Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Conditions
Keywords
exocrine pancreatic insufficiency, cystic fibrosis, pancreatic enzyme replacement therapy
Brief summary
Liprotamase consists of 3 soluble, non-porcine digestive enzymes, lipase, protease, and amylase, combined in a fixed ratio. Liprotamase is stable in the stomach and can be formulated without enteric coating for administration either as a capsule or as a dosing solution dissolved in water or juice.
Detailed description
The purpose of the present study is to evaluate the safety of long-term use of liprotamase in the management of cystic fibrosis-related exocrine pancreatic insufficiency
Interventions
DRUGLiprotamase
Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
Sponsors
Anthera Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
7 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female subjects with cystic fibrosis-related exocrine pancreatic insufficiency who received liprotamase and completed Study AN-EPI3331 (SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis)
Exclusion criteria
* Any medical, psychological, or social condition that may put the subject at increased risk by participating in this study. * Females who are nursing, pregnant, intending to become pregnant, or intending to nurse during the time of the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety, as Measured by Number of Participants With Adverse Events Including Clinical or Laboratory Abnormalities | 12 months | Descriptive analysis |
Countries
Czechia, Hungary, Israel, Poland, Spain, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Liprotamase Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
Liprotamase: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement | 25 |
| Total | 25 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 2 |
| Overall Study | Lack of Efficacy | 1 |
| Overall Study | Physician Decision | 1 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Liprotamase |
|---|---|
| Age, Continuous | 22.94 years STANDARD_DEVIATION 6.84 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 25 Participants |
| Sex: Female, Male Female | 11 Participants |
| Sex: Female, Male Male | 14 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 25 |
| other Total, other adverse events | 9 / 25 |
| serious Total, serious adverse events | 3 / 25 |
Outcome results
Primary
Safety, as Measured by Number of Participants With Adverse Events Including Clinical or Laboratory Abnormalities
Descriptive analysis
Time frame: 12 months
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Liprotamase | Safety, as Measured by Number of Participants With Adverse Events Including Clinical or Laboratory Abnormalities | Safety population | 25 Participants |
| Liprotamase | Safety, as Measured by Number of Participants With Adverse Events Including Clinical or Laboratory Abnormalities | total number of subjects with adverse events | 9 Participants |