Study Investigating a Delayed-Release Pancrelipase in Patients With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis

Study Overview
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Brief Summary
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This study will assess the safety and tolerability of pancrelipase delayed release capsules in subjects up to 6 years of age with Pancreatic Exocrine Insufficiency (PEI) due to Cystic Fibrosis.

Official Title
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An Open Label, Multi-center, Study to Assess the Safety and Tolerability of Pancrelipase Delayed Release Capsules in Infants and Children Less Than 7 Years of Age With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis

Conditions
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Cystic Fibrosis, Pancreatic Exocrine Insufficiency

Intervention / Treatment
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* Drug: Pancrelipase Delayed Release


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Confirmed Cystic Fibrosis (CF) diagnosis by two positive chloride sweat tests or gene analysis Current or historical human fecal elastase < 50µg/gstool Weight greater than 3.75 kg Age 1 month to 6 years Currently receiving treatment with a commercially available pancreatic enzyme product on a stable dose for more than 3 months Clinically stable condition without evidence of acute respiratory disease or any other acute condition Exclusion Criteria: Ileus or acute abdomen History of fibrosing colonopathy, Celiac disease, gastrectomy, Crohn´s disease and small bowel surgery other than minor resection due to meconium ileus without resulting in malabsorption syndrome History of distal ileal obstruction syndrome within 6 months of enrollment Use of an immunosuppressive drug Any type of malignancy involving the digestive tract in the last 5 years Known infection with HIV

Ages Eligible for Study
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Minimum Age: 1 Month
Maximum Age: 6 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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: A<br> | Drug: Pancrelipase Delayed Release<br>* 3,000, 6,000 and 12,000 unit Lipase Capsules<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Patients With at Least One Treatment Emergent Adverse Event (TEAE) | Treatment Emergent Adverse Events are defined as adverse events started at or after the first administration of study drug and include those events started prior to the first administration but which worsened after the first intake. | 10 Days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Stool Fat (% Fat) | The stool fat content was calculated as percent fat of dry solid weight per bowel movement. Stool fat per patient was derived as mean over three bowel movements sampled (i.e. one sample per day). | Last 3 days in a 10-day treatment period | 
| Fat Intake (g) | The mean daily fat intake was determined as the average of daily fat intake over a 3-day period. | Last 3 days in a 10-day treatment period | 
| Total Calorie Intake (kcal) | The total calorie intake was determined as the average of total calorie of daily food intake intake over a 3-day period. | Last 3 days in a 10-day treatment period | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cystic Fibrosis, Pancreatic Exocrine Insufficiency