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

Study Overview
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Brief Summary
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This study will assess the effect of pancrelipase delayed release 12,000 unit capsules on fat and nitrogen absorption in subjects 7 - 11 with pancreatic exocrine insufficiency due to Cystic Fibrosis.

Official Title
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A Double-blind, Randomized, Multi-center, Placebo-controlled, Cross-over Study to Assess the Efficacy and Safety of Pancrelipase Delayed Release 12,000 Unit Capsules in Subjects Aged 7 - 11 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
* Drug: Placebo Comparator


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Confirmed CF diagnosis by two positive chloride sweat tests or gene analysis Confirmed PEI by historical Coefficient of fat Absorption < 70% without supplementation or current or historical fecal elastase < 50µg/stool (within the last 12 months) 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 Stable body weight and agrees to abstain from sexual activity 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: 7 Years
Maximum Age: 11 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: Randomized
Intervention Model: Crossover Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: A<br> | Drug: Pancrelipase Delayed Release<br>* 12,000 unit Capsules, dosed individually based on fat intake.<br>|
| Placebo Comparator: B<br> | Drug: Placebo Comparator<br>* Placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Coefficient of Fat Absorption (%) | This coefficient is calculated from fat intake and fat excretion : 100*[fat intake-fat excretion]/fat intake. Stools were collected on 3 days during the 5 days treatment period. Higher values indicate a better response. | 5 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Coefficient of Nitrogen Absorption (%) | This coefficient is calculated from nitrogen intake and nitrogen excretion : 100*[nitrogen intake-nitrogen excretion]/nitrogen intake. Stools were collected on 3 days during the 5 days treatment period. Higher values indicate a better response. | 5 days | 
| Total Fat Excretion (Grams) | Total amount of fat excreted during the stool collection period. Stools were collected on 3 days during the 5 days treatment period. Lower values indicate a better response. | 5 days | 
| Total Stool Weight (Grams) | Total weight of the stools collected during the stool collection period. Stools were collected on 3 days during the 5 days treatment period. Lower values indicate a better response. | 5 days | 
| Stool Frequency | Stool frequency is the average of the daily number of stools recorded during the treatment period. Lower values indicate a better response. | 5 days | 
| Percentage of Days With no Flatulence. | The percentage of days with no flatulence is calculated from the diary during the treatment period: 100*(number of days with no flatulence/number of days recorded in diary). Higher values indicate a better response. | 5 days | 
| Percentage of Days With Formed/Normal Stools. | The percentage of days with formed/normal stools is calculated from the diary during the treatment period: 100*(number of days with formed/normal stools/number of days with any stool). Higher values indicate a better response. | 5 days | 
| Percentage of Days With no Abdominal Pain. | The percentage of days with no abdominal pain is calculated from the diary during the treatment period: 100*(number of days with no abdominal pain / number of days recorded in diary). Higher values indicate a better response. | 5 days | 

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