RESULT: Reliable, Emergent Solution Using Liprotamase Treatment

NCT03051490 | Unknown Phase 3 DMC FDA Drug

• 1 other identifier: AN-EPI3333
• Study Type: interventional
• Target: 140
• Locations: 7 countries
• Sites: 50

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).

Conditions

Exocrine Pancreatic Insufficiency; Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Coefficient of Fat Absorption (CFA)

  Non-inferiority of Liprotamase to approved porcine PERT

  8 weeks

Secondary Outcomes (2)

• Coefficient of Nitrogen Absorption (CNA)

  8 weeks

• Safety, as measured by number of participants with adverse events or laboratory abnormalities

  6 months

Study Arms (2)

Liprotamase

EXPERIMENTAL

Individually-optimized dose to be administered orally

Drug: Liprotamase

porcine PERT

ACTIVE COMPARATOR

Individually-optimized dose to be administered orally

Drug: porcine PERT

Interventions

Liprotamase DRUG

oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement

Liprotamase

porcine PERT DRUG

oral, enterically-coated, pig-derived, pancreatic enzyme replacement

porcine PERT

Eligibility Criteria

• Age: 7 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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%

Sponsors & Collaborators

• Anthera Pharmaceuticals: lead

Study Sites (50)

Investigator Site 139

Little Rock, Arkansas, 72202, United States

Location

Investigator Site #123

Long Beach, California, 90806, United States

Location

Investigator Site 107

Los Angeles, California, 90033, United States

Location

Investigator Site 143

Orange, California, 92868, United States

Location

Investigator Site 150

Denver, Colorado, 80206, United States

Location

Investigator Site 147

Wilmington, Delaware, 19803, United States

Location

Investigator Site 117

Altamonte Springs, Florida, 32701, United States

Location

Investigator Site 102

Jacksonville, Florida, 32207, United States

Location

Investigator Site 130

Miami, Florida, 33136, United States

Location

Investigator Site 151

West Palm Beach, Florida, 33407, United States

Location

Investigator Site 110

Atlanta, Georgia, 30342, United States

Location

Investigator Site 109

Glenview, Illinois, 60025, United States

Location

Investigator Site 148

Iowa City, Iowa, 52242, United States

Location

Investigator Site #122

Louisville, Kentucky, 40202, United States

Location

Investigator Site 132

Portland, Maine, 04102, United States

Location

Investigator Site 124

Ann Arbor, Michigan, 48109, United States

Location

Investigator Site 140

Kalamazoo, Michigan, 49008, United States

Location

Investigator Site 134

Jackson, Mississippi, 39216, United States

Location

Investigator Site 135

Las Vegas, Nevada, 89107, United States

Location

Investigator Site 118

Durham, North Carolina, 27710, United States

Location

Investigator Site #103

Cleveland, Ohio, 44106, United States

Location

Site Investigator #113

Toledo, Ohio, 43606, United States

Location

Investigator Site 101

Oklahoma City, Oklahoma, 73104, United States

Location

Investigator Site 136

Oklahoma City, Oklahoma, 73112, United States

Location

Investigator Site 106

Hershey, Pennsylvania, 17033, United States

Location

Investigator Site 111

Dallas, Texas, 75235, United States

Location

Investigator Site 116

Houston, Texas, 77030, United States

Location

Investigator Site 112

Richmond, Virginia, 23298, United States

Location

Investigator Site 302

Törökbálint, Pest County, 2045, Hungary

Location

Investigator Site 304

Mosdós, Somogy County, 7257, Hungary

Location

Investigator Site 306

Budapest, 1089, Hungary

Location

Investigator Site 307

Budapest, 1121, Hungary

Location

Investigator Site 601

Jerusalem, 9124001, Israel

Location

Investigator Site 901

Kaunas, LT-50161, Lithuania

Location

Investigator Site #902

Vilnius, LT-10207, Lithuania

Location

Investigator Site 203

Karpacz, 58-540, Poland

Location

Site 206

Lodz, 90-239, Poland

Location

Investigator Site 201

Lublin, 20-093, Poland

Location

Investigator Site 205

Lublin, 20-362, Poland

Location

Investigator Site 202

Rabka-Zdrój, 34-700, Poland

Location

Investigator Site 209

Rzeszów, 35-612, Poland

Location

Investigator 204

Sopot, 81-713, Poland

Location

Investigator Site 210

Łomianki, 05-092, Poland

Location

Investigator Site 405

El Palmar, Murcia, 30120, Spain

Location

Investigator Site 401

Madrid, 28046, Spain

Location

Investigator Site 403

Madrid, 28046, Spain

Location

Investigator Site 402

Málaga, 29009, Spain

Location

Investigator Site 404

Valencia, 46026, Spain

Location

Investigatior Site 701

Southampton, Hampshire, SO 16 6YD, United Kingdom

Location

Investigator Site 702

Exeter, EX2 5DW, United Kingdom

Location

MeSH Terms

Conditions

Exocrine Pancreatic Insufficiency; Cystic Fibrosis

Interventions

liprotamase lipase

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Monica Gangal

  Anthera Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Masking Details: unblinded to treatment randomization; blinded to primary efficacy variable
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 9, 2017
• First Posted: February 13, 2017
• Study Start: April 28, 2017
• Primary Completion: February 28, 2018
• Study Completion: June 1, 2018
• Last Updated: March 8, 2018

Record last verified: 2018-01

Locations

HU (4); IL (1); US (28); ES (5); PL (8); LI (2); GB (2)