RESULT: Reliable, Emergent Solution Using Liprotamase Treatment

Study Overview
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Brief Summary
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Liprotamase powder is a non-porcine, soluble and stable mixture of biotechnology-derived lipase, protease, and amylase digestive enzymes. The purpose of the present study is to to evaluate the non-inferiority of liprotamase compared with porcine-derived, enterically-coated pancreatic enzyme replacement therapy (PERT). The primary efficacy endpoint of the study will be comparative efficacy measured as the change from baseline in the coefficient of fat absorption (CFA) in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI).

Official Title
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A Phase 3, Randomized, Open-Label, Assessor-Blind, Non-Inferiority, Active-Comparator Study Evaluating the Efficacy and Safety of Liprotamase in Subjects With Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency

Conditions
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Exocrine Pancreatic Insufficiency, Cystic Fibrosis

Intervention / Treatment
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* Drug: Liprotamase
* Drug: porcine PERT


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of Cystic Fibrosis based on presentation, genotype and/or sweat chloride Fecal elastase <100 mcg/g stool Good disease control with porcine PERT prior to enrollment Good nutritional status Exclusion Criteria: History or diagnosis of fibrosing colonopathy Distal intestinal obstruction syndrome in 6 months prior to screening Receiving enteral tube feedings Chronic diarrheal illness unrelated to pancreatic insufficiency Liver abnormalities, or liver or lung transplant, or significant bowel resection Forced expiratory volume in 1 second (FEV1) <30%

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: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Liprotamase<br>Individually-optimized dose to be administered orally | Drug: Liprotamase<br>* oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement<br>|
| Active Comparator: porcine PERT<br>Individually-optimized dose to be administered orally | Drug: porcine PERT<br>* oral, enterically-coated, pig-derived, pancreatic enzyme replacement<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Coefficient of Fat Absorption (CFA) | Non-inferiority of Liprotamase to approved porcine PERT | 8 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Coefficient of Nitrogen Absorption (CNA) | Non-inferiority of Liprotamase to approved porcine PERT | 8 weeks | 
| Safety, as measured by number of participants with adverse events or laboratory abnormalities | Change from baseline | 6 months |