RESULT: Reliable, Emergent Solution Using Liprotamase Treatment
- Conditions
- Exocrine Pancreatic InsufficiencyCystic Fibrosis
- Interventions
- Drug: porcine PERT
- Registration Number
- NCT03051490
- Lead Sponsor
- Anthera Pharmaceuticals
- Brief Summary
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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 140
- 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
- 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 PERT porcine PERT Individually-optimized dose to be administered orally
- Primary Outcome Measures
Name Time Method Coefficient of Fat Absorption (CFA) 8 weeks Non-inferiority of Liprotamase to approved porcine PERT
- Secondary Outcome Measures
Name Time Method Coefficient of Nitrogen Absorption (CNA) 8 weeks Non-inferiority of Liprotamase to approved porcine PERT
Safety, as measured by number of participants with adverse events or laboratory abnormalities 6 months Change from baseline
Trial Locations
- Locations (50)
Investigator Site 139
🇺🇸Little Rock, Arkansas, United States
Investigator Site #123
🇺🇸Long Beach, California, United States
Investigator Site 107
🇺🇸Los Angeles, California, United States
Investigator Site 143
🇺🇸Orange, California, United States
Investigator Site 150
🇺🇸Denver, Colorado, United States
Investigator Site 147
🇺🇸Wilmington, Delaware, United States
Investigator Site 117
🇺🇸Altamonte Springs, Florida, United States
Investigator Site 102
🇺🇸Jacksonville, Florida, United States
Investigator Site 130
🇺🇸Miami, Florida, United States
Investigator Site 151
🇺🇸West Palm Beach, Florida, United States
Scroll for more (40 remaining)Investigator Site 139🇺🇸Little Rock, Arkansas, United States