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RESULT: Reliable, Emergent Solution Using Liprotamase Treatment

Primary Purpose

Exocrine Pancreatic Insufficiency, Cystic Fibrosis

Status
Unknown status
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Liprotamase
porcine PERT
Sponsored by
Anthera Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Exocrine Pancreatic Insufficiency

Eligibility Criteria

7 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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%

Sites / Locations

  • Investigator Site 139
  • Investigator Site #123
  • Investigator Site 107
  • Investigator Site 143
  • Investigator Site 150
  • Investigator Site 147
  • Investigator Site 117
  • Investigator Site 102
  • Investigator Site 130
  • Investigator Site 151
  • Investigator Site 110
  • Investigator Site 109
  • Investigator Site 148
  • Investigator Site #122
  • Investigator Site 132
  • Investigator Site 124
  • Investigator Site 140
  • Investigator Site 134
  • Investigator Site 135
  • Investigator Site 118
  • Investigator Site #103
  • Site Investigator #113
  • Investigator Site 101
  • Investigator Site 136
  • Investigator Site 106
  • Investigator Site 111
  • Investigator Site 116
  • Investigator Site 112
  • Investigator Site 302
  • Investigator Site 304
  • Investigator Site 306
  • Investigator Site 307
  • Investigator Site 601
  • Investigator Site 901
  • Investigator Site #902
  • Investigator Site 203
  • Investigator Site 210
  • Investigator Site 201
  • Investigator Site 205
  • Investigator Site 202
  • Investigator Site 209
  • Investigator 204
  • Site 206
  • Investigator Site 405
  • Investigator Site 401
  • Investigator Site 403
  • Investigator Site 402
  • Investigator Site 404
  • Investigatior Site 701
  • Investigator Site 702

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

Liprotamase

porcine PERT

Arm Description

Individually-optimized dose to be administered orally

Individually-optimized dose to be administered orally

Outcomes

Primary Outcome Measures

Coefficient of Fat Absorption (CFA)
Non-inferiority of Liprotamase to approved porcine PERT

Secondary Outcome Measures

Coefficient of Nitrogen Absorption (CNA)
Non-inferiority of Liprotamase to approved porcine PERT
Safety, as measured by number of participants with adverse events or laboratory abnormalities
Change from baseline

