A Study to Evaluate the Mode of Administration and Safety of EUR-1008 (APT-1008) in Infants 1 to 12 Months of Age

NCT01100606 | Completed Phase 4 Results DMC

• 1 other identifier: PR-011
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 6

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).

Conditions

Cystic Fibrosis; Exocrine Pancreatic Insufficiency

Keywords

Exocrine Pancreatic Insufficiency; Infants with CF

Outcome Measures

Primary Outcomes (2)

• Treatment Difference for Acceptability of Treatment

  Acceptability questionnaire consists of 9 question (Q) to assess ease, time, overall satisfaction of study drug. Rated on 5-point scale for Q1-Q5 and Q7-Q9; Q6 was not rated and asked for name of previous PEP administered. Q1=overall ease of administration (1=not at all easy,2=somewhat,3=easy,4=very,5=extremely); Q2=time of administration (1=very short\[\<2 min\],2=short\[2-5 min\],3=moderate\[5-15 min\],4=long\[15-25 min\],5=very long\[\>25 min\]);Q3=overall infant acceptance(1=very easily,2=easily,3=same,4=with difficulty,5=with great difficulty);Q4=clear/complete instructions(1=not clear,2=somewhat,3=clear,4=very,5=extremely);Q5=overall satisfaction with dosing method (1=not satisfied,2=somewhat,3=satisfied,4=very,5=extremely);Q7=comparative ease of administration (1=much worse,2=worse,3=same,4=better,5=much better);Q8=comparative infant acceptance (1=much more difficult,2=more,3=same,4=easier,5=much easier);Q9=comparative overall satisfaction (1=much less,2=less,3=same,4=more,5=much more).

  Baseline up to end of study (Day 21)

• Question 6 (Previous Pancreatic Enzyme Product [PEP])

  Acceptability questionnaire consists of 9 questions (Q) to assess the ease, time, overall satisfaction of study drug. Q6 included "name of previous PEP administered". Q6 was reported as number of participants who used any PEP prior to screening.

  Baseline

Secondary Outcomes (8)

• Daily Number of Stools

  Up to Day 10 in first and second treatment periods, end of study (Day 21)

• Number of Stools Categorized as Per Consistency

  Up to Day 10 in first and second treatment periods, end of study (Day 21)

• Number of Stools With Signs of Blood and Visible Oil or Grease

  Up to Day 10 in first and second treatment periods, end of study (Day 21)

• Number of Abdominal Symptoms: Bloating

  Up to Day 10 in first and second treatment periods, end of study (Day 21)

• Number of Abdominal Symptoms: Flatulence

  Up to Day 10 in first and second treatment periods, end of study (Day 21)

Study Arms (2)

EUR-1008 (APT-1008) in Apple Juice

EXPERIMENTAL

Drug: EUR-1008 (APT-1008)

EUR-1008 (APT-1008) in Apple Sauce

EXPERIMENTAL

Drug: EUR-1008 (APT-1008)

Interventions

EUR-1008 (APT-1008) DRUG

EUR-1008 (APT-1008) (Zenpep® \[pancrelipase\] delayed release