Clinical trial NCT02734810: SIMPLICITY: Studying Impacts on Malabsorption With Liprotamase in Cystic Fibrosis
Official title: A Phase 3, Open-Label 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
Keywords: Exocrine Pancreatic Insufficiency
Cystic Fibrosis
Liprotamase
Pancreatic Enzyme Replacement Therapy (PERT)
Lead sponsor: Anthera Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2016-04-12
Enrollment: 15
Sex: ALL
Minimum age (years): 28
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Liprotamase consists of 3 soluble, non-porcine digestive enzymes, lipase, protease, and amylase, combined in a fixed ratio. Liprotamase is stable in the stomach and can be formulated without enteric coating for administration either as a capsule or as a dosing solution dissolved in water or juice. The purpose of the present study is to provide efficacy and safety data for a new, soluble formulation of liprotamase, Liprotamase Powder for Oral Solution, in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI).


Eligibility criteria: Inclusion Criteria:

* For Part A: males or females ≥7 years of age
* For Part B: males or females 28 days to \<7 years
* Diagnosis of cystic fibrosis based on presentation, genotype and/or sweat chloride
* Low fecal elastase
* Fair-to-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

Primary outcomes:
measure: Safety
timeFrame: 1 week
Secondary outcomes:
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Investigator Site 139
state: Arkansas
city: Orange
country: United States
facility: Investigator Site
state: California
city: Aurora
country: United States
facility: Investigator Site 114
state: Colorado
city: Altamonte Springs
country: United States
facility: Investigator Site 117
state: Florida
city: Hollywood
country: United States
facility: Investigator Site 138
state: Florida
city: Miami
country: United States
facility: Investigator Site 130
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: Wichita
country: United States
facility: Investigator Site 105
state: Kansas
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: Las Vegas
country: United States
facility: Investigator Site 135
state: Nevada
city: Durham
country: United States
facility: Investigator Site 118
state: North Carolina
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: Morgantown
country: United States
facility: Investigator Site 129
state: West Virginia
Study officials:
References: