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

Phase 3

Completed

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.

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
Creon micro, minimicrospheres	Pancreatin	-

Primary Outcome Measures

Name	Time	Method
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
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.

Secondary Outcome Measures

Name	Time	Method
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 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 Subjects With Adverse Events	4 months
Pulse	3 months	Change from Baseline at Day 84

Locations (9): Site Reference ID/Investigator# 80715
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Orenburg, Russian Federation
Site Reference ID/Investigator# 80716
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Barnaul, Russian Federation
Site Reference ID/Investigator# 80698
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Novosibirsk, Russian Federation
Site Reference ID/Investigator# 80713
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Novosibirsk, Russian Federation
Site Reference ID/Investigator# 80693
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Moscow, Russian Federation
Site Reference ID/Investigator# 80694
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St. Petersburg, Russian Federation
Site Reference ID/Investigator# 80714
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Tomsk, Russian Federation
Site Reference ID/Investigator# 80697
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Voronezh, Russian Federation
Site Reference ID/Investigator# 80696
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Yaroslavl, Russian Federation