Clinical trial NCT02279498: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Exocrine Pancreatic Insufficiency
Cystic Fibrosis
Lead sponsor: Anthera Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2014-10-31
Enrollment: 128
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 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).

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%

Primary outcomes:
measure: Treatment Difference in Coefficient of Fat Absorption (CFA) Change From Baseline
timeFrame: Baseline, 7 weeks
Secondary outcomes:
measure: Coefficient of Fat Absorption (CFA)
timeFrame: Baseline, 7 weeks
measure: Coefficient of Nitrogen Absorption (CNA)
timeFrame: Baseline, 7 weeks
Other outcomes:
Locations:
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: Aurora
country: United States
facility: Investigator Site 114
state: Colorado
city: Gainesville
country: United States
facility: Investigator Site 120
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: Orlando
country: United States
facility: Investigator Site 117
state: Florida
city: Atlanta
country: United States
facility: Investigator Site 110
state: Georgia
city: Chicago
country: United States
facility: Investigator Site 127
state: Illinois
city: Glenview
country: United States
facility: Investigator Site 109
state: Illinois
city: Indianapolis
country: United States
facility: Investigator Site 104
state: Indiana
city: Wichita
country: United States
facility: Investigator Site 105
state: Kansas
city: Lexington
country: United States
facility: Investigator Site 128
state: Kentucky
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: East Lansing
country: United States
facility: Investigator Site 126
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: Cleveland
country: United States
facility: Investigator Site 103
state: Ohio
city: Toledo
country: United States
facility: Investigator Site 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: Portland
country: United States
facility: Investigator Site 119
state: Oregon
city: Hershey
country: United States
facility: Investigator Site 106
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Investigator Site 115
state: Pennsylvania
city: Dallas
country: United States
facility: Investigator Site 111
state: Texas
city: Fort Worth
country: United States
facility: Investigator Site 125
state: Texas
city: Houston
country: United States
facility: Investigator Site 116
state: Texas
city: Burlington
country: United States
facility: Investigator Site 121
state: Vermont
city: Richmond
country: United States
facility: Investigator Site 112
state: Virginia
city: Morgantown
country: United States
facility: Investigator Site 129
state: West Virginia
city: Edmonton
country: Canada
facility: Investigator Site 133
state: Alberta
city: Brno
country: Czechia
facility: Investigator Site 501
city: Plzen
country: Czechia
facility: Investigator Site 502
city: Szeged
country: Hungary
facility: Investigator Site 305
state: Csongrad County
city: Debrecen
country: Hungary
facility: Investigator Site 303
state: Hajdu-Bihar
city: Torokbalint
country: Hungary
facility: Investigator Site 302
state: Pest County
city: Mosdos
country: Hungary
facility: Investigator Site 304
state: Somogy County
city: Ajka
country: Hungary
facility: Investigator Site 301
state: Veszprem County
city: Jerusalem
country: Israel
facility: Investigator Site 601
city: Bialystok
country: Poland
facility: Investigator Site 208
city: Karpacz
country: Poland
facility: Investigator Site 203
city: Lodz
country: Poland
facility: Investigator Site 206
city: Lublin
country: Poland
facility: Investigator Site 201
city: Lublin
country: Poland
facility: Investigator Site 205
city: Rabka Zdroj
country: Poland
facility: Investigator Site 202
city: Rzeszow
country: Poland
facility: Investigator Site 209
city: Sopot
country: Poland
facility: Investigator Site 204
city: Warszawa
country: Poland
facility: Investigator Site 207
city: Madrid
country: Spain
facility: Investigator Site 403
city: Madrid
country: Spain
facility: Investigator Site 401
city: Malaga
country: Spain
facility: Investigator Site 402
city: Valencia
country: Spain
facility: Investigator Site 404
Study officials:
affiliation: Anthera Pharmaceuticals
name: Monica Gangal
role: STUDY_DIRECTOR
References:
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.