Clinical Trial: Study to Evaluate the Safety and Efficacy of EUR-1008 (APT-1008) Pancreatic Enzyme Product in Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Randomized, Double-Blind, Placebo-Controlled, Two-Treatment, Crossover Study to Evaluate the Safety and Efficacy of Eurand Pancreatic Enzyme Product (PEP) in Patients With Cystic Fibrosis and

Brief Summary: The primary efficacy objective of this study is to compare the coefficient of fat absorption (CFA) following oral administration of Aptalis Pharma's (formerly Eurand Pharmaceuticals) pancreatic enzyme product (PEP) capsules and placebo in participants with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI).

Detailed Summary: This is a randomized, double-blind, placebo-controlled, 2-treatment, crossover, multicenter trial in participants with CF and EPI. The study consists of a screening period (1 to 14 days), a washout period (2 days), a dose titration/stabilization period (6 to 9 days), a blinded randomized treatment period (6 to 7 days), an open-label normalization period 1 (5 to 14 days), a blinded crossover treatment period (6 to 7 days), followed by an open-label normalization period 2 (7 days). The order of treatments (placebo followed by EUR-1008 [APT-1008] or EUR-1008 [APT-1008] followed by placebo) will be determined by randomization at the beginning of randomization treatment period only and will be carried through the crossover treatment period. The starting dose will be 1,000 lipase units per kilogram per meal (lipase units/kg/meal), which will be titrated to control symptoms of EPI, with the total dose not exceeding 10,000 lipase units/kg/day.
Sponsor: Forest Laboratories

Current Primary Outcome: Percent Coefficient of Fat Absorption (CFA%) [ Time Frame: Day 3 up to Day 6 of hospital treatment in first and second double-blind intervention periods ]

Percent CFA was calculated as ([fat intake - fat excretion]/fat intake)multiplied by 100, determined in the stools collected during the 72-hour hospitalization period. Mean percent CFA was calculated for Day 3 to Day 6 during hospital treatment in first and second double-blind (DB) intervention periods.


Original Primary Outcome: Compare the coefficient of fat absorption (CFA) following oral administration of Eurand PEP capsules or placebo; safety objectives will concentrate on the frequency, duration, and severity of treatment emergent, adverse events (AEs) and

Current Secondary Outcome:

  • Percent Coefficient of Nitrogen Absorption (CNA%) [ Time Frame: Day 3 up to Day 6 of hospital treatment in first and second double-blind intervention periods ]
    Percent CNA was calculated as ([nitrogen intake-nitrogen excretion]/nitrogen intake)*100, determined in the stools collected during the 72-hour hospitalization period. Nitrogen intake was calculated as protein intake/6.2. Nitrogen excretion was measured as total fecal nitrogen. Mean percent CNA was calculated for Day 3 to Day 6 during hospital treatment in first and second double-blind intervention periods.
  • Lipid Levels [ Time Frame: End of treatment (Day 6 during first and second double-blind intervention periods) ]
    Lipid levels were reported for total cholesterol (TC) and high-density lipoprotein cholesterol (HDL-C) from fasted blood and urine samples. Mean lipid levels for Day 6 during first and second double-blind intervention periods were calculated.
  • Vitamin A Levels [ Time Frame: End of treatment (Day 6 during first and second double-blind intervention periods) ]
    Mean Vitamin A levels for Day 6 during first and second double-blind intervention periods were calculated.
  • Vitamin E Levels [ Time Frame: End of treatment (Day 6 during first and second double-blind intervention periods) ]
    Mean Vitamin E levels for Day 6 during first and second double-blind intervention periods were calculated.
  • Mean Daily Number of Stools [ Time Frame: Day 3 up to Day 6 during first and second double-blind intervention periods ]
    Mean daily number of stools of each participant was calculated from frequency of stools by the participant per day. Mean daily number of stools during the collection period (Day 3 to Day 6 in first and second double-blind intervention periods) for total participants was summarized.
  • Percentage of Stool Categorized as Per Consistency [ Time Frame: Day 3 up to Day 6 during first and second double-blind intervention periods ]
    Stool consistency was categorized as hard, formed/normal, soft, watery, or overt diarrhea. Percentage of stools of a specific consistency for each participant at first and second double-blind intervention periods was calculated. Mean percentage of stool consistency during the collection period (Day 3 to Day 6 in first and second intervention periods) for total participants was summarized.
  • Mean Number of Abdominal Symptoms [ Time Frame: Day 3 up to Day 6 during first and second double-blind intervention periods ]
    Abdominal symptoms included abdominal pain, flatulence and bloating. Symptoms were classified by severity as mild (no impairment of daily activities), moderate (slight impairment of daily activities), or severe (unable to perform daily activities). Mean number of symptom of specific severity per day for each participant was calculated. Mean number of symptoms per day was calculated for Day 3 to Day 6 in first and second double-blind intervention periods for total participants.


Original Secondary Outcome: Compare changes in the coefficient of nitrogen absorption (CNA), cholesterol, fat-soluble vitamins, weight, body mass index (BMI), and symptoms of EPI after the oral administration of Eurand PEP capsules and placebo.

Information By: Forest Laboratories

Dates:
Date Received: February 27, 2006
Date Started: May 2006
Date Completion:
Last Updated: February 7, 2017
Last Verified: February 2017