Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Conditions
Keywords
Cystic fibrosis-related exocrine pancreatic insufficiency
Brief summary
This is an open-label, single-arm clinical study investigating the long-term safety of ALTU-135 treatment in patients with cystic fibrosis (CF)-related exocrine pancreatic insufficiency (PI).
Interventions
DRUGLiprotamase
Administered orally
Sponsors
Anthera Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
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 * A genotype with two identifiable mutations consistent with CF, or * Sweat chloride \> 60 milliequivalents per liter (mEq/L) by quantitative pilocarpine iontophoresis. * Clinically stable with no evidence of acute upper or lower respiratory tract infection * Exocrine pancreatic insufficiency (PI) determined by fecal elastase \< 100 micrograms per gram (µg/g) measured at Screening * Able to take pancreatic enzyme supplementation in the form of capsules * Able to perform the testing and procedures required for this study, as judged by the Investigator
Exclusion criteria
* Age \<7 years * Pregnancy, breastfeeding or of childbearing potential and not willing to use birth control * Participation in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to Screening * History of fibrosing colonopathy * History of liver transplant, lung transplant or significant surgical resection of the bowel * Any chronic diarrheal illness unrelated to PI * Abnormal liver function (except for patients with Gilbert Syndrome) * Signs and/or symptoms of liver cirrhosis or portal hypertension (e.g., splenomegaly, ascites, esophageal varices), or documented liver disease unrelated to CF * Unable to discontinue enteral tube feedings during the study * 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 * Baseline coefficient of fat absorption (CFA) ≥ 93% from the Phase III efficacy study * Patient is unlikely to complete the study, as determined by the Investigator
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of participants with an adverse event | Baseline up to 12 months |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline up to 12 months in body weight Z-scores | Baseline, up to 12 months |
| Change from baseline up to 12 months in height Z-scores (up to 17 years in participants age) | Baseline, up to 12 months |
| Change from baseline up to 12 months in Body Mass Index (BMI) Z-scores | Baseline, up to 12 months |
Countries
United States
Outcome results
None listed