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

Status

Indicates the current recruitment status or the expanded access status.
Unknown

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Anticipated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
140

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

3 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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Anthera Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 28, 2017

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 28, 2018

Study Completion (Anticipated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 1, 2018

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 9, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 9, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 13, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 8, 2018

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 6, 2018

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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