SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis
- Conditions
- Exocrine Pancreatic InsufficiencyCystic Fibrosis
- Interventions
- Drug: LiprotamaseDrug: porcine (pig) PERT
- Registration Number
- NCT02279498
- Lead Sponsor
- Anthera Pharmaceuticals
- 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
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 128
- 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
- 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%
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Liprotamase Liprotamase Individually-optimized dose to be administered orally porcine (pig) PERT porcine (pig) PERT 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
Trial Locations
- Locations (54)
Investigator Site 132
🇺🇸Portland, Maine, United States
Investigator Site 115
🇺🇸Pittsburgh, Pennsylvania, United States
Investigator Site 120
🇺🇸Gainesville, Florida, United States
Investigator Site 102
🇺🇸Jacksonville, Florida, United States
Investigator Site 134
🇺🇸Jackson, Mississippi, United States
Investigator Site 133
🇨🇦Edmonton, Alberta, Canada
Investigator Site 121
🇺🇸Burlington, Vermont, United States
Investigator Site 110
🇺🇸Atlanta, Georgia, United States
Investigator Site 123
🇺🇸Long Beach, California, United States
Investigator Site 209
🇵🇱Rzeszow, Poland
Investigator Site 107
🇺🇸Los Angeles, California, United States
Investigator Site 127
🇺🇸Chicago, Illinois, United States
Investigator Site 401
🇪🇸Madrid, Spain
Investigator Site 103
🇺🇸Cleveland, Ohio, United States
Investigator Site 501
🇨🇿Brno, Czechia
Investigator Site 106
🇺🇸Hershey, Pennsylvania, United States
Investigator Site 113
🇺🇸Toledo, Ohio, United States
Investigator Site 502
🇨🇿Plzen, Czechia
Investigator Site 303
🇭🇺Debrecen, Hajdu-Bihar, Hungary
Investigator Site 206
🇵🇱Lodz, Poland
Investigator Site 104
🇺🇸Indianapolis, Indiana, United States
Investigator Site 130
🇺🇸Miami, Florida, United States
Investigator Site 135
🇺🇸Las Vegas, Nevada, United States
Investigator Site 116
🇺🇸Houston, Texas, United States
Investigator Site 111
🇺🇸Dallas, Texas, United States
Investigator Site 304
🇭🇺Mosdos, Somogy County, Hungary
Investigator Site 105
🇺🇸Wichita, Kansas, United States
Investigator Site 109
🇺🇸Glenview, Illinois, United States
Investigator Site 128
🇺🇸Lexington, Kentucky, United States
Investigator Site 126
🇺🇸East Lansing, Michigan, United States
Investigator Site 125
🇺🇸Fort Worth, Texas, United States
Investigator Site 129
🇺🇸Morgantown, West Virginia, United States
Investigator Site 301
🇭🇺Ajka, Veszprem County, Hungary
Investigator Site 305
🇭🇺Szeged, Csongrad County, Hungary
Investigator Site 302
🇭🇺Torokbalint, Pest County, Hungary
Investigator Site 601
🇮🇱Jerusalem, Israel
Investigator Site 203
🇵🇱Karpacz, Poland
Investigator Site 208
🇵🇱Bialystok, Poland
Investigator Site 201
🇵🇱Lublin, Poland
Investigator Site 205
🇵🇱Lublin, Poland
Investigator Site 202
🇵🇱Rabka Zdroj, Poland
Investigator Site 204
🇵🇱Sopot, Poland
Investigator Site 207
🇵🇱Warszawa, Poland
Investigator Site 403
🇪🇸Madrid, Spain
Investigator Site 402
🇪🇸Malaga, Spain
Investigator Site 404
🇪🇸Valencia, Spain
Investigator Site 122
🇺🇸Louisville, Kentucky, United States
Investigator Site 124
🇺🇸Ann Arbor, Michigan, United States
Investigator Site 101
🇺🇸Oklahoma City, Oklahoma, United States
Investigator Site 114
🇺🇸Aurora, Colorado, United States
Investigator Site 117
🇺🇸Orlando, Florida, United States
Investigator Site 136
🇺🇸Oklahoma City, Oklahoma, United States
Investigator Site 119
🇺🇸Portland, Oregon, United States
Investigator Site 112
🇺🇸Richmond, Virginia, United States