Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Conditions
Keywords
cystic fibrosis-related pancreatic insufficiency
Brief summary
This is a clinical trial which will evaluate the efficacy of Liprotamase treatment in cystic fibrosis (CF) patients with exocrine pancreatic insufficiency (PI).
Detailed description
This trial is divided into four distinct periods: 1. Baseline Period during which each patient is taken off pancreatic enzyme medications. 2. An Open-Label Treatment Period during which all patients will receive ALTU-135 (liprotamase). 3. Inpatient, Double Blind Treatment Period during which half of patients will be withdrawn from treatment and will receive Placebo. 4. Second Open-Label Treatment Period during which all patients will resume treatment with ALTU-135 (liprotamase).
Interventions
DRUGLiprotamase
Administered orally
DRUGPlacebo
Administered orally
Sponsors
Anthera Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
7 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Females of childbearing potential must be willing to use birth control * 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 * Clinically stable with no evidence of acute upper or lower respiratory tract infection * PI determined by fecal elastase \<100 µg/g stool measured at screening * Able to take pancreatic enzyme supplementation in the form of capsules * Able to perform the testing (e.g., stool collections) and inpatient stays required for this study, as judged by the Investigator * Baseline coefficient of fat absorption (CFA) less than or equal to 80%
Exclusion criteria
* CFA \>80% at Baseline * Pregnancy, breastfeeding or of childbearing potential and not willing to use birth control during the study * History of fibrosing colonopathy * History of liver transplant, lung transplant or significant surgical resection of the bowel * Any acute or chronic diarrheal illness unrelated to PI * Unable to discontinue enteral tube feedings during the study * Known hypersensitivity to food additives * Inability to consume the diet required by the study, in the judgment of the Investigator * Participation in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to screening * Abnormal liver function (except for patients with Gilbert Syndrome) * Signs and/or symptoms of liver cirrhosis, portal hypertension or documented liver disease unrelated to CF * Distal intestinal obstruction syndrome (DIOS) in the last six months prior to screening * Unable to discontinue the use of pancreatic enzymes * Any condition that the Investigator believes would interfere with the intent of this study or would make participation not in the best interest of the patient * Patient is unlikely to complete the study, as determined by the Investigator
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from open label baseline to end of 6 day double blind treatment period in Coefficient of Fat Absorption (CFA) | Open label baseline, end of 6 day double blind treatment period |
Secondary
| Measure | Time frame |
|---|---|
| Change from open label baseline to end of 6 day double blind treatment period in Coefficient of nitrogen absorption (CNA) | Open label baseline, end of 6 day double blind treatment period |
| Change from open label baseline to end of 6 day double blind treatment period in number of stools | Open label baseline, end of 6 day double blind treatment period |
| Change from open label baseline to end of 6 day double blind treatment period in stool weight | Open label baseline, end of 6 day double blind treatment period |
| Change from open label baseline to end of 6 day double blind treatment period in maximum blood glucose | Open label baseline, end of 6 day double blind treatment period |
Countries
United States
Outcome results
None listed