RESULT: Reliable, Emergent Solution Using Liprotamase Treatment

Cystic Fibrosis Clinical Trial

Official title:

A Phase 3, Randomized, Open-Label, Assessor-Blind, Non-Inferiority, Active-Comparator Study Evaluating the Efficacy and Safety of Liprotamase in Subjects With Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03051490
• Other study ID #: AN-EPI3333
• Status: Active, not recruiting
• Phase: Phase 3
• First received: February 9, 2017
• Last updated: March 6, 2018
• Start date: April 28, 2017
• Est. completion date: June 2018

Study information

• Verified date: January 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 biotechnology-derived lipase, protease, and amylase digestive enzymes. The purpose of the present study is to to evaluate the non-inferiority of liprotamase compared with porcine-derived, enterically-coated pancreatic enzyme replacement therapy (PERT). The primary efficacy endpoint of the study will be comparative efficacy measured as the change from baseline in the coefficient of fat absorption (CFA) in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 140
• Est. completion date: June 2018
• Est. primary completion date: February 28, 2018
• 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

• Good disease control with porcine PERT prior to enrollment

• 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 PERT
oral, enterically-coated, pig-derived, pancreatic enzyme replacement

Locations

Country	Name	City	State
Hungary	Investigator Site 306	Budapest
Hungary	Investigator Site 307	Budapest
Hungary	Investigator Site 304	Mosdós	Somogy County
Hungary	Investigator Site 302	Torokbalint	Pest County
Israel	Investigator Site 601	Jerusalem
Lithuania	Investigator Site 901	Kaunas
Lithuania	Investigator Site #902	Vilnius
Poland	Investigator Site 203	Karpacz
Poland	Site 206	Lódz
Poland	Investigator Site 210	Lomianki
Poland	Investigator Site 201	Lublin
Poland	Investigator Site 205	Lublin
Poland	Investigator Site 202	Rabka-Zdrój
Poland	Investigator Site 209	Rzeszów
Poland	Investigator 204	Sopot
Spain	Investigator Site 405	El Palmar	Murcia
Spain	Investigator Site 401	Madrid
Spain	Investigator Site 403	Madrid
Spain	Investigator Site 402	Málaga
Spain	Investigator Site 404	Valencia
United Kingdom	Investigator Site 702	Exeter
United Kingdom	Investigatior Site 701	Southampton	Hampshire
United States	Investigator Site 117	Altamonte Springs	Florida
United States	Investigator Site 124	Ann Arbor	Michigan
United States	Investigator Site 110	Atlanta	Georgia
United States	Investigator Site #103	Cleveland	Ohio
United States	Investigator Site 111	Dallas	Texas
United States	Investigator Site 150	Denver	Colorado
United States	Investigator Site 118	Durham	North Carolina
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 148	Iowa City	Iowa
United States	Investigator Site 134	Jackson	Mississippi
United States	Investigator Site 102	Jacksonville	Florida
United States	Investigator Site 140	Kalamazoo	Michigan
United States	Investigator Site 135	Las Vegas	Nevada
United States	Investigator Site 139	Little Rock	Arkansas
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 101	Oklahoma City	Oklahoma
United States	Investigator Site 136	Oklahoma City	Oklahoma
United States	Investigator Site 143	Orange	California
United States	Investigator Site 132	Portland	Maine
United States	Investigator Site 112	Richmond	Virginia
United States	Site Investigator #113	Toledo	Ohio
United States	Investigator Site 151	West Palm Beach	Florida
United States	Investigator Site 147	Wilmington	Delaware

Sponsors (1)

Lead Sponsor	Collaborator
Anthera Pharmaceuticals

Countries where clinical trial is conducted

United States,  Hungary,  Israel,  Lithuania,  Poland,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Coefficient of Fat Absorption (CFA)	Non-inferiority of Liprotamase to approved porcine PERT	8 weeks
Secondary	Coefficient of Nitrogen Absorption (CNA)	Non-inferiority of Liprotamase to approved porcine PERT	8 weeks
Secondary	Safety, as measured by number of participants with adverse events or laboratory abnormalities	Change from baseline	6 months