RESULT: Reliable, Emergent Solution Using Liprotamase Treatment

Sponsor

Anthera Pharmaceuticals (Industry)

Overall Status

Unknown status

CT.gov ID

NCT03051490

Collaborator

(none)

140

Enrollment

Locations

Arms

13.1

Anticipated Duration (Months)

2.8

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief 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).

Condition or Disease	Intervention/Treatment	Phase
Exocrine Pancreatic Insufficiency Cystic Fibrosis	Drug: Liprotamase Drug: porcine PERT	Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

140 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Masking Description:

unblinded to treatment randomization; blinded to primary efficacy variable

Primary Purpose:

Treatment

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

Actual Study Start Date :

Apr 28, 2017

Actual Primary Completion Date :

Feb 28, 2018

Anticipated Study Completion Date :

Jun 1, 2018

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Liprotamase Individually-optimized dose to be administered orally	Drug: Liprotamase oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
Active Comparator: porcine PERT Individually-optimized dose to be administered orally	Drug: porcine PERT oral, enterically-coated, pig-derived, pancreatic enzyme replacement

Arm

Intervention/Treatment

Experimental: Liprotamase

Individually-optimized dose to be administered orally

Drug: Liprotamase

oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement

Active Comparator: porcine PERT

Individually-optimized dose to be administered orally

Drug: porcine PERT

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

Outcome Measures

Primary Outcome Measures

Coefficient of Fat Absorption (CFA) [8 weeks]
Non-inferiority of Liprotamase to approved porcine PERT

Secondary Outcome Measures

Coefficient of Nitrogen Absorption (CNA) [8 weeks]
Non-inferiority of Liprotamase to approved porcine PERT
Safety, as measured by number of participants with adverse events or laboratory abnormalities [6 months]
Change from baseline

Eligibility Criteria

Criteria

Ages Eligible for Study:

7 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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%

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Investigator Site 139	Little Rock	Arkansas	United States	72202
2	Investigator Site #123	Long Beach	California	United States	90806
3	Investigator Site 107	Los Angeles	California	United States	90033
4	Investigator Site 143	Orange	California	United States	92868
5	Investigator Site 150	Denver	Colorado	United States	80206
6	Investigator Site 147	Wilmington	Delaware	United States	19803
7	Investigator Site 117	Altamonte Springs	Florida	United States	32701
8	Investigator Site 102	Jacksonville	Florida	United States	32207
9	Investigator Site 130	Miami	Florida	United States	33136
10	Investigator Site 151	West Palm Beach	Florida	United States	33407
11	Investigator Site 110	Atlanta	Georgia	United States	30342
12	Investigator Site 109	Glenview	Illinois	United States	60025
13	Investigator Site 148	Iowa City	Iowa	United States	52242
14	Investigator Site #122	Louisville	Kentucky	United States	40202
15	Investigator Site 132	Portland	Maine	United States	04102
16	Investigator Site 124	Ann Arbor	Michigan	United States	48109
17	Investigator Site 140	Kalamazoo	Michigan	United States	49008
18	Investigator Site 134	Jackson	Mississippi	United States	39216
19	Investigator Site 135	Las Vegas	Nevada	United States	89107
20	Investigator Site 118	Durham	North Carolina	United States	27710
21	Investigator Site #103	Cleveland	Ohio	United States	44106
22	Site Investigator #113	Toledo	Ohio	United States	43606
23	Investigator Site 101	Oklahoma City	Oklahoma	United States	73104
24	Investigator Site 136	Oklahoma City	Oklahoma	United States	73112
25	Investigator Site 106	Hershey	Pennsylvania	United States	17033
26	Investigator Site 111	Dallas	Texas	United States	75235
27	Investigator Site 116	Houston	Texas	United States	77030
28	Investigator Site 112	Richmond	Virginia	United States	23298
29	Investigator Site 302	Torokbalint	Pest County	Hungary	2045
30	Investigator Site 304	Mosdós	Somogy County	Hungary	7257
31	Investigator Site 306	Budapest		Hungary	1089
32	Investigator Site 307	Budapest		Hungary	1121
33	Investigator Site 601	Jerusalem		Israel	9124001
34	Investigator Site 901	Kaunas		Lithuania	LT-50161
35	Investigator Site #902	Vilnius		Lithuania	LT-10207
36	Investigator Site 203	Karpacz		Poland	58-540
37	Investigator Site 210	Lomianki		Poland	05-092
38	Investigator Site 201	Lublin		Poland	20-093
39	Investigator Site 205	Lublin		Poland	20-362
40	Investigator Site 202	Rabka-Zdrój		Poland	34-700
41	Investigator Site 209	Rzeszów		Poland	35-612
42	Investigator 204	Sopot		Poland	81-713
43	Site 206	Łódź		Poland	90-239
44	Investigator Site 405	El Palmar	Murcia	Spain	30120
45	Investigator Site 401	Madrid		Spain	28046
46	Investigator Site 403	Madrid		Spain	28046
47	Investigator Site 402	Málaga		Spain	29009
48	Investigator Site 404	Valencia		Spain	46026
49	Investigatior Site 701	Southampton	Hampshire	United Kingdom	SO 16 6YD
50	Investigator Site 702	Exeter		United Kingdom	EX2 5DW

Sponsors and Collaborators

Anthera Pharmaceuticals

Investigators

Study Director: Monica Gangal, Anthera Pharmaceuticals

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Anthera Pharmaceuticals

ClinicalTrials.gov Identifier:

NCT03051490

Other Study ID Numbers:

AN-EPI3333

First Posted:

Feb 13, 2017

Last Update Posted:

Mar 8, 2018

Last Verified:

Jan 1, 2018

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Cystic Fibrosis

Exocrine Pancreatic Insufficiency

Fibrosis

Study Results

No Results Posted as of Mar 8, 2018