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

• Sponsor: Anthera Pharmaceuticals
• Information provided by (Responsible Party): Anthera Pharmaceuticals

Tracking Information

• First Submitted Date: October 28, 2014
• First Posted Date: October 31, 2014
• Results First Submitted Date: April 10, 2018
• Results First Posted Date: August 14, 2018
• Last Update Posted Date: August 14, 2018
• Actual Study Start Date: June 2015
• Actual Primary Completion Date: October 2016 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (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 (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 (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 (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: SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis
• 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

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: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Exocrine Pancreatic Insufficiency
• Cystic Fibrosis

Intervention

• 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

• 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

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: May 19, 2017): 128
• Original Estimated Enrollment (submitted: October 28, 2014): 126
• Actual Study Completion Date: January 20, 2017
• Actual Primary Completion Date: October 2016 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

• Sexes Eligible for Study: All

• Ages: 7 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Canada, Czechia, Hungary, Israel, Poland, Spain, United States
• Removed Location Countries: Czech Republic

Administrative Information

• NCT Number: NCT02279498
• Other Study ID Numbers: AN-EPI3331
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Anthera Pharmaceuticals
• Study Sponsor: Anthera Pharmaceuticals
• Collaborators: Not Provided

Investigators

• Study Director: Monica Gangal; Anthera Pharmaceuticals

• PRS Account: Anthera Pharmaceuticals
• Verification Date: July 2018