Clinical trial NCT01100606: A Study to Evaluate the Mode of Administration and Safety of EUR-1008 (APT-1008) in Infants 1 to 12 Months of Age
Official title: A Multicenter, Randomized, Open-Label, Crossover Study to Evaluate the Mode of Administration and Safety of EUR-1008 in Infants 1 to 12 Months of Age With Exocrine Pancreatic Insufficiency (EPI) Associated With Cystic Fibrosis (CF)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Cystic Fibrosis
Exocrine Pancreatic Insufficiency
Keywords: Exocrine Pancreatic Insufficiency
Infants with CF
Lead sponsor: Forest Laboratories
Sponsor class: INDUSTRY
First posted: 2010-04-09
Enrollment: 15
Sex: ALL
Minimum age (years): 1
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief 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).

Detailed description: This is a multicenter, randomized, open-label, crossover study in pediatric participants with EPI due to CF. The study will be carried out in infants between 1 and 12 months of age.

The study comprises of a screening period (up to 10 days) followed by 2 treatment periods (10 days each). During the screening period, all participants will be administered Zenpep® 5,000 (pancrelipase) mixed with a small amount of apple sauce. Once determined eligible for participation, participants will be randomized into 1 of 2 treatment sequences. Each sequence corresponds to taking one treatment in the first period and the other treatment in the second period, and were administered EUR-1008 (APT-1008) (Zenpep® \[pancrelipase\] delayed release capsule) 3,000 lipase units capsule either mixed with apple juice using a syringe nurser or apple sauce using a spoon.

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

Primary outcomes:
measure: Treatment Difference for Acceptability of Treatment
timeFrame: Baseline up to end of study (Day 21)
measure: Question 6 (Previous Pancreatic Enzyme Product [PEP])
timeFrame: Baseline
Secondary outcomes:
measure: Daily Number of Stools
timeFrame: Up to Day 10 in first and second treatment periods, end of study (Day 21)
measure: Number of Stools Categorized as Per Consistency
timeFrame: Up to Day 10 in first and second treatment periods, end of study (Day 21)
measure: Number of Stools With Signs of Blood and Visible Oil or Grease
timeFrame: Up to Day 10 in first and second treatment periods, end of study (Day 21)
measure: Number of Abdominal Symptoms: Bloating
timeFrame: Up to Day 10 in first and second treatment periods, end of study (Day 21)
measure: Number of Abdominal Symptoms: Flatulence
timeFrame: Up to Day 10 in first and second treatment periods, end of study (Day 21)
measure: Number of Abdominal Pain Symptoms
timeFrame: Up to Day 10 in first and second treatment periods, end of study (Day 21)
measure: Number of Participants With Abnormal Clinical Laboratory and Vital Signs Findings
timeFrame: Baseline up to end of study (Day 21)
measure: Number of Participants With Abnormal Findings With Respect to Oral Mucosa
timeFrame: Baseline up to end of study (Day 21)
Other outcomes:
Locations:
city: Jacksonville
country: United States
facility: Nemours Children's Clinic
state: Florida
city: Las Vegas
country: United States
facility: Children's Lung Specialists Ltd.
state: Nevada
city: Akron
country: United States
facility: Akron Children's Hospital
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Hershey
country: United States
facility: Penn State Milton S. Hershey Medical Center
state: Pennsylvania
city: Houston
country: United States
facility: Cystic Fibrosis Care Center
state: Texas
Study officials:
affiliation: Forest Laboratories
name: Aptalis Medical Information
role: STUDY_DIRECTOR
References: