SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis

Status:	Completed
Conditions:	Pulmonary
Therapuetic Areas:	Pulmonary / Respiratory Diseases
Healthy:	No
Age Range:	7 - Any
Updated:	8/16/2018
Start Date:	June 2015
End Date:	January 20, 2017

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

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.

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).

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%

We found this trial at

sites

Clinical Trial Facility in Gainesville Florida

Gainesville, Florida 32610

711

from 43215

Gainesville, FL