Phase 3
N=33
Study to Evaluate the Safety and Efficacy of EUR-1008 (APT-1008) Pancreatic Enzyme Product in Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency
Cystic Fibrosis · Exocrine Pancreatic Insufficiency
Bottom Line
View on ClinicalTrials.gov: NCT00297167 ↗Enrolled (actual)
33
Serious AEs
3.0%
Results posted
Apr 2014
Primary outcome: Primary: Percent Coefficient of Fat Absorption (CFA%) — 88.28; 62.76 percent CFA — p=<0.001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- EUR-1008 (APT-1008) (Drug); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 7+ yrs
- Sex
- All
- Sponsor
- Forest Laboratories
- Primary completion
- Nov 2006
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Coefficient of Fat Absorption (CFA%) |
88.28; 62.76 | <0.001 sig |
| SECONDARY Percent Coefficient of Nitrogen Absorption (CNA%) |
87.17; 65.67 | <0.001 sig |
| SECONDARY Lipid Levels |
128.8; 109.1; 45.5; 37.2 | — |
| SECONDARY Vitamin A Levels |
422.3; 363.2 | — |
| SECONDARY Vitamin E Levels |
8.25; 6.69 | — |
| SECONDARY Mean Daily Number of Stools |
1.76; 2.66 | — |
| SECONDARY Percentage of Stool Categorized as Per Consistency |
16.54; 6.24; 53.86; 33.25; 29.24; 57.11 | — |
| SECONDARY Mean Number of Abdominal Symptoms |
0.10; 0.31; 0.05; 0.10; 0.00; 0.09 | — |
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).
Eligibility 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 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
Data sourced from ClinicalTrials.gov (NCT00297167). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.