Clinical Trial: Study of Pancreatic Enzyme Product in Pediatric Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency

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

Official Title: An Open-Label Study to Evaluate the Efficacy and Safety of Pancreatic Enzyme Product (PEP) Microtabs in Pediatric Patients With Cystic Fibrosis and Exocrine Pancreatic Insufficie

Brief Summary: This is an open-label study to evaluate the efficacy and safety of Aptalis' (formerly Eurand) pancreatic enzyme product (PEP) microtabs in pediatric participants under age 7 with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI).

Detailed Summary:

The study sample will consist of evaluable participants, all of whom will be children younger than 7 years of age. Participants will receive EUR-1008 (APT-1008) Microtabs formulation. The study design involves a 4-day screening period, a 7-day dose stabilization period, and a 7-day treatment period (excluding an end-of-study evaluation).

The optimal dose of EUR-1008 (APT-1008) Microtabs, determined during the dose stabilization period, will be used during the treatment period. Participants are instructed to consume a predefined diet.

Current Primary Outcome:

• Percentage of Participants Who Were Responders After 1 Week of Treatment With Study Medication [ Time Frame: Day 11 ]
  Responders were defined as those participants without steatorrhea (defined as less than 30 percent (%) fecal fat content) and without signs and symptoms of malabsorption after 1 week of treatment with study medication.
• Percentage of Participants Who Were Responders After 2 Weeks of Treatment With Study Medication [ Time Frame: Day 18 (end of treatment) ]
  Responders were defined as those participants without steatorrhea (defined as less than 30% fecal fat content) and without signs and symptoms of malabsorption after 2 weeks of treatment with study medication.

Original Primary Outcome: Percent responders without steatorrhea & without malabsorption signs & symptoms after 1 & 2 weeks of treatment. Lack of steatorrhea (defined as <30% fecal fat content) assessed from the fecal fat content readings on Days 11 & 18 compared to baseline. [ Time Frame: 29 days ]

Current Secondary Outcome:

• Change From Baseline in Weight at Day 12, 19 [ Time Frame: Baseline, Day 12, 19 ]
• Mean Daily Number of Stools [ Time Frame: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period) ]
  Mean daily number of stools of each participant was calculated from frequency of stools by the participant per day. Mean daily number of stools at each period for total participants was summarized.
• Percentage of Stool Categorized by Consistency [ Time Frame: Baseline, Day 5 up to Day 11 (dose stabilization period) and Day 12 up to Day 18 (treatment period) ]
  Stool consistency was categorized as hard, formed/normal, soft, watery or overt diarrhea. Percentage of stools of a specific consistency of each participant was calculated as the number of stools with a specific consistency relative to the total number of stools during the collection period. Mean percentage of stool with specific consistency at each period for total participants was summarized.
• Mean Number of Abdominal Symptoms: Bloating [ Time Frame: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period) ]
  Bloating is swelling of the intestinal tract caused by excessive gas formation. Symptoms of bloating 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 symptoms of specific severity for each participant was calculated from frequency of symptoms by the participant per day. Mean number of symptoms at each period for total participants was summarized.
• Mean Number of Abdominal Symptoms: Flatulence [ Time Frame: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period) ]
  Flatulence is presence of excessive gas in the digestive tract. Symptoms of flatulence was 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 symptoms of specific severity for each participant was calculated from frequency of symptoms by the participant per day. Mean number of symptoms at each period for total participants was summarized.
• Mean Number of Pain Symptoms [ Time Frame: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period) ]
  Symptoms of pain was 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 symptoms of specific severity for each participant was calculated from frequency of symptoms by the participant per day. Mean number of symptoms at each period for total participants was summarized.
• Physician's and Parent's or Legal Guardians Assessment of Improvement in Clinical Symptoms [ Time Frame: Day 19 (end of study) ]
  Clinical symptoms of exocrine pancreatic insufficiency (EPI) were assessed by the physician and parent or guardian to determine if the participant showed improvement in symptoms of EPI at end of study after the dose stabilization period. EPI is a syndrome characterized by clinical symptoms of poor absorption of fats, proteins, and to a lesser extent, carbohydrates, which manifests primarily in patients with cystic fibrosis. Number of participants with improvement in clinical symptoms was reported.
• Percentage of Blood in Stool [ Time Frame: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period) ]
  Mean percentage of stools with blood at each period for total participants was summarized.
• Percentage of Stool With Visible Oil or Grease [ Time Frame: Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period) ]
  Mean percentage of oil or grease at each period for total participants was summarized.

Original Secondary Outcome:

• Weight change, nutritional status, stool frequency and consistency, and incidences of bloating, pain, and flatus. [ Time Frame: 29 days ]
• Physician's and parent's or legal guardian's judgment of improvement of clinical symptoms. [ Time Frame: 29 days ]

Dates:
Date Received: September 21, 2009
Date Started: May 2006
Date Completion:
Last Updated: February 8, 2017
Last Verified: February 2017