Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Conditions
Keywords
Exocrine Pancreatic Insufficiency, Cystic Fibrosis, Liprotamase, Pancreatic Enzyme Replacement Therapy (PERT)
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. The purpose of the present study is to provide efficacy and safety data for a new, soluble formulation of liprotamase, Liprotamase Powder for Oral Solution, in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI).
Interventions
DRUGLiprotamase Powder for Oral Solution
Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy
Sponsors
Anthera Pharmaceuticals
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
28 Days to No maximum
Healthy volunteers
No
Inclusion criteria
* For Part A: males or females ≥7 years of age * For Part B: males or females 28 days to \<7 years * Diagnosis of cystic fibrosis based on presentation, genotype and/or sweat chloride * Low fecal elastase * Fair-to-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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety | 1 week | Number of subjects reporting 1 or more adverse events |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Part A Liprotamase Powder for Oral Solution in Subjects aged ≥7 years of age
Liprotamase Powder for Oral Solution: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy | 15 |
| Part B Liprotamase Powder for Oral Solution in Subjects aged 28 days to \<7 years
Liprotamase Powder for Oral Solution: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy | 0 |
| Total | 15 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 1 | 0 |
| Overall Study | Lack of Efficacy | 1 | 0 |
Baseline characteristics
| Characteristic | Part A | Total | Part B |
|---|---|---|---|
| Age, Continuous | 24.1 years | 24.1 years | — |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 15 Participants | 15 Participants | 0 Participants |
| Region of Enrollment United States | 15 participants | 15 participants | — |
| Sex: Female, Male Female | 3 Participants | 3 Participants | 0 Participants |
| Sex: Female, Male Male | 12 Participants | 12 Participants | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 15 | 0 / 0 |
| other Total, other adverse events | 3 / 15 | 0 / 0 |
| serious Total, serious adverse events | 1 / 15 | 0 / 0 |
Outcome results
Primary
Safety
Number of subjects reporting 1 or more adverse events
Time frame: 1 week
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Part A | Safety | 3 Participants |
| Part B | Safety | 0 Participants |