Cystic Fibrosis, Pancreatic Insufficiency
Conditions
Keywords
Pancreatic Insufficiency, Cystic Fibrosis, Enzymes
Brief summary
The purpose of this study is to determine the most effective dose of a new enzyme therapy on oral nutrient absorption in cystic fibrosis (CF) subjects with exocrine pancreatic insufficiency.
Interventions
DRUGLiprotamase
Administered orally as either Size 2 capsules (20,000 Units lipase; 20,000 Units protease; and 3,000 Units amylase) or Size 5 capsules (5,000 Units lipase; 5,000 Units protease; and 750 Units amylase)
DRUGPlacebo
Administered orally in either Size 2 or Size 5 capsules
Sponsors
Cystic Fibrosis Foundation
Anthera Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
7 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Pancreatic insufficiency (PI) documented by fecal elastase \<100 mcg/gram measured at screening * Diagnosis of CF based upon the following criteria: * two clinical features consistent with CF AND * either genotype with two identifiable mutations consistent with CF OR * sweat chloride \> 60 mEq/L by quantitative pilocarpine iontophoresis * Forced Expiratory Volume Over One Second (FEV1) ≥30% predicted * Clinically stable with no evidence of acute upper or lower respiratory tract infection
Exclusion criteria
* Pregnancy, breastfeeding or of childbearing potential and/or not willing to use accepted methods of birth control during enrollment in the study * History of fibrosing colonopathy * History of liver transplant or lung transplant * Unable to discontinue enteral tube feedings during the study * Subject weight \<40 kg * Known hypersensitivity to food additives * Participation in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to screening * Unable to discontinue the use of pancreatic enzymes for a 72-hour period or any other condition that the investigator believes would interfere with the intent of this study or would make study participation not in the best interest of the subject
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline up to 19 days in Coefficient of fat absorption (CFA) | Baseline, up to 19 days |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline up to 19 day in Coefficient of nitrogen absorption (CNA) | Baseline, up to 19 days |
| Change from baseline up to 19 days in stool weight | Baseline, up to 19 days |
| Change from baseline to 29 day endpoint in Cystic Fibrosis Questionnaire (CFQ) scale scores | Baseline, 29 days |
| Change from baseline up to 19 days in number of Stools | Baseline, up to 19 days |
| Change from baseline to 28 days in maximum change in glucose response | Baseline through 28 days |
Countries
United States
Outcome results
None listed