Exocrine Pancreatic InsufficiencyPhase 3NCT02279498
SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis
A Phase 3 study testing Liprotamase and porcine (pig) PERT in people with Exocrine Pancreatic Insufficiency and Cystic Fibrosis, run by Anthera Pharmaceuticals at 54 sites in 7 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 7 years and older
- Conditions the study is for
- Exocrine Pancreatic Insufficiency, Cystic Fibrosis
- What is being tested?
- Liprotamase and porcine (pig) PERT
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 128 participants
- Where
- 54 sitesin 7 countries
- Study dates
- Jun 2015to 20 Jan 2017
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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%
Trial locations and contacts
54 sites in 7 countries.
54 sites in 7 countries
United States32 sites
Investigator Site 124
Ann Arbor, Michigan, 48109-5212
Investigator Site 110
Atlanta, Georgia, 30342
Investigator Site 114
Aurora, Colorado, 80045
Investigator Site 121
Burlington, Vermont, 05405
Investigator Site 127
Chicago, Illinois, 60611
Investigator Site 103
Cleveland, Ohio, 44106
Investigator Site 111
Dallas, Texas, 75390
Investigator Site 126
East Lansing, Michigan, 48823
Investigator Site 125
Fort Worth, Texas, 76104
Investigator Site 120
Gainesville, Florida, 32610
Investigator Site 109
Glenview, Illinois, 60025
Investigator Site 106
Hershey, Pennsylvania, 17033
Investigator Site 116
Houston, Texas, 77030
Investigator Site 104
Indianapolis, Indiana, 46202
Investigator Site 134
Jackson, Mississippi, 39216
Investigator Site 102
Jacksonville, Florida, 32207
Investigator Site 135
Las Vegas, Nevada, 89107
Investigator Site 128
Lexington, Kentucky, 40506
Investigator Site 123
Long Beach, California, 90806
Investigator Site 107
Los Angeles, California, 90033
Investigator Site 122
Louisville, Kentucky, 40202
Investigator Site 130
Miami, Florida, 33136
Investigator Site 129
Morgantown, West Virginia, 26506
Investigator Site 101
Oklahoma City, Oklahoma, 73104
Investigator Site 136
Oklahoma City, Oklahoma, 73112
Investigator Site 117
Orlando, Florida, 32803
Investigator Site 115
Pittsburgh, Pennsylvania, 15224
Investigator Site 132
Portland, Maine, 04102
Investigator Site 119
Portland, Oregon, 97239
Investigator Site 112
Richmond, Virginia, 23219
Investigator Site 113
Toledo, Ohio, 43606
Investigator Site 105
Wichita, Kansas, 67214
Poland9 sites
Investigator Site 208
Bialystok, 15-044
Investigator Site 203
Karpacz, 58-540
Investigator Site 206
Lodz, 90-329
Investigator Site 201
Lublin, 20-093
Investigator Site 205
Lublin, 20-362
Investigator Site 202
Rabka-Zdrój, 34-700
Investigator Site 209
Rzeszów, 35-312
Investigator Site 204
Sopot, 81-713
Investigator Site 207
Warsaw, 01-195
Hungary5 sites
Investigator Site 301
Ajka, Veszprém megye, 8400
Investigator Site 303
Debrecen, Hajdú-Bihar, 4031
Investigator Site 304
Mosdós, Somogy County, 7257
Investigator Site 305
Szeged, Csongrád megye, 6720
Investigator Site 302
Törökbálint, Pest County, 2045
Spain4 sites
Investigator Site 403
Madrid, 28006
Investigator Site 401
Madrid, 28046
Investigator Site 402
Málaga, 29009
Investigator Site 404
Valencia, 46026
Czechia2 sites
Investigator Site 501
Brno, 62500
Investigator Site 502
Pilsen, 305 99
About this study
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
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.
Read the 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).
- Conditions
- Exocrine Pancreatic Insufficiency, Cystic Fibrosis
- Being tested
- Liprotamase, porcine (pig) PERT
- Medical subject headings
- Exocrine Pancreatic Insufficiency, Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LiprotamaseDrug
oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
porcine (pig) PERTDrug
oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source
| Study group | Role | What they receive |
|---|---|---|
| Liprotamase | Experimental | Individually-optimized dose to be administered orally Receives: Drug: Liprotamase |
| porcine (pig) PERT | Active comparator | Individually-optimized dose to be administered orally Receives: Drug: porcine (pig) PERT |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline, 7 weeks
Secondary outcomes (2)
Coefficient of Fat Absorption (CFA)
Change from baseline in coefficient of fat absorption
Measured: Baseline, 7 weeks
Coefficient of Nitrogen Absorption (CNA)
Change from baseline in coefficient of nitrogen absorption
Measured: Baseline, 7 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Oct 2014 (estimated)
- Study startJun 2015
- Primary completionOct 2016
- Study completion20 Jan 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Exocrine Pancreatic InsufficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 128 participants (actual)
Sponsor and investigators
- Lead sponsor
- Anthera Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Monica Gangal, Study director, Anthera Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02279498
- Sponsor's protocol ID
- AN-EPI3331
- Status last verified
- Jul 2018
- First submitted
- 28 Oct 2014
- Last updated
- 14 Aug 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
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.
PubMed 32761612
Study at a glance
From the registry record, last updated by the sponsor on 14 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02279498
- Acronym / sponsor ID
- AN-EPI3331
- Conditions
- Exocrine Pancreatic Insufficiency, Cystic Fibrosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 128 participants (actual)
- Lead sponsor
- Anthera PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Jun 2015 (actual)
- Primary completion
- Oct 2016 (actual)
- Study completion
- 20 Jan 2017 (actual)
- First posted
- 31 Oct 2014 (estimated)
- Results
- Posted 14 Aug 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Liprotamase | Experimental | Drug: LiprotamaseIndividually-optimized dose to be administered orally |
| porcine (pig) PERT | Active comparator | Drug: porcine (pig) PERTIndividually-optimized dose to be administered orally |
Who may qualify
- Ages
- 7 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Liprotamaseoral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement | Drug | Liprotamase |
| porcine (pig) PERToral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source | Drug | porcine (pig) PERT |
Primary outcome
Treatment Difference in Coefficient of Fat Absorption (CFA) Change From Baseline
Time frame: Baseline, 7 weeks
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
2 secondary outcomes
Coefficient of Fat Absorption (CFA)
Time frame: Baseline, 7 weeks
Coefficient of Nitrogen Absorption (CNA)
Time frame: Baseline, 7 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Aug 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 32761612Somaraju 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Monica Gangal study director
Anthera Pharmaceuticals
54 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Liprotamase and porcine (pig) PERT in people with Exocrine Pancreatic Insufficiency and Cystic Fibrosis, run by Anthera Pharmaceuticals at 54 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Aug 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 54 sites in United States, Poland, Hungary, Spain, Czechia and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Aug 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment Difference in Coefficient of Fat Absorption (CFA) Change From Baseline (time frame: Baseline, 7 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02279498, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Aug 2018.