Oral TheraCLEC™ - Total in Cystic Fibrosis Subjects With Exocrine Pancreatic Insufficiency

Study Overview
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Brief Summary
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The purpose of this study is to determine the most effective dose of a new enzyme therapy on oral nutrient absorption in cystic fibrosis (CF) subjects with exocrine pancreatic insufficiency.

Official Title
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A Phase 2, Randomized, Double Blind, Parallel Dose Ranging Study of Oral TheraCLEC™ - Total in Cystic Fibrosis Subjects With Exocrine Pancreatic Insufficiency

Conditions
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Cystic Fibrosis, Pancreatic Insufficiency

Intervention / Treatment
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* Drug: Liprotamase
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Pancreatic insufficiency (PI) documented by fecal elastase <100 mcg/gram measured at screening Diagnosis of CF based upon the following criteria: two clinical features consistent with CF AND either genotype with two identifiable mutations consistent with CF OR sweat chloride > 60 mEq/L by quantitative pilocarpine iontophoresis Forced Expiratory Volume Over One Second (FEV1) ≥30% predicted Clinically stable with no evidence of acute upper or lower respiratory tract infection Exclusion Criteria: Pregnancy, breastfeeding or of childbearing potential and/or not willing to use accepted methods of birth control during enrollment in the study History of fibrosing colonopathy History of liver transplant or lung transplant Unable to discontinue enteral tube feedings during the study Subject weight <40 kg Known hypersensitivity to food additives Participation in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to screening Unable to discontinue the use of pancreatic enzymes for a 72-hour period or any other condition that the investigator believes would interfere with the intent of this study or would make study participation not in the best interest of the subject

Ages Eligible for Study
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Minimum Age: 7 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: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Low Liprotamase Dose<br>Liprotamase in a fixed combination of lipase (5,000 units), protease (5,000 units) and amylase (750 units) administered orally (one Size 5 capsule of liprotamase and five Size 2 capsules of placebo) with each of three meals and two snacks daily for 28 days | Drug: Liprotamase<br>* Administered orally as either Size 2 capsules (20,000 Units lipase; 20,000 Units protease; and 3,000 Units amylase) or Size 5 capsules (5,000 Units lipase; 5,000 Units protease; and 750 Units amylase)<br>* Other names: TheraCLEC - Total;Drug: Placebo<br>* Administered orally in either Size 2 or Size 5 capsules<br>|
| Experimental: Mid Liprotamase Dose<br>Liprotamase in a fixed combination of lipase (25,000 units), protease (25,000 units) and amylase (3,750 units) administered orally (one Size 5 capsule of liprotamase, one Size 2 capsule of liprotamase, and four Size 2 capsules of placebo) with each of three meals and two snacks daily for 28 days | Drug: Liprotamase<br>* Administered orally as either Size 2 capsules (20,000 Units lipase; 20,000 Units protease; and 3,000 Units amylase) or Size 5 capsules (5,000 Units lipase; 5,000 Units protease; and 750 Units amylase)<br>* Other names: TheraCLEC - Total;Drug: Placebo<br>* Administered orally in either Size 2 or Size 5 capsules<br>|
| Experimental: High Liprotamase Dose<br>Liprotamase in a fixed combination of lipase (100,000 units), protease (100,000 units) and amylase (15,000 units) administered orally (one Size 5 capsule of placebo and five Size 2 capsules of liprotamase) with each of three meals and two snacks daily for 28 days | Drug: Liprotamase<br>* Administered orally as either Size 2 capsules (20,000 Units lipase; 20,000 Units protease; and 3,000 Units amylase) or Size 5 capsules (5,000 Units lipase; 5,000 Units protease; and 750 Units amylase)<br>* Other names: TheraCLEC - Total;Drug: Placebo<br>* Administered orally in either Size 2 or Size 5 capsules<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline up to 19 days in Coefficient of fat absorption (CFA) |  | Baseline, up to 19 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline up to 19 day in Coefficient of nitrogen absorption (CNA) |  | Baseline, up to 19 days | 
| Change from baseline up to 19 days in stool weight |  | Baseline, up to 19 days | 
| Change from baseline to 29 day endpoint in Cystic Fibrosis Questionnaire (CFQ) scale scores |  | Baseline, 29 days | 
| Change from baseline up to 19 days in number of Stools |  | Baseline, up to 19 days | 
| Change from baseline to 28 days in maximum change in glucose response |  | Baseline through 28 days | 

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