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

• Status: Completed
• Trial end date: 2006-11-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Forest Laboratories

Criteria

Inclusion Criteria:

• Participants with age greater than or equal to (>=) 7 years at the time of enrollment

• Participants with weight 70 kg or less and be in an adequate nutritional status as indicated by a body mass index (BMI) >=20 kg/m^2 for ages 18 and above, or a BMI above the twenty fifth percentile for participants aged 7 to 17 years

• Participants with confirmed diagnosis of CF who have 2 clinical features consistent with CF, and have either a genotype with 2 identifiable mutations consistent with CF or a sweat chloride concentration that is more than 60 milliequivalent per liter by quantitative pilocarpine iontophoresis

• Participants with confirmed diagnosis of EPI who are currently receiving treatment with a commercially available PEP and have documented with a fecal elastase of <100 microgram per gram stool (if no documentation was available, a stool sample was taken at Screening for determination of fecal elastase).

• Clinically stable participants with no evidence of acute respiratory disease or any other acute condition

• Participants who are willing and able to interrupt current CF treatment for CF-related malabsorption along with any medications that may affect gastric motility or stomach power of hydrogen (pH)

• Participants 18 years of age and older had to a) understand the requirements of the study, b) provide written informed consent, c) agree to abide by the study restrictions, and d) return for the required assessments

• Participants 7 to 17 years of age must have a parent(s) or legal guardian who provides written informed consent, agree to abide by the study restrictions

• Females participants of childbearing potential must have a negative serum pregnancy test at screening and must agree to use adequate birth control during the study

Exclusion Criteria:

• Participants with fibrosing colonopathy, hyperuricemia or hyperuricosuria

• Participants who are allergic to pork or other porcine PEPs

• Participants with forced expiratory volume (FEV1) <30 percent of predicted FEV1 at screening

• Participants with any acute systemic administration of an antibiotic for any reason in the previous 4 weeks; however, a low stable dose of an antibiotic or chronic treatment with an inhalatory antibiotic is allowed

• Participants with hepatic insufficiency as defined by history or presence of ascites or serum albumin level of < 3.0 milligram/deciliter, or a coagulopathy with an international normalized ratio that is greater than 1.7

• Participants who have used an acute dose of any steroid in the previous 2 weeks; however, low chronic doses of a steroid is allowed

• Participants with history of or current diagnosis of distal ileal obstruction syndrome (DIOS) as evidenced or suggested by constipation, abdominal pain, anorexia, early satiety, recurrent vomiting, and palpable fecal mass on physical examination (the absence of DIOS will be confirmed by an X-ray of the abdomen taken at screening)

• Participants with any solid organ transplant or surgery affecting the bowel. Participants with a history of appendectomy and inguinal (non-incarcerated) hernioplasty or meconium ileus without the need for bowel resection could be enrolled. Gastrointestinal-tube-fed patients, in absence of dumping syndrome, were also eligible

• Participants with history of or current screening evaluation of hyperglycemia as defined by an 8-hour fasting blood glucose (FBG) of >126 mg/dL, or of CF-related diabetes as determined according to the Cystic Fibrosis Foundation (CFF) Consensus Conference of January 1999 (Section IX Part II), that is: a) FBG >126 mg/dL (7.0 millimoles per liter [mmol/L]) on two or more occasions b)FBG >126 mg/dL (7.0 mmol/L) plus casual (without regard to time of day or last meal consumed) glucose level >200 mg/dL (11.1 mmol/L) c)Casual (previously called random) glucose levels >200 mg/dL (11.1 mmol/L) on two or more occasions with symptoms

• Participants using an enzyme preparation in excess of 10,000-lipase units/kg/day

• Participants using an immunosuppressive drug

• Participants who are expecting an inability to tolerate the washout period and/or the placebo treatment

• Participants participating in an investigational study of a drug, biologic, or device not currently approved for marketing, within 30 days of screening visit

• Female participants who are pregnant or breastfeeding, or unwilling to use effective birth control during study

• Participants with any condition that would, in the investigator's opinion, limit the participant's ability to complete the study