This trial is active, not recruiting.

Conditions exocrine pancreatic insufficiency, cystic fibrosis
Treatments liprotamase, porcine (pig) pert
Phase phase 3
Sponsor Anthera Pharmaceuticals
Start date June 2015
End date February 2017
Trial size 126 participants
Trial identifier NCT02279498, AN-EPI3331


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.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
Individually-optimized dose to be administered orally
oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
(Active Comparator)
Individually-optimized dose to be administered orally
porcine (pig) pert
oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source

Primary Outcomes

Coefficient of Fat Absorption (CFA)
time frame: Two time points, change from baseline CFA after up to 8 weeks of stabilized therapy] [Designated as safety issue: No]

Secondary Outcomes

Safety, as measured by number of participants with adverse events or laboratory abnormalities
time frame: 18-20 weeks

Eligibility Criteria

Male or female participants at least 7 years old.

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%

Additional Information

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
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by Anthera Pharmaceuticals.