RESULT: Reliable, Emergent Solution Using Liprotamase Treatment

Phase 3

• Conditions: Exocrine Pancreatic Insufficiency; Cystic Fibrosis
• Interventions: Drug: Liprotamase; 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

Study Timeline

• Study First Submitted: 2017-02-09
• Study First Submitted QC: 2017-02-09
• Study First Posted: 2017-02-13
• Study Start: 2017-04-28
• Status Verified: 2018-01
• Primary Completion: 2018-02-28
• Last Update Submitted: 2018-03-06
• Last Update Posted: 2018-03-08
• Study Completion: 2018-06

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 140

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

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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%

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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 #103; 🇺🇸 Cleveland, Ohio, United States

Investigator Site 304; 🇭🇺 Mosdós, Somogy County, Hungary

Investigator Site 307; 🇭🇺 Budapest, Hungary

Investigator Site 901; 🇱🇹 Kaunas, Lithuania

Investigator Site #902; 🇱🇹 Vilnius, Lithuania

Investigator Site 203; 🇵🇱 Karpacz, Poland

Investigator Site 130; 🇺🇸 Miami, Florida, United States

Investigator Site 118; 🇺🇸 Durham, North Carolina, United States

Investigator Site 136; 🇺🇸 Oklahoma City, Oklahoma, United States

Investigator Site 124; 🇺🇸 Ann Arbor, Michigan, United States

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 147; 🇺🇸 Wilmington, Delaware, United States

Investigator Site 150; 🇺🇸 Denver, Colorado, United States

Investigator Site 143; 🇺🇸 Orange, California, United States

Investigator Site 117; 🇺🇸 Altamonte Springs, Florida, United States

Investigator Site 102; 🇺🇸 Jacksonville, Florida, United States

Investigator Site 151; 🇺🇸 West Palm Beach, Florida, United States

Investigator Site 109; 🇺🇸 Glenview, Illinois, United States

Investigator Site 110; 🇺🇸 Atlanta, Georgia, United States

Investigator Site 132; 🇺🇸 Portland, Maine, United States

Investigator Site #122; 🇺🇸 Louisville, Kentucky, United States

Investigator Site 148; 🇺🇸 Iowa City, Iowa, United States

Investigator Site 140; 🇺🇸 Kalamazoo, Michigan, United States

Investigator Site 134; 🇺🇸 Jackson, Mississippi, United States

Site Investigator #113; 🇺🇸 Toledo, Ohio, United States

Investigator Site 101; 🇺🇸 Oklahoma City, Oklahoma, United States

Investigator Site 111; 🇺🇸 Dallas, Texas, United States

Investigator Site 106; 🇺🇸 Hershey, Pennsylvania, United States

Investigator Site 116; 🇺🇸 Houston, Texas, United States

Investigator Site 112; 🇺🇸 Richmond, Virginia, United States

Investigator Site 302; 🇭🇺 Torokbalint, Pest County, Hungary

Investigator Site 306; 🇭🇺 Budapest, Hungary

Investigator Site 601; 🇮🇱 Jerusalem, Israel

Investigator Site 210; 🇵🇱 Lomianki, Poland

Investigator Site 201; 🇵🇱 Lublin, Poland

Investigator Site 205; 🇵🇱 Lublin, Poland

Investigator Site 202; 🇵🇱 Rabka-Zdrój, Poland

Investigator Site 209; 🇵🇱 Rzeszów, Poland

Site 206; 🇵🇱 Łódź, Poland

Investigator 204; 🇵🇱 Sopot, Poland

Investigator Site 401; 🇪🇸 Madrid, Spain

Investigator Site 402; 🇪🇸 Málaga, Spain

Investigator Site 405; 🇪🇸 El Palmar, Murcia, Spain

Investigator Site 403; 🇪🇸 Madrid, Spain

Investigator Site 404; 🇪🇸 Valencia, Spain

Investigatior Site 701; 🇬🇧 Southampton, Hampshire, United Kingdom

Investigator Site 702; 🇬🇧 Exeter, United Kingdom

Investigator Site 135; 🇺🇸 Las Vegas, Nevada, United States