Clinical trial NCT03051490: 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
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Exocrine Pancreatic Insufficiency
Cystic Fibrosis
Lead sponsor: Anthera Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2017-02-13
Enrollment: 140
Sex: ALL
Minimum age (years): 7
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


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%

Primary outcomes:
measure: Coefficient of Fat Absorption (CFA)
timeFrame: 8 weeks
Secondary outcomes:
measure: Coefficient of Nitrogen Absorption (CNA)
timeFrame: 8 weeks
measure: Safety, as measured by number of participants with adverse events or laboratory abnormalities
timeFrame: 6 months
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Investigator Site 139
state: Arkansas
city: Long Beach
country: United States
facility: Investigator Site #123
state: California
city: Los Angeles
country: United States
facility: Investigator Site 107
state: California
city: Orange
country: United States
facility: Investigator Site 143
state: California
city: Denver
country: United States
facility: Investigator Site 150
state: Colorado
city: Wilmington
country: United States
facility: Investigator Site 147
state: Delaware
city: Altamonte Springs
country: United States
facility: Investigator Site 117
state: Florida
city: Jacksonville
country: United States
facility: Investigator Site 102
state: Florida
city: Miami
country: United States
facility: Investigator Site 130
state: Florida
city: West Palm Beach
country: United States
facility: Investigator Site 151
state: Florida
city: Atlanta
country: United States
facility: Investigator Site 110
state: Georgia
city: Glenview
country: United States
facility: Investigator Site 109
state: Illinois
city: Iowa City
country: United States
facility: Investigator Site 148
state: Iowa
city: Louisville
country: United States
facility: Investigator Site #122
state: Kentucky
city: Portland
country: United States
facility: Investigator Site 132
state: Maine
city: Ann Arbor
country: United States
facility: Investigator Site 124
state: Michigan
city: Kalamazoo
country: United States
facility: Investigator Site 140
state: Michigan
city: Jackson
country: United States
facility: Investigator Site 134
state: Mississippi
city: Las Vegas
country: United States
facility: Investigator Site 135
state: Nevada
city: Durham
country: United States
facility: Investigator Site 118
state: North Carolina
city: Cleveland
country: United States
facility: Investigator Site #103
state: Ohio
city: Toledo
country: United States
facility: Site Investigator #113
state: Ohio
city: Oklahoma City
country: United States
facility: Investigator Site 101
state: Oklahoma
city: Oklahoma City
country: United States
facility: Investigator Site 136
state: Oklahoma
city: Hershey
country: United States
facility: Investigator Site 106
state: Pennsylvania
city: Dallas
country: United States
facility: Investigator Site 111
state: Texas
city: Houston
country: United States
facility: Investigator Site 116
state: Texas
city: Richmond
country: United States
facility: Investigator Site 112
state: Virginia
city: Torokbalint
country: Hungary
facility: Investigator Site 302
state: Pest County
city: Mosdós
country: Hungary
facility: Investigator Site 304
state: Somogy County
city: Budapest
country: Hungary
facility: Investigator Site 306
city: Budapest
country: Hungary
facility: Investigator Site 307
city: Jerusalem
country: Israel
facility: Investigator Site 601
city: Kaunas
country: Lithuania
facility: Investigator Site 901
city: Vilnius
country: Lithuania
facility: Investigator Site #902
city: Karpacz
country: Poland
facility: Investigator Site 203
city: Lomianki
country: Poland
facility: Investigator Site 210
city: Lublin
country: Poland
facility: Investigator Site 201
city: Lublin
country: Poland
facility: Investigator Site 205
city: Rabka-Zdrój
country: Poland
facility: Investigator Site 202
city: Rzeszów
country: Poland
facility: Investigator Site 209
city: Sopot
country: Poland
facility: Investigator 204
city: Łódź
country: Poland
facility: Site 206
city: El Palmar
country: Spain
facility: Investigator Site 405
state: Murcia
city: Madrid
country: Spain
facility: Investigator Site 401
city: Madrid
country: Spain
facility: Investigator Site 403
city: Málaga
country: Spain
facility: Investigator Site 402
city: Valencia
country: Spain
facility: Investigator Site 404
city: Southampton
country: United Kingdom
facility: Investigatior Site 701
state: Hampshire
city: Exeter
country: United Kingdom
facility: Investigator Site 702
Study officials:
affiliation: Anthera Pharmaceuticals
name: Monica Gangal
role: STUDY_DIRECTOR
References: