Phase 4
N=15
A Study to Evaluate the Mode of Administration and Safety of EUR-1008 (APT-1008) in Infants 1 to 12 Months of Age
Cystic Fibrosis · Exocrine Pancreatic Insufficiency
Bottom Line
View on ClinicalTrials.gov: NCT01100606 ↗Enrolled (actual)
15
Serious AEs
0.0%
Results posted
Apr 2014
Primary outcome: Primary: Treatment Difference for Acceptability of Treatment — 1.5; 3.5; 2.5; 1.0 units on a scale
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- EUR-1008 (APT-1008) (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Forest Laboratories
- Primary completion
- Dec 2010
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Treatment Difference for Acceptability of Treatment |
1.5; 3.5; 2.5; 1.0; 3.5; 1.5 | — |
| PRIMARY Question 6 (Previous Pancreatic Enzyme Product [PEP]) |
4; 9; 1; 1 | — |
| SECONDARY Daily Number of Stools |
2.76; 2.44 | — |
| SECONDARY Number of Stools Categorized as Per Consistency |
0.04; 0.14; 1.77; 1.37; 0.62; 0.83 | — |
| SECONDARY Number of Stools With Signs of Blood and Visible Oil or Grease |
0; 0.01; 0.09; 0.17 | — |
| SECONDARY Number of Abdominal Symptoms: Bloating |
0.22; 0.26; 0; 0; 0; 0 | — |
| SECONDARY Number of Abdominal Symptoms: Flatulence |
1.0; 0.62; 0.31; 0.32; 0.04; 0 | — |
| SECONDARY Number of Abdominal Pain Symptoms |
0.10; 0.13; 0.09; 0.07; 0; 0 | — |
| SECONDARY Number of Participants With Abnormal Clinical Laboratory and Vital Signs Findings |
— | — |
| SECONDARY Number of Participants With Abnormal Findings With Respect to Oral Mucosa |
0; 0 | — |
Summary
A study to determine the safety, effectiveness, and acceptability of 2 methods of administration of EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) 3,000 lipase units capsule, a pancreatic enzyme product (PEP), in infants with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). This study is sponsored by Aptalis Pharma (formerly Eurand).
Eligibility Criteria
Inclusion Criteria
- Participants with diagnosis of CF based on the following criteria: one clinical feature consistent with CF, and either a genotype with 2 identifiable mutations known to cause CF or a sweat chloride concentration that is greater than 60 milliequivalent per liter by quantitative pilocarpine iontophoresis
- Participants who have the need for a PEP defined as monoclonal fecal elastase less than 200 microgram per gram (mcg/g) stool
- Caregiver must be willing to switch participant from their previous PEP (if any) to Zenpep®
- Participants who have a height to weight ratio target at greater than tenth percentile
- Participants who are clinically stable with no evidence of concomitant illness or acute upper or lower respiratory tract infection during the 7-day interval prior to screening and preceding accession into this clinical study
Exclusion Criteria
- Participants who are less than 1 month old or are greater than 12 months old
- Participants with history of meconium ileus or small bowel atresia in the newborn period that required surgery
- Participants who are allergic to pork or other porcine PEPs
- Participants with any respiratory condition or other serious comorbidity (for example patent ductus arteriosus [PDA], or necrotizing enterocolitis [NEC]) that in the investigator's opinion would result in an intervention requiring hospitalization or intensive pulmonary or other treatment during the trial
- Participants with other comorbidities independent of CF that, in the investigator's opinion, would result in an inability to participate in the study or excess risk to the participant that is above the standard of care
- Participants with acute respiratory infection in the previous 14 days requiring antibiotics
- Participants who required change in antacid dose in the 7 days before screening
- Participants with administration of oral, intramuscular (IM), intravenous (IV) glucocorticoids in the 4 weeks prior to screening
- Participants with any condition that would, in the investigator's opinion, limit the participant's ability to complete the study
- Participants currently participating in or has participated in an investigational study, with the exception of observational studies, within 30 days of the screening visit
Data sourced from ClinicalTrials.gov (NCT01100606). 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.