RESULT: Reliable, Emergent Solution Using Liprotamase Treatment

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

Status

UNKNOWN

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03051490

Enrollment

140

Registered

2017-02-13

Start date

2017-04-28

Completion date

2018-06-30

Last updated

2018-03-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic Fibrosis, Exocrine Pancreatic Insufficiency

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).

Interventions

DRUGLiprotamase

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

DRUGporcine PERT

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

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

NONE

Masking description

unblinded to treatment randomization; blinded to primary efficacy variable

Eligibility

Sex/Gender

ALL

Age

7 Years to No maximum

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%

Design outcomes

Primary

Measure	Time frame	Description
Coefficient of Fat Absorption (CFA)	8 weeks	Non-inferiority of Liprotamase to approved porcine PERT

Secondary

Measure	Time frame	Description
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

Countries

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

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026