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

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ClinicalTrials.gov Identifier: NCT02279498
Recruitment Status : Completed
First Posted : October 31, 2014
Results First Posted : August 14, 2018
Last Update Posted : August 14, 2018
Sponsor:
Information provided by (Responsible Party):
Anthera Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE October 28, 2014
First Posted Date  ICMJE October 31, 2014
Results First Submitted Date  ICMJE April 10, 2018
Results First Posted Date  ICMJE August 14, 2018
Last Update Posted Date August 14, 2018
Actual Study Start Date  ICMJE June 2015
Actual Primary Completion Date October 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 17, 2018)
Treatment Difference in Coefficient of Fat Absorption (CFA) Change From Baseline [ Time Frame: Baseline, 7 weeks ]
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
Original Primary Outcome Measures  ICMJE
 (submitted: October 28, 2014)
Coefficient of Fat Absorption (CFA) [ Time Frame: Two time points, baseline and after stabilized therapy] [Designated as safety issue: No] ]
Non-inferiority of Liprotamase to approved PERT
Change History Complete list of historical versions of study NCT02279498 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: July 17, 2018)
  • Coefficient of Fat Absorption (CFA) [ Time Frame: Baseline, 7 weeks ]
    Change from baseline in coefficient of fat absorption
  • Coefficient of Nitrogen Absorption (CNA) [ Time Frame: Baseline, 7 weeks ]
    Change from baseline in coefficient of nitrogen absorption
Original Secondary Outcome Measures  ICMJE
 (submitted: October 28, 2014)
Safety [ Time Frame: 18-20 weeks ]
Change from baseline
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis
Official Title  ICMJE 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
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.

Detailed 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).
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Exocrine Pancreatic Insufficiency
  • Cystic Fibrosis
Intervention  ICMJE
  • Drug: Liprotamase
    oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
  • Drug: porcine (pig) PERT
    oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source
Study Arms  ICMJE
  • Experimental: Liprotamase
    Individually-optimized dose to be administered orally
    Intervention: Drug: Liprotamase
  • Active Comparator: porcine (pig) PERT
    Individually-optimized dose to be administered orally
    Intervention: Drug: porcine (pig) PERT
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 19, 2017)
128
Original Estimated Enrollment  ICMJE
 (submitted: October 28, 2014)
126
Actual Study Completion Date  ICMJE January 20, 2017
Actual Primary Completion Date October 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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%
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 7 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Czechia,   Hungary,   Israel,   Poland,   Spain,   United States
Removed Location Countries Czech Republic
 
Administrative Information
NCT Number  ICMJE NCT02279498
Other Study ID Numbers  ICMJE AN-EPI3331
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Anthera Pharmaceuticals
Study Sponsor  ICMJE Anthera Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Monica Gangal Anthera Pharmaceuticals
PRS Account Anthera Pharmaceuticals
Verification Date July 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP