SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis

Phase 3

Completed

Brief Summary: Liprotamase powder is a non-porcine, soluble and stable mixture of three digestive enzymes including lipase, protease, and amylase. The purpose of the present study is to provide additional efficacy and safety data compared to approved, porcine-derived, enterically-coated and encapsulated pancreatic enzyme replacement therapy. The primary efficacy endpoint of the study will be comparative efficacy measured as the change in the coefficient of fat absorption (CFA) in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI).

Liprotamase is stable in stomach and digestive fluids allowing administration in a variety of convenient formulations and with a number of foods without enteric coating.

Detailed Description: Porcine derived enzymes are used for pancreatic enzyme replacement therapy in patients with cystic fibrosis (CF). Liprotamase is a biotechnology-derived enzyme replacement without enteric coating. This is an open-label, assessor blind, parallel group, multicenter, international trial to evaluate the noninferiority of liprotamase and pancrelipase in CF patients aged ≥7 years with pancreatic insufficiency. Subjects were randomized to liprotamase or pancrelipase, dose-matched to pre-study lipase doses. The lower bound of the 95% confidence interval (CI) for noninferiority was -15% for treatment difference in change from baseline coefficient of fat absorption (CFA).

Recruitment & Eligibility

Inclusion Criteria

Diagnosis of Cystic Fibrosis based on presentation, genotype and/or sweat chloride
Fecal elastase <100 mcg/g stool
Minimum Coefficient of Fat (CFA) at screening while on stable PERT therapy
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%

Arm && Interventions

Group	Intervention	Description
porcine (pig) PERT	porcine (pig) PERT	Individually-optimized dose to be administered orally
Liprotamase	Liprotamase	Individually-optimized dose to be administered orally

Primary Outcome Measures

Name	Time	Method
Treatment Difference in Coefficient of Fat Absorption (CFA) Change From Baseline	Baseline, 7 weeks	The primary endpoint evaluates the difference between treatment arms in change from baseline in coefficient of fat absorption (CFA). As such, descriptive statistics for individual treatment arms are not provided in this measure, but are reported in the secondary endpoints

Secondary Outcome Measures

Name	Time	Method
Coefficient of Nitrogen Absorption (CNA)	Baseline, 7 weeks	Change from baseline in coefficient of nitrogen absorption
Coefficient of Fat Absorption (CFA)	Baseline, 7 weeks	Change from baseline in coefficient of fat absorption

Locations (54): Investigator Site 123
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Long Beach, California, United States
Investigator Site 107
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Los Angeles, California, United States
Investigator Site 114
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Aurora, Colorado, United States
Investigator Site 120
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Gainesville, Florida, United States
Investigator Site 102
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Jacksonville, Florida, United States
Investigator Site 130
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Miami, Florida, United States
Investigator Site 117
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Orlando, Florida, United States
Investigator Site 110
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Atlanta, Georgia, United States
Investigator Site 127
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Chicago, Illinois, United States
Investigator Site 109
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Glenview, Illinois, United States
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Investigator Site 123
🇺🇸Long Beach, California, United States