NCT02279498

Brief 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.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
128

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Jun 2015

Geographic Reach
7 countries

54 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 28, 2014

Completed
3 days until next milestone

First Posted

Study publicly available on registry

October 31, 2014

Completed
7 months until next milestone

Study Start

First participant enrolled

June 1, 2015

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2016

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

January 20, 2017

Completed
1.6 years until next milestone

Results Posted

Study results publicly available

August 14, 2018

Completed
Last Updated

August 14, 2018

Status Verified

July 1, 2018

Enrollment Period

1.3 years

First QC Date

October 28, 2014

Results QC Date

April 10, 2018

Last Update Submit

July 17, 2018

Conditions

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (2)

  • Coefficient of Fat Absorption (CFA)

    Baseline, 7 weeks

  • Coefficient of Nitrogen Absorption (CNA)

    Baseline, 7 weeks

Study Arms (2)

Liprotamase

EXPERIMENTAL

Individually-optimized dose to be administered orally

Drug: Liprotamase

porcine (pig) PERT

ACTIVE COMPARATOR

Individually-optimized dose to be administered orally

Drug: porcine (pig) PERT

Interventions

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

Liprotamase

oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source

porcine (pig) PERT

Eligibility Criteria

Age7 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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%

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (54)

Investigator Site 123

Long Beach, California, 90806, United States

Location

Investigator Site 107

Los Angeles, California, 90033, United States

Location

Investigator Site 114

Aurora, Colorado, 80045, United States

Location

Investigator Site 120

Gainesville, Florida, 32610, United States

Location

Investigator Site 102

Jacksonville, Florida, 32207, United States

Location

Investigator Site 130

Miami, Florida, 33136, United States

Location

Investigator Site 117

Orlando, Florida, 32803, United States

Location

Investigator Site 110

Atlanta, Georgia, 30342, United States

Location

Investigator Site 127

Chicago, Illinois, 60611, United States

Location

Investigator Site 109

Glenview, Illinois, 60025, United States

Location

Investigator Site 104

Indianapolis, Indiana, 46202, United States

Location

Investigator Site 105

Wichita, Kansas, 67214, United States

Location

Investigator Site 128

Lexington, Kentucky, 40506, United States

Location

Investigator Site 122

Louisville, Kentucky, 40202, United States

Location

Investigator Site 132

Portland, Maine, 04102, United States

Location

Investigator Site 124

Ann Arbor, Michigan, 48109-5212, United States

Location

Investigator Site 126

East Lansing, Michigan, 48823, United States

Location

Investigator Site 134

Jackson, Mississippi, 39216, United States

Location

Investigator Site 135

Las Vegas, Nevada, 89107, United States

Location

Investigator Site 103

Cleveland, Ohio, 44106, United States

Location

Investigator Site 113

Toledo, Ohio, 43606, United States

Location

Investigator Site 101

Oklahoma City, Oklahoma, 73104, United States

Location

Investigator Site 136

Oklahoma City, Oklahoma, 73112, United States

Location

Investigator Site 119

Portland, Oregon, 97239, United States

Location

Investigator Site 106

Hershey, Pennsylvania, 17033, United States

Location

Investigator Site 115

Pittsburgh, Pennsylvania, 15224, United States

Location

Investigator Site 111

Dallas, Texas, 75390, United States

Location

Investigator Site 125

Fort Worth, Texas, 76104, United States

Location

Investigator Site 116

Houston, Texas, 77030, United States

Location

Investigator Site 121

Burlington, Vermont, 05405, United States

Location

Investigator Site 112

Richmond, Virginia, 23219, United States

Location

Investigator Site 129

Morgantown, West Virginia, 26506, United States

Location

Investigator Site 133

Edmonton, Alberta, T6G IC9, Canada

Location

Investigator Site 501

Brno, 62500, Czechia

Location

Investigator Site 502

Pilsen, 305 99, Czechia

Location

Investigator Site 305

Szeged, Csongrád megye, 6720, Hungary

Location

Investigator Site 303

Debrecen, Hajdú-Bihar, 4031, Hungary

Location

Investigator Site 302

Törökbálint, Pest County, 2045, Hungary

Location

Investigator Site 304

Mosdós, Somogy County, 7257, Hungary

Location

Investigator Site 301

Ajka, Veszprém megye, 8400, Hungary

Location

Investigator Site 601

Jerusalem, 9124001, Israel

Location

Investigator Site 208

Bialystok, 15-044, Poland

Location

Investigator Site 203

Karpacz, 58-540, Poland

Location

Investigator Site 206

Lodz, 90-329, Poland

Location

Investigator Site 201

Lublin, 20-093, Poland

Location

Investigator Site 205

Lublin, 20-362, Poland

Location

Investigator Site 202

Rabka-Zdrój, 34-700, Poland

Location

Investigator Site 209

Rzeszów, 35-312, Poland

Location

Investigator Site 204

Sopot, 81-713, Poland

Location

Investigator Site 207

Warsaw, 01-195, Poland

Location

Investigator Site 403

Madrid, 28006, Spain

Location

Investigator Site 401

Madrid, 28046, Spain

Location

Investigator Site 402

Málaga, 29009, Spain

Location

Investigator Site 404

Valencia, 46026, Spain

Location

Related Publications (1)

  • Somaraju URR, Solis-Moya A. Pancreatic enzyme replacement therapy for people with cystic fibrosis. Cochrane Database Syst Rev. 2020 Aug 5;8(8):CD008227. doi: 10.1002/14651858.CD008227.pub4.

MeSH Terms

Conditions

Exocrine Pancreatic InsufficiencyCystic Fibrosis

Interventions

liprotamase lipase

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Results Point of Contact

Title
Monica Gangal
Organization
Anthera Pharmaceuticals

Study Officials

  • Monica Gangal

    Anthera Pharmaceuticals

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 28, 2014

First Posted

October 31, 2014

Study Start

June 1, 2015

Primary Completion

October 1, 2016

Study Completion

January 20, 2017

Last Updated

August 14, 2018

Results First Posted

August 14, 2018

Record last verified: 2018-07

Locations