Secondary	Number of adverse events	Number of adverse events coded by System Organ Class (SOC) and Preferred Term (PT), using the Medical Dictionary for Regulatory Activities (MedDRA).	From Visit 1 to Follow-up telephone contact
Secondary	Subjects withdrawn due to treatment emergent adverse events (TEAEs)	Number and percentage of patients withdrawn due to TEAEs	From Visit 1 to Follow-up telephone contact
Secondary	Tolerability Assessment	Recording of occurence and severity of nausea, vomiting, bloating, abdominal pain/cramps, anal irritation and fatigue/weakness, according to a 4 point scale: 0 =No distress; =Mild distress; =Moderate distress; = Severe distress	Visit 3 (Day 2)