Exocrine Pancreatic InsufficiencyPhase 3NCT03051490
RESULT: Reliable, Emergent Solution Using Liprotamase Treatment
A Phase 3 study testing Liprotamase and porcine PERT in people with Exocrine Pancreatic Insufficiency and Cystic Fibrosis, run by Anthera Pharmaceuticals at 50 sites in 7 countries.
Trial 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). 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 PERT
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 140 participants
- Where
- 50 sitesin 7 countries
- Study dates
- 28 Apr 2017to Jun 2018
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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
- Good disease control with porcine PERT prior to enrollment
- 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
50 sites in 7 countries.
50 sites in 7 countries
United States28 sites
Investigator Site 117
Altamonte Springs, Florida, 32701
Investigator Site 124
Ann Arbor, Michigan, 48109
Investigator Site 110
Atlanta, Georgia, 30342
Investigator Site #103
Cleveland, Ohio, 44106
Investigator Site 111
Dallas, Texas, 75235
Investigator Site 150
Denver, Colorado, 80206
Investigator Site 118
Durham, North Carolina, 27710
Investigator Site 109
Glenview, Illinois, 60025
Investigator Site 106
Hershey, Pennsylvania, 17033
Investigator Site 116
Houston, Texas, 77030
Investigator Site 148
Iowa City, Iowa, 52242
Investigator Site 134
Jackson, Mississippi, 39216
Investigator Site 102
Jacksonville, Florida, 32207
Investigator Site 140
Kalamazoo, Michigan, 49008
Investigator Site 135
Las Vegas, Nevada, 89107
Investigator Site 139
Little Rock, Arkansas, 72202
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 101
Oklahoma City, Oklahoma, 73104
Investigator Site 136
Oklahoma City, Oklahoma, 73112
Investigator Site 143
Orange, California, 92868
Investigator Site 132
Portland, Maine, 04102
Investigator Site 112
Richmond, Virginia, 23298
Site Investigator #113
Toledo, Ohio, 43606
Investigator Site 151
West Palm Beach, Florida, 33407
Investigator Site 147
Wilmington, Delaware, 19803
Poland8 sites
Investigator Site 203
Karpacz, 58-540
Site 206
Lodz, 90-239
Investigator Site 210
Łomianki, 05-092
Investigator Site 201
Lublin, 20-093
Investigator Site 205
Lublin, 20-362
Investigator Site 202
Rabka-Zdrój, 34-700
Investigator Site 209
Rzeszów, 35-612
Investigator 204
Sopot, 81-713
Spain5 sites
Investigator Site 405
El Palmar, Murcia, 30120
Investigator Site 401
Madrid, 28046
Investigator Site 403
Madrid, 28046
Investigator Site 402
Málaga, 29009
Investigator Site 404
Valencia, 46026
Hungary4 sites
Investigator Site 306
Budapest, 1089
Investigator Site 307
Budapest, 1121
Investigator Site 304
Mosdós, Somogy County, 7257
Investigator Site 302
Törökbálint, Pest County, 2045
Lithuania2 sites
Investigator Site 901
Kaunas, LT-50161
Investigator Site #902
Vilnius, LT-10207
United Kingdom2 sites
Investigator Site 702
Exeter, EX2 5DW
Investigatior Site 701
Southampton, Hampshire, SO 16 6YD
About this study
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
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).
- Conditions
- Exocrine Pancreatic Insufficiency, Cystic Fibrosis
- Being tested
- Liprotamase, porcine 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 PERTDrug
oral, enterically-coated, pig-derived, pancreatic enzyme replacement
| Study group | Role | What they receive |
|---|---|---|
| Liprotamase | Experimental | Individually-optimized dose to be administered orally Receives: Drug: Liprotamase |
| porcine PERT | Active comparator | Individually-optimized dose to be administered orally Receives: Drug: porcine PERT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Coefficient of Fat Absorption (CFA)
Non-inferiority of Liprotamase to approved porcine PERT
Measured: 8 weeks
Secondary outcomes (2)
Coefficient of Nitrogen Absorption (CNA)
Non-inferiority of Liprotamase to approved porcine PERT
Measured: 8 weeks
Safety, as measured by number of participants with adverse events or laboratory abnormalities
Change from baseline
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2017
- Study start28 Apr 2017
- Primary completion28 Feb 2018
- Study completionJun 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- 140 participants (estimated)
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
- NCT03051490
- Sponsor's protocol ID
- AN-EPI3333
- Status last verified
- Jan 2018
- First submitted
- 9 Feb 2017
- Last updated
- 8 Mar 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03051490
- Acronym / sponsor ID
- AN-EPI3333
- Conditions
- Exocrine Pancreatic Insufficiency, Cystic Fibrosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 140 participants (estimated)
- Lead sponsor
- Anthera PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 28 Apr 2017 (actual)
- Primary completion
- 28 Feb 2018 (actual)
- Study completion
- Jun 2018 (estimated)
- First posted
- 13 Feb 2017 (actual)
- Results
- Not posted in the source record
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 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 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 PERT | Active comparator | Drug: porcine 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
- Good disease control with porcine PERT prior to enrollment
- 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 PERToral, enterically-coated, pig-derived, pancreatic enzyme replacement | Drug | porcine PERT |
Primary outcome
Coefficient of Fat Absorption (CFA)
Time frame: 8 weeks
Non-inferiority of Liprotamase to approved porcine PERT
2 secondary outcomes
Coefficient of Nitrogen Absorption (CNA)
Time frame: 8 weeks
Safety, as measured by number of participants with adverse events or laboratory abnormalities
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Mar 2018.
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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
50 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 PERT in people with Exocrine Pancreatic Insufficiency and Cystic Fibrosis, run by Anthera Pharmaceuticals at 50 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: status not confirmed, as of the sponsor's last update on 8 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 50 sites in United States, Poland, Spain, Hungary, Lithuania 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?
Results are not available in the source record.
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?
Coefficient of Fat Absorption (CFA) (time frame: 8 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 8 Mar 2018.