Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency
Conditions
Interventions
Product Name: Liprotamase
Pharmaceutical Form: Capsule
INN or Proposed INN: NOT ASSIGNED
CAS Number: 9001-62-1
Other descriptive name: LIPASE
Concentration unit: IU international unit(s)
Concentration
Sponsors
ANTHERA Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects are eligible for enrollment if they meet the following inclusion criteria: 1. Male or female subjects who received liprotamase and completed Study AN-EPI3331. 2. Signed informed consent by subject and/or subject’s legally authorized representative. Are the trial subjects under 18? yes Number of subjects for this age range: 35 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 35 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Any medical, psychological, or social condition that, at the discretion of the investigator, may put the subject at increased risk by participating in this study or confound interpretation of study results. 2. Treatment with any PERT other than study drug, investigational drugs, or devices 3. Females who are nursing, pregnant, intending to become pregnant, or intending to nurse during the time of the study, or who have a positive pregnancy test at Visit Day 1. All sexually-active subjects of reproductive potential are required to remain on a reliable method of birth control. Females and males are required to continue using a reliable method of birth control throughout the study, and for at least 3 months following completion of study therapy. A reliable method of birth control is defined as one of the following: oral or injectable contraceptives, intrauterine device, contraceptive implants, tubal ligation, hysterectomy, or a double-barrier method (diaphragm with spermicidal foam or jelly, or a condom), abstinence or vasectomy.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to evaluate the safety of long-term use of liprotamase in the management of Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency (EPI). Safety will be evaluated based on the occurrence of adverse events (AEs) and serious AEs (SAEs); including but not limited to abnormal laboratory results, abnormal findings in physical examinations and abnormal vital signs deemed clinically significant by the investigator.;Secondary Objective: The secondary endpoints will include: • Change in weight, height, and BMI from baseline. • Changes in measures of growth including weight, height, and BMI relative to population anthropomorphic measures. • Signs and symptoms of malabsorption including stool frequency, stool consistency, bloating, steatorrhea, abdominal pain, and flatulence compared with baseline.;Primary end point(s): The primary objective of this study is to evaluate the safety of long-term use of liprotamase in the management of EPI due to CF amongst subjects who received liprotamase and completed the prior study with liprotamase (AN-EPI3331). Safety will be evaluated based on adverse events (AEs) and serious AEs (SAEs); including but not limited to abnormal laboratory results, abnormal findings in physical examinations, and abnormal vital signs deemed clinically significant by the investigator. By-subject listings of all AEs, including abnormal and clinically significant vital sign parameters and physical examination results will be presented.;Timepoint(s) of evaluation of this end point: At each study Visit during the treatment period. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): In this study efficacy will be evaluated from measures of body weight and BMI (for adults) and growth (for pediatric subjects) relative to baseline and/or relative to published population growth charts. In addition, efficacy (i.e. control of EPI) will be evaluated from the signs and symptoms of malabsorption. The secondary endpoints will include: • Change in weight, height, and BMI from baseline. • Changes in measures of growth including weight, height, and BMI relative to population anthropomorphic measures. • Signs and symptoms of malabsorption including stool frequency, stool consistency, bloating, steatorrhea, abdominal pain, and flatulence compared with baseline. For efficacy analyses of change from baseline, baseline will be defined as the last observation prior to initiation of liprotamase dosing.;Timepoint(s) of evaluation of this end point: At each study Visit during the treatment period. | — |
Countries
Canada, Czech Republic, Hungary, Israel, Poland, Spain, United States
Contacts
Public ContactNicole Ramza
Anthera Pharmaceuticals, Inc.
Outcome results
None listed