search
Back to results

SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis

Primary Purpose

Exocrine Pancreatic Insufficiency, Cystic Fibrosis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Liprotamase
porcine (pig) 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
  • Minimum Coefficient of Fat (CFA) at screening while on stable PERT therapy
  • 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 123
  • Investigator Site 107
  • Investigator Site 114
  • Investigator Site 120
  • Investigator Site 102
  • Investigator Site 130
  • Investigator Site 117
  • Investigator Site 110
  • Investigator Site 127
  • Investigator Site 109
  • Investigator Site 104
  • Investigator Site 105
  • Investigator Site 128
  • Investigator Site 122
  • Investigator Site 132
  • Investigator Site 124
  • Investigator Site 126
  • Investigator Site 134
  • Investigator Site 135
  • Investigator Site 103
  • Investigator Site 113
  • Investigator Site 101
  • Investigator Site 136
  • Investigator Site 119
  • Investigator Site 106
  • Investigator Site 115
  • Investigator Site 111
  • Investigator Site 125
  • Investigator Site 116
  • Investigator Site 121
  • Investigator Site 112
  • Investigator Site 129
  • Investigator Site 133
  • Investigator Site 501
  • Investigator Site 502
  • Investigator Site 305
  • Investigator Site 303
  • Investigator Site 302
  • Investigator Site 304
  • Investigator Site 301
  • Investigator Site 601
  • Investigator Site 208
  • Investigator Site 203
  • Investigator Site 206
  • Investigator Site 201
  • Investigator Site 205
  • Investigator Site 202
  • Investigator Site 209
  • Investigator Site 204
  • Investigator Site 207
  • Investigator Site 403
  • Investigator Site 401
  • Investigator Site 402
  • Investigator Site 404

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

Liprotamase

porcine (pig) PERT

Arm Description

Individually-optimized dose to be administered orally

Individually-optimized dose to be administered orally

Outcomes

Primary Outcome Measures

Treatment Difference in Coefficient of Fat Absorption (CFA) Change From Baseline
The primary endpoint evaluates the difference between treatment arms in change from baseline in coefficient of fat absorption (CFA). As such, descriptive statistics for individual treatment arms are not provided in this measure, but are reported in the secondary endpoints

Secondary Outcome Measures

Coefficient of Fat Absorption (CFA)
Change from baseline in coefficient of fat absorption
Coefficient of Nitrogen Absorption (CNA)
Change from baseline in coefficient of nitrogen absorption

Full Information

First Posted
October 28, 2014
Last Updated
July 17, 2018
Sponsor
Anthera Pharmaceuticals
search

1. Study Identification

Unique Protocol Identification Number
NCT02279498
Brief Title
SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis
Official Title
A Phase 3, Randomized, Open-Label, Assessor-Blind, Noninferiority, 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
July 2018
Overall Recruitment Status
Completed
Study Start Date
June 2015 (Actual)
Primary Completion Date
October 2016 (Actual)
Study Completion Date
January 20, 2017 (Actual)

3. Sponsor/Collaborators

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

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Liprotamase powder is a non-porcine, soluble and stable mixture of three digestive enzymes including lipase, protease, and amylase. The purpose of the present study is to provide additional efficacy and safety data compared to approved, porcine-derived, enterically-coated and encapsulated pancreatic enzyme replacement therapy. The primary efficacy endpoint of the study will be comparative efficacy measured as the change in the coefficient of fat absorption (CFA) in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI). Liprotamase is stable in stomach and digestive fluids allowing administration in a variety of convenient formulations and with a number of foods without enteric coating.
Detailed Description
Porcine derived enzymes are used for pancreatic enzyme replacement therapy in patients with cystic fibrosis (CF). Liprotamase is a biotechnology-derived enzyme replacement without enteric coating. This is an open-label, assessor blind, parallel group, multicenter, international trial to evaluate the noninferiority of liprotamase and pancrelipase in CF patients aged ≥7 years with pancreatic insufficiency. Subjects were randomized to liprotamase or pancrelipase, dose-matched to pre-study lipase doses. The lower bound of the 95% confidence interval (CI) for noninferiority was -15% for treatment difference in change from baseline coefficient of fat absorption (CFA).

