RESULT: Reliable, Emergent Solution Using Liprotamase Treatment

March 6, 2018 updated by: Anthera Pharmaceuticals

A Phase 3, Randomized, Open-Label, Assessor-Blind, Non-Inferiority, Active-Comparator Study Evaluating the Efficacy and Safety of Liprotamase in Subjects With Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency

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).

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

140

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Hungary
      • Budapest, Hungary, 1089
        • Investigator Site 306
      • Budapest, Hungary, 1121
        • Investigator Site 307
    • Pest County
      • Torokbalint, Pest County, Hungary, 2045
        • Investigator Site 302
    • Somogy County
      • Mosdós, Somogy County, Hungary, 7257
        • Investigator Site 304
  • Israel
      • Jerusalem, Israel, 9124001
        • Investigator Site 601
  • Lithuania
      • Kaunas, Lithuania, LT-50161
        • Investigator Site 901
      • Vilnius, Lithuania, LT-10207
        • Investigator Site #902
  • Poland
      • Karpacz, Poland, 58-540
        • Investigator site 203
      • Lomianki, Poland, 05-092
        • Investigator site 210
      • Lublin, Poland, 20-093
        • Investigator site 201
      • Lublin, Poland, 20-362
        • Investigator site 205
      • Rabka-Zdrój, Poland, 34-700
        • Investigator site 202
      • Rzeszów, Poland, 35-612
        • Investigator site 209
      • Sopot, Poland, 81-713
        • Investigator 204
      • Łódź, Poland, 90-239
        • Site 206
  • Spain
      • Madrid, Spain, 28046
        • Investigator Site 401
      • Madrid, Spain, 28046
        • Investigator Site 403
      • Málaga, Spain, 29009
        • Investigator Site 402
      • Valencia, Spain, 46026
        • Investigator Site 404
    • Murcia
      • El Palmar, Murcia, Spain, 30120
        • Investigator Site 405
  • United Kingdom
      • Exeter, United Kingdom, EX2 5DW
        • Investigator Site 702
    • Hampshire
      • Southampton, Hampshire, United Kingdom, SO 16 6YD
        • Investigatior Site 701
  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Investigator Site 139
    • California
      • Long Beach, California, United States, 90806
        • Investigator Site #123
      • Los Angeles, California, United States, 90033
        • Investigator site 107
      • Orange, California, United States, 92868
        • Investigator Site 143
    • Colorado
      • Denver, Colorado, United States, 80206
        • Investigator Site 150
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Investigator Site 147
    • Florida
      • Altamonte Springs, Florida, United States, 32701
        • Investigator Site 117
      • Jacksonville, Florida, United States, 32207
        • Investigator Site 102
      • Miami, Florida, United States, 33136
        • Investigator Site 130
      • West Palm Beach, Florida, United States, 33407
        • Investigator Site 151
    • Georgia
      • Atlanta, Georgia, United States, 30342
        • Investigator site 110
    • Illinois
      • Glenview, Illinois, United States, 60025
        • Investigator site 109
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Investigator Site 148
    • Kentucky
      • Louisville, Kentucky, United States, 40202
        • Investigator Site #122
    • Maine
      • Portland, Maine, United States, 04102
        • Investigator Site 132
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Investigator Site 124
      • Kalamazoo, Michigan, United States, 49008
        • Investigator Site 140
    • Mississippi
      • Jackson, Mississippi, United States, 39216
        • Investigator Site 134
    • Nevada
      • Las Vegas, Nevada, United States, 89107
        • Investigator Site 135
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Investigator Site 118
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • Investigator Site #103
      • Toledo, Ohio, United States, 43606
        • Site Investigator #113
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Investigator Site 101
      • Oklahoma City, Oklahoma, United States, 73112
        • Investigator Site 136
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Investigator site 106
    • Texas
      • Dallas, Texas, United States, 75235
        • Investigator site 111
      • Houston, Texas, United States, 77030
        • Investigator Site 116
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Investigator site 112

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

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%

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Liprotamase
Individually-optimized dose to be administered orally
Drug: Liprotamase
oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
Active Comparator: porcine PERT
Individually-optimized dose to be administered orally
Drug: porcine PERT
oral, enterically-coated, pig-derived, pancreatic enzyme replacement

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Coefficient of Fat Absorption (CFA)
Time Frame: 8 weeks
Non-inferiority of Liprotamase to approved porcine PERT
8 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Coefficient of Nitrogen Absorption (CNA)
Time Frame: 8 weeks
Non-inferiority of Liprotamase to approved porcine PERT
8 weeks
Safety, as measured by number of participants with adverse events or laboratory abnormalities
Time Frame: 6 months
Change from baseline
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Anthera Pharmaceuticals

Investigators

  • Study Director: Monica Gangal, Anthera Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 28, 2017

Primary Completion (Actual)

February 28, 2018

Study Completion (Anticipated)

June 1, 2018

Study Registration Dates

First Submitted

February 9, 2017

First Submitted That Met QC Criteria

February 9, 2017

First Posted (Actual)

February 13, 2017

Study Record Updates

Last Update Posted (Actual)

March 8, 2018

Last Update Submitted That Met QC Criteria

March 6, 2018

Last Verified

January 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AN-EPI3333

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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