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

Study Overview
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Brief Summary
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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
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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.

Official Title
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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

Conditions
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Pancreatic Exocrine Insufficiency

Intervention / Treatment
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* Drug: Pancreatin


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Maximum Age: 4 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Creon micro, minimicrospheres<br> | Drug: Pancreatin<br>* 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<br>* Other names: Creon micro;|

What is the study measuring?
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Primary Outcome Measures

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

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Subjects With Adverse Events |  | 4 months | 
| Pulse | Change from Baseline at Day 84 | 3 months | 
| Number of Participants With Findings During Physical Examination | 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. | 3 months | 
| Number of Participants With Clinical Relevant Safety Laboratory Values | (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) | 3 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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cystic fibrosis