Evaluation of Safety and Tolerability of Creon Micro in Children Younger Than Four Years With Pancreatic Exocrine Insufficiency

An Open Label, Multicenter Study Evaluating Safety and Tolerability of Creon Micro in Pediatric Subjects From at Least One Month to Less Than Four Years of Age With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01747330

Enrollment

Registered

2012-12-11

Start date

2012-06-30

Completion date

2012-12-31

Last updated

2014-07-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pancreatic Exocrine Insufficiency

Keywords

cystic fibrosis

Brief summary

The purpose of this study is to evaluate the safety and tolerability of Creon micro in children younger than four years with pancreatic exocrine insufficiency.

Detailed description

A prospective open-label, multi-center study evaluating safety and tolerability of Creon Micro in pediatric subjects from at least one month to less than four years of age with Pancreatic Exocrine Insufficiency (PEI) due to Cystic Fibrosis (CF). Referring to the European Medicines Agency (EMEA) pediatric age criteria for the purpose of the study, subjects are categorized as infants and toddlers (\< 2 years) and children, (2 years to \< 4 years). In total, 40 subjects will be enrolled (20 subjects in each age subgroup). Subjects receive their prescribed pancreatic enzyme supplementation at the individual dose until start of treatment with Creon Micro, if the subjects were on pancreatic enzyme supplementation before start of the study. Subjects will be treated with Creon Micro during the study. The treatment duration is 3 months.

Interventions

DRUGPancreatin

Doses of Pancreatin \<2500 lipase u/kg/feed or \<4000 lipase U/g fat/intake or \<10000 lipase U/kg/day given orally are used

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

No minimum to 4 Years

Healthy volunteers

Inclusion criteria

* Children younger than four years with confirmed cystic fibrosis diagnosis and a body weight of at least 2 kilograms

Exclusion criteria

* Ileus or acute abdomen * history of fibrosing colonopathy * history of distal ileal obstruction without surgery * solid organ transplant or surgery affecting the large bowel, other than appendectomy

Design outcomes

Primary

Measure	Time frame	Description
Subject's Acceptance of Treatment	3 months	Acceptance to Creon Micro. The caregiver should give his/her opinion based on the following scale: very good, good, moderate, and unsatisfactory.
Body Weight	3 months	change from baseline at day 84
Height	3 months	change from baseline at day 84
Stool Frequency	3 months	Average daily stool frequency during treatment period: Number of bowel movements per day
Stool Consistency	3 months	Assessment of stool consistency by the caregiver on a daily basis: hard, formed/normal, soft, watery

Secondary

Measure	Time frame	Description
Pulse	3 months	Change from Baseline at Day 84
Number of Participants With Findings During Physical Examination	3 months	A physical examination was conducted by the physician. All abnormal findings were recorded as medical histories if present prior to start of study drug or as AEs otherwise. There was no separate documentation of physical examination findings in this study.
Number of Participants With Clinical Relevant Safety Laboratory Values	3 months	(hematology: hemoglobin, hematocrit, RBC count, WBC count, platelet count; biochemistry: glucose (fasting), creatinine, alkaline phosphatase, total bilirubin, ALAT (alanine amino transferase), ASAT (aspartate amino transferase), gamma-glutamyl transferase, uric acid, calcium, phosphate, potassium, serum pancreatic lipase; urinalysis (dipstick): glucose, blood, albumin, pH)
Number of Subjects With Adverse Events	4 months	—

Countries

Russia

Participant flow

Participants by arm

Arm	Count
Creon Micro, Minimicrospheres Pancreatin: Doses of pancreatin \<2500 lipase u/kg/feed or \<4000 lipase u/g fat/intake or \<10000 lipase u/kg/day given orally are used	40
Total	40

Baseline characteristics

Characteristic	Creon Micro, Minimicrospheres
Age, Categorical <=18 years	40 Participants
Age, Categorical >=65 years	0 Participants
Age, Categorical Between 18 and 65 years	0 Participants
Age, Continuous	1.7 years STANDARD_DEVIATION 1.2
Region of Enrollment Russian Federation	40 participants
Sex: Female, Male Female	13 Participants
Sex: Female, Male Male	27 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	— / —
other Total, other adverse events	12 / 40
serious Total, serious adverse events	0 / 40

Outcome results

Primary