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)
Allocation
Randomized
Enrollment
128 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Liprotamase
Arm Type
Experimental
Arm Description
Individually-optimized dose to be administered orally
Arm Title
porcine (pig) 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 (pig) PERT
Intervention Description
oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source
Primary Outcome Measure Information:
Title
Treatment Difference in Coefficient of Fat Absorption (CFA) Change From Baseline
Description
The primary endpoint evaluates the difference between treatment arms in change from baseline in coefficient of fat absorption (CFA). As such, descriptive statistics for individual treatment arms are not provided in this measure, but are reported in the secondary endpoints
Time Frame
Baseline, 7 weeks
Secondary Outcome Measure Information:
Title
Coefficient of Fat Absorption (CFA)
Description
Change from baseline in coefficient of fat absorption
Time Frame
Baseline, 7 weeks
Title
Coefficient of Nitrogen Absorption (CNA)
Description
Change from baseline in coefficient of nitrogen absorption
Time Frame
Baseline, 7 weeks

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 Minimum Coefficient of Fat (CFA) at screening while on stable PERT therapy 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 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 114
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Investigator Site 120
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
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 117
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Facility Name
Investigator Site 110
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Facility Name
Investigator Site 127
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Investigator Site 109
City
Glenview
State/Province
Illinois
ZIP/Postal Code
60025
Country
United States
Facility Name
Investigator Site 104
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Investigator Site 105
City
Wichita
State/Province
Kansas
ZIP/Postal Code
67214
Country
United States
Facility Name
Investigator Site 128
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40506
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
48109-5212
Country
United States
Facility Name
Investigator Site 126
City
East Lansing
State/Province
Michigan
ZIP/Postal Code
48823
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 103
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
Investigator Site 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 119
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Investigator Site 106
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Investigator Site 115
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Investigator Site 111
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Investigator Site 125
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
Investigator Site 116
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Investigator Site 121
City
Burlington
State/Province
Vermont
ZIP/Postal Code
05405
Country
United States
Facility Name
Investigator Site 112
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23219
Country
United States
Facility Name
Investigator Site 129
City
Morgantown
State/Province
West Virginia
ZIP/Postal Code
26506
Country
United States
Facility Name
Investigator Site 133
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G IC9
Country
Canada
Facility Name
Investigator Site 501
City
Brno
ZIP/Postal Code
62500
Country
Czechia
Facility Name
Investigator Site 502
City
Plzen
ZIP/Postal Code
305 99
Country
Czechia
Facility Name
Investigator Site 305
City
Szeged
State/Province
Csongrad County
ZIP/Postal Code
6720
Country
Hungary
Facility Name
Investigator Site 303
City
Debrecen
State/Province
Hajdu-Bihar
ZIP/Postal Code
4031
Country
Hungary
Facility Name
Investigator Site 302
City
Torokbalint
State/Province
Pest County
ZIP/Postal Code
2045
Country
Hungary
Facility Name
Investigator Site 304
City
Mosdos
State/Province
Somogy County
ZIP/Postal Code
7257
Country
Hungary
Facility Name
Investigator Site 301
City
Ajka
State/Province
Veszprem County
ZIP/Postal Code
8400
Country
Hungary
Facility Name
Investigator Site 601
City
Jerusalem
ZIP/Postal Code
9124001
Country
Israel
Facility Name
Investigator Site 208
City
Bialystok
ZIP/Postal Code
15-044
Country
Poland
Facility Name
Investigator Site 203
City
Karpacz
ZIP/Postal Code
58-540
Country
Poland
Facility Name
Investigator Site 206
City
Lodz
ZIP/Postal Code
90-329
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 Zdroj
ZIP/Postal Code
34-700
Country
Poland
Facility Name
Investigator Site 209
City
Rzeszow
ZIP/Postal Code
35-312
Country
Poland
Facility Name
Investigator Site 204
City
Sopot
ZIP/Postal Code
81-713
Country
Poland
Facility Name
Investigator Site 207
City
Warszawa
ZIP/Postal Code
01-195
Country
Poland
Facility Name
Investigator Site 403
City
Madrid
ZIP/Postal Code
28006
Country
Spain
Facility Name
Investigator Site 401
City
Madrid
ZIP/Postal Code
28046
Country
Spain
Facility Name
Investigator Site 402
City
Malaga
ZIP/Postal Code
29009
Country
Spain
Facility Name
Investigator Site 404
City
Valencia
ZIP/Postal Code
46026
Country
Spain

12. IPD Sharing Statement

Citations:
PubMed Identifier
32761612
Citation
Somaraju URR, Solis-Moya A. Pancreatic enzyme replacement therapy for people with cystic fibrosis. Cochrane Database Syst Rev. 2020 Aug 5;8(8):CD008227. doi: 10.1002/14651858.CD008227.pub4.
Results Reference
derived

Learn more about this trial

SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis

We'll reach out to this number within 24 hrs