Patients with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency MedDRA version: 9.1 Level: LLT Classification code 10011766 Term: Cystic fibrosis pancreatic
Conditions
Interventions
Product Name: ALTU-135
Product Code: ALTU-135
Pharmaceutical Form: Capsule*
INN or Proposed INN: NA
CAS Number: 9001-62-1
Other descriptive name: Lipase CLEC
Concentration unit: U unit(s)
Concentrati
Sponsors
Altus Pharmaceuticals Inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Each patient must meet the following criteria to be enrolled in this study. 1. = 7 years of age. 2. Females of childbearing potential must be willing to use birth control (IUD; oral, transdermal or parenteral contraceptives; abstinence). 3. Diagnosis of CF based upon the following criteria: a. two clinical features consistent with CF; and b. either genotype with two identifiable mutations consistent with CF, c. or sweat chloride > 60 mEq/L by quantitative pilocarpine iontophoresis. 4. Clinically stable with no evidence of acute upper or lower respiratory tract infection. 5. PI determined by fecal elastase =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Patients who meet any of the following criteria will be excluded from the study. 1. 80% at Baseline. 3. Pregnancy, breastfeeding or of childbearing potential and not willing to use birth control (IUD; oral, transdermal or parenteral contraceptives; abstinence) during the study. 4. History of fibrosing colonopathy. 5. History of liver transplant, lung transplant or significant surgical resection of the bowel. Note: Significant resection of the bowel is defined as any resection of the terminal ileum, or ileo-cecal valve. Patients who have had qualitative, long-term changes in nutritional status after any other bowel resection (e.g., increased, or new, need for supplementation compared to pre-op in order to maintain the same nutritional status) should also be excluded. 6. Any acute or chronic diarrheal illness unrelated to PI (e.g. infectious gastroenteritis, sprue, lactose intolerance, inflammatory bowel disease). 7. Unable to discontinue enteral tube feedings during the study. 8. Known hypersensitivity to food additives. 9. Inability to consume the PP diets, in the judgment of the Investigator. 10. Participation in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to the Screening Visit. 11. ALT or AST level > 5x ULN, or total bilirubin level > 1.5x ULN at the Screening Visit or at Baseline (except for patients with Gilbert Syndrome). 12. Signs and/or symptoms of liver cirrhosis or portal hypertension (e.g., splenomegaly, ascites, esophageal varices), or documented liver disease unrelated to CF‡. 13. DIOS in the last six months prior to the Screening Visit. 14. Unable to discontinue the use of pancreatic enzymes. 15. 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. 16. Patient is unlikely to complete the study, as determined by the Investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the efficacy of ALTU-135 for the treatment of fat malabsorption in patients with CF-related exocrine PI. Primary: • Mean change in CFA from Baseline Period to Double-Blind Treatment Period. Secondary: • Mean change in CNA from Baseline Period to Double-Blind Treatment Period. • Proportion of patients who have a maximum increase in blood glucose of = 10 mg/dl in the SCT (non-diabetic patients only) during the Double-Blind Treatment Period. Mean changes in stool weight and frequency from Baseline Period to Double-Blind Treatment Period. Tertiary: • Mean difference in maximum change in blood glucose level following starch challenge from Baseline Period to Double-Blind Treatment Period. • Mean percent change in CFA and CNA from Baseline Period to Double-Blind Treatment Period. • Mean relative improvement in CFA and CNA from Baseline Period to Double-Blind Treatment Period. ;Secondary Objective: • To determine the efficacy of ALTU-135 for the treatment of protein malabsorption in patients with CF-related exocrine PI. • To determine the efficacy of ALTU-135 treatment in improving the absorption of carbohydrates in patients with CF-related exocrine PI. • To determine the efficacy of ALTU-135 treatment in decreasing stool weight and frequency in patients with CF-related exocrine PI. Safety Endpoints: • Safety endpoints include frequency and severity of treatment-emergent AEs; mean changes in clinical laboratory parameters and vital signs; toxicity shifts in LFT parameters; proportion of patients with abnormal clinical laboratory and vital signs results. ;Primary end point(s): Mean change in CFA from Baseline Period to Double-Blind Treatment Period. to determine the efficacy of ALTU-135 for the treatment of fat malabsorption in patients with CF-related exocrine PI. | — |
Countries
Italy, Poland, Slovakia
Outcome results
None listed