Exocrine Pancreatic Insufficiency: Cystic Fibrosis
Conditions
Keywords
Exocrine Pancreatic Insufficiency, Cystic Fibrosis, Pancreatic Insufficiency
Brief summary
The purpose of the study is to further evaluate the safety and efficacy of EUR-1008 as compared to Kreon® in the treatment of exocrine pancreatic insufficiency associated with Cystic Fibrosis in subjects 12 years of age and older.
Interventions
DRUGEUR-1008 25,000 Units
EUR-1008 25,000 Units is a PEP with pancreas powder as the active ingredient.18 Pancreas powder contains various enzymes having proteolytic, lipolytic, and amylolytic activity. Each capsule contains approximately 25,000 Ph. Eur. lipase units. EUR-1008 25,000 consists of orally administered capsules containing enteric-coated beads.
DRUGKreon 25,000 Units
Kreon 25,000 is a PEP consisting of porcine-derived pancreatic enzymes.18 Each capsule contains approximately 25,000 Ph. Eur. lipase units. Kreon 25,000 consists of orally administered capsules containing enteric-coated spheres.
Sponsors
Forest Laboratories
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Definitive diagnosis of CF based on the following: * One clinical feature consistent with CF and * Either a genotype with 2 identifiable mutations known to cause CF or a sweat chloride concentration \>60 mEq/L by pilocarpine iontophoresis 2. Pancreatic insufficiency documented by a monoclonal faecal elastase (FE) 100 μg/g stool at screening (test results within the previous 12 months are acceptable) 3. Currently receiving pancreatic enzyme replacement therapy 4. Adequate nutritional status based on the following: body mass index (BMI) \>19 kg/m2 in adult subjects or a BMI percentile 10th percentile for age in adolescent (12 to 17 years age group) subjects 5. Are clinically stable with no evidence of concomitant illness or acute upper or lower respiratory tract infection that requires antibiotics during the 7-day interval prior to screening and preceding entry into this clinical study
Exclusion criteria
1. Age \<12 years 2. Known contraindication, hypersensitivity, or intolerance to pork or other porcine PEPs 3. Current uncontrolled diabetes mellitus 4. History of solid organ transplantation 5. History of surgery affecting the bowel function and weight gain
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Coefficient of Fat Absorption over 72 hours (CFA-72h) | 72 hours | During the last 72 hours of each treatment period, the CFA-72h will be calculated using fat intake data from the diet and fat excretion data from stools. Fat intake will be calculated by the dietician in collaboration with the study investigator using a validated tool. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Coefficient of nitrogen absorption | 72 hours | Coefficient of nitrogen absorption at the end of each treatment period as assessed by a specialised central laboratory by means of Dumas combustion method. |
| Control of signs and symptoms of EPI | 2- 14 day periods | Control of signs and symptoms of EPI (as recorded in subject diaries). The following will be captured: * Stools frequency (number/day) * Stools consistency (hard, formed/normal; soft, watery, overt diarrhoea) * Fat or grease visible in stools (Yes/No) * Abdominal pain (mild, moderate, severe) * Bloating (mild, moderate, severe) * Flatulence (mild, moderate, severe) |
| Impact on overall health, daily life, perceived well-being, and symptoms | 58 days | Impact on overall health, daily life, perceived well-being, and symptoms evaluated using the CFQ (administered by designated study personnel prior to randomisation and at the end of each treatment period). |
| Total cholesterol, calculated LDL-C, HDL-C | 58 days | Total cholesterol, calculated LDL-C, HDL-C (sampling performed prior to randomisation and at the end of each treatment period). |
| Body weight | 58 days. | Body weight at baseline (Visit 2 \[Day 0\]) and at the end of each treatment period. |
| Standard safety laboratory tests | 58 days | Standard safety laboratory tests, analysed by central laboratory: * Haematology: red blood cell count, haemoglobin, haematocrit, total leukocytes with diff count, and platelets * Serum biochemistry: alanine aminotransferase, aspartate aminotransferase, alkaline phosphatase, total protein, albumin, total bilirubin, direct and indirect bilirubin, blood urea nitrogen, uric acid, creatinine, fasting plasma glucose, fasting cholesterol evaluations (total cholesterol, LDL-C, HDL-C, and triglycerides), fat-soluble vitamins (A, D, and E) and serum electrolytes |
| Vital signs | 78 days | Vital signs including blood pressure, heart rate, respirations and body temperature. |
| Fat-soluble vitamins A, D, and E | 58 days | Fat-soluble vitamins A, D, and E (sampling performed prior to randomisation and at the end of each treatment period). |
| Treatment Emergent Adverse Events | 78 days | Frequency, duration, and severity of treatment-emergent adverse events (TEAEs); |
Countries
Belgium, Bulgaria, France, Germany, Italy, Poland, United Kingdom
Outcome results
None listed