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

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02279498
• Other study ID #: AN-EPI3331
• Status: Completed
• Phase: Phase 3
• Start date: June 2015
• Est. completion date: January 20, 2017

Study information

• Verified date: July 2018
• Source: Anthera Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 128
• Est. completion date: January 20, 2017
• Est. primary completion date: October 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 7 Years and older

Eligibility

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%

Related Conditions & MeSH terms

• Cystic Fibrosis
• Exocrine Pancreatic Insufficiency
• Fibrosis

Intervention

Drug:
• Liprotamase
oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
• porcine (pig) PERT
oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source

Locations

Country	Name	City	State
Canada	Investigator Site 133	Edmonton	Alberta
Czechia	Investigator Site 501	Brno
Czechia	Investigator Site 502	Plzen
Hungary	Investigator Site 301	Ajka	Veszprem County
Hungary	Investigator Site 303	Debrecen	Hajdu-Bihar
Hungary	Investigator Site 304	Mosdos	Somogy County
Hungary	Investigator Site 305	Szeged	Csongrad County
Hungary	Investigator Site 302	Torokbalint	Pest County
Israel	Investigator Site 601	Jerusalem
Poland	Investigator Site 208	Bialystok
Poland	Investigator Site 203	Karpacz
Poland	Investigator Site 206	Lodz
Poland	Investigator Site 201	Lublin
Poland	Investigator Site 205	Lublin
Poland	Investigator Site 202	Rabka Zdroj
Poland	Investigator Site 209	Rzeszow
Poland	Investigator Site 204	Sopot
Poland	Investigator Site 207	Warszawa
Spain	Investigator Site 401	Madrid
Spain	Investigator Site 403	Madrid
Spain	Investigator Site 402	Malaga
Spain	Investigator Site 404	Valencia
United States	Investigator Site 124	Ann Arbor	Michigan
United States	Investigator Site 110	Atlanta	Georgia
United States	Investigator Site 114	Aurora	Colorado
United States	Investigator Site 121	Burlington	Vermont
United States	Investigator Site 127	Chicago	Illinois
United States	Investigator Site 103	Cleveland	Ohio
United States	Investigator Site 111	Dallas	Texas
United States	Investigator Site 126	East Lansing	Michigan
United States	Investigator Site 125	Fort Worth	Texas
United States	Investigator Site 120	Gainesville	Florida
United States	Investigator Site 109	Glenview	Illinois
United States	Investigator Site 106	Hershey	Pennsylvania
United States	Investigator Site 116	Houston	Texas
United States	Investigator Site 104	Indianapolis	Indiana
United States	Investigator Site 134	Jackson	Mississippi
United States	Investigator Site 102	Jacksonville	Florida
United States	Investigator Site 135	Las Vegas	Nevada
United States	Investigator Site 128	Lexington	Kentucky
United States	Investigator Site 123	Long Beach	California
United States	Investigator Site 107	Los Angeles	California
United States	Investigator Site 122	Louisville	Kentucky
United States	Investigator Site 130	Miami	Florida
United States	Investigator Site 129	Morgantown	West Virginia
United States	Investigator Site 101	Oklahoma City	Oklahoma
United States	Investigator Site 136	Oklahoma City	Oklahoma
United States	Investigator Site 117	Orlando	Florida
United States	Investigator Site 115	Pittsburgh	Pennsylvania
United States	Investigator Site 119	Portland	Oregon
United States	Investigator Site 132	Portland	Maine
United States	Investigator Site 112	Richmond	Virginia
United States	Investigator Site 113	Toledo	Ohio
United States	Investigator Site 105	Wichita	Kansas

Sponsors (1)

Lead Sponsor	Collaborator
Anthera Pharmaceuticals

Countries where clinical trial is conducted

United States,  Canada,  Czechia,  Hungary,  Israel,  Poland,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Treatment Difference in Coefficient of Fat Absorption (CFA) Change From Baseline	The primary endpoint evaluates the difference between treatment arms in change from baseline in coefficient of fat absorption (CFA). As such, descriptive statistics for individual treatment arms are not provided in this measure, but are reported in the secondary endpoints	Baseline, 7 weeks
Secondary	Coefficient of Fat Absorption (CFA)	Change from baseline in coefficient of fat absorption	Baseline, 7 weeks
Secondary	Coefficient of Nitrogen Absorption (CNA)	Change from baseline in coefficient of nitrogen absorption	Baseline, 7 weeks