Full Information

First Posted
February 9, 2017
Last Updated
March 6, 2018
Sponsor
Anthera Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT03051490
Brief Title
RESULT: Reliable, Emergent Solution Using Liprotamase Treatment
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
January 2018
Overall Recruitment Status
Unknown status
Study Start Date
April 28, 2017 (Actual)
Primary Completion Date
February 28, 2018 (Actual)
Study Completion Date
June 2018 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Anthera Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Exocrine Pancreatic Insufficiency, Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Masking Description
unblinded to treatment randomization; blinded to primary efficacy variable
Allocation
Randomized
Enrollment
140 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Liprotamase
Arm Type
Experimental
Arm Description
Individually-optimized dose to be administered orally
Arm Title
porcine PERT
Arm Type
Active Comparator
Arm Description
Individually-optimized dose to be administered orally
Intervention Type
Drug
Intervention Name(s)
Liprotamase
Intervention Description
oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
Intervention Type
Drug
Intervention Name(s)
porcine PERT
Intervention Description
oral, enterically-coated, pig-derived, pancreatic enzyme replacement
Primary Outcome Measure Information:
Title
Coefficient of Fat Absorption (CFA)
Description
Non-inferiority of Liprotamase to approved porcine PERT
Time Frame
8 weeks
Secondary Outcome Measure Information:
Title
Coefficient of Nitrogen Absorption (CNA)
Description
Non-inferiority of Liprotamase to approved porcine PERT
Time Frame
8 weeks
Title
Safety, as measured by number of participants with adverse events or laboratory abnormalities
Description
Change from baseline
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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%
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Monica Gangal
Organizational Affiliation
Anthera Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Investigator Site 139
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
Investigator Site #123
City
Long Beach
State/Province
California
ZIP/Postal Code
90806
Country
United States
Facility Name
Investigator Site 107
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Investigator Site 143
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Investigator Site 150
City
Denver
State/Province
Colorado
ZIP/Postal Code
80206
Country
United States
Facility Name
Investigator Site 147
City
Wilmington
State/Province
Delaware
ZIP/Postal Code
19803
Country
United States
Facility Name
Investigator Site 117
City
Altamonte Springs
State/Province
Florida
ZIP/Postal Code
32701
Country
United States
Facility Name
Investigator Site 102
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32207
Country
United States
Facility Name
Investigator Site 130
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Investigator Site 151
City
West Palm Beach
State/Province
Florida
ZIP/Postal Code
33407
Country
United States
Facility Name
Investigator Site 110
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Facility Name
Investigator Site 109
City
Glenview
State/Province
Illinois
ZIP/Postal Code
60025
Country
United States
Facility Name
Investigator Site 148
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
Investigator Site #122
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Facility Name
Investigator Site 132
City
Portland
State/Province
Maine
ZIP/Postal Code
04102
Country
United States
Facility Name
Investigator Site 124
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Investigator Site 140
City
Kalamazoo
State/Province
Michigan
ZIP/Postal Code
49008
Country
United States
Facility Name
Investigator Site 134
City
Jackson
State/Province
Mississippi
ZIP/Postal Code
39216
Country
United States
Facility Name
Investigator Site 135
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89107
Country
United States
Facility Name
Investigator Site 118
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Investigator Site #103
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
Site Investigator #113
City
Toledo
State/Province
Ohio
ZIP/Postal Code
43606
Country
United States
Facility Name
Investigator Site 101
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Facility Name
Investigator Site 136
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73112
Country
United States
Facility Name
Investigator Site 106
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Investigator Site 111
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Facility Name
Investigator Site 116
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Investigator Site 112
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Facility Name
Investigator Site 302
City
Torokbalint
State/Province
Pest County
ZIP/Postal Code
2045
Country
Hungary
Facility Name
Investigator Site 304
City
Mosdós
State/Province
Somogy County
ZIP/Postal Code
7257
Country
Hungary
Facility Name
Investigator Site 306
City
Budapest
ZIP/Postal Code
1089
Country
Hungary
Facility Name
Investigator Site 307
City
Budapest
ZIP/Postal Code
1121
Country
Hungary
Facility Name
Investigator Site 601
City
Jerusalem
ZIP/Postal Code
9124001
Country
Israel
Facility Name
Investigator Site 901
City
Kaunas
ZIP/Postal Code
LT-50161
Country
Lithuania
Facility Name
Investigator Site #902
City
Vilnius
ZIP/Postal Code
LT-10207
Country
Lithuania
Facility Name
Investigator Site 203
City
Karpacz
ZIP/Postal Code
58-540
Country
Poland
Facility Name
Investigator Site 210
City
Lomianki
ZIP/Postal Code
05-092
Country
Poland
Facility Name
Investigator Site 201
City
Lublin
ZIP/Postal Code
20-093
Country
Poland
Facility Name
Investigator Site 205
City
Lublin
ZIP/Postal Code
20-362
Country
Poland
Facility Name
Investigator Site 202
City
Rabka-Zdrój
ZIP/Postal Code
34-700
Country
Poland
Facility Name
Investigator Site 209
City
Rzeszów
ZIP/Postal Code
35-612
Country
Poland
Facility Name
Investigator 204
City
Sopot
ZIP/Postal Code
81-713
Country
Poland
Facility Name
Site 206
City
Łódź
ZIP/Postal Code
90-239
Country
Poland
Facility Name
Investigator Site 405
City
El Palmar
State/Province
Murcia
ZIP/Postal Code
30120
Country
Spain
Facility Name
Investigator Site 401
City
Madrid
ZIP/Postal Code
28046
Country
Spain
Facility Name
Investigator Site 403
City
Madrid
ZIP/Postal Code
28046
Country
Spain
Facility Name
Investigator Site 402
City
Málaga
ZIP/Postal Code
29009
Country
Spain
Facility Name
Investigator Site 404
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Facility Name
Investigatior Site 701
City
Southampton
State/Province
Hampshire
ZIP/Postal Code
SO 16 6YD
Country
United Kingdom
Facility Name
Investigator Site 702
City
Exeter
ZIP/Postal Code
EX2 5DW
Country
United Kingdom

12. IPD Sharing Statement

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RESULT: Reliable, Emergent Solution Using Liprotamase Treatment

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