Clinical trial NCT03106922: A Trial to Evaluate the Efficacy and Safety of PMF104 Compared to a Conventional PEG-electrolyte Solution in Children
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
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Bowel Preparation Before Colonoscopy
Keywords: Polyethylene glycol
Bowel cleansing
Colonoscopy
Lead sponsor: Alfasigma S.p.A.
Sponsor class: INDUSTRY
First posted: 2017-04-11
Enrollment: 430
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Colon cleansing efficacy
timeFrame: Visit 3 (Day 2)
Secondary outcomes:
measure: Time to reach clear watery stools
timeFrame: Day 1
measure: Proportion of patients in whom caecal intubation was achieved during colonoscopy
timeFrame: Visit 3 (Day 2)
measure: Proportion of patients achieving a BBPS score greater than or equal to 5
timeFrame: Visit 3 (Day 2)
measure: Proportion of patients that needs a rescue dose
timeFrame: Day 1
measure: Compliance
timeFrame: Day 1
measure: Acceptability
timeFrame: Day 1
measure: Palatability
timeFrame: Day 1
measure: Number of adverse events
timeFrame: From Visit 1 to Follow-up telephone contact
measure: Subjects withdrawn due to treatment emergent adverse events (TEAEs)
timeFrame: From Visit 1 to Follow-up telephone contact
measure: Tolerability Assessment
timeFrame: Visit 3 (Day 2)
Other outcomes:
Locations:
city: Brussel
country: Belgium
facility: Pediatric Gastroenterology, Hepatology and Nutrition Children's University Hospital
city: Lille
country: France
facility: Hôpital Jeanne de Flandre
city: Lyon
country: France
facility: University and Pediatric Hospital of Lyon
city: Paris
country: France
facility: Hôpital Armand-Trousseau
city: Bologna
country: Italy
facility: Ospedale Maggiore "C.A. Pizzardi" AUSL Bologna
city: Florence
country: Italy
facility: Azienda Ospedaliero-Universitaria Meyer
city: Messina
country: Italy
facility: Azienda Ospedaliera Universitaria "G. Martino"
city: Rome
country: Italy
facility: Azienda Policlinico Umberto I
city: Rome
country: Italy
facility: Ospedale Pediatrico Bambino Gesù
Study officials:
affiliation: Azienda Policlinico Umberto I
name: Salvatore Cucchiara, MD
role: PRINCIPAL_INVESTIGATOR
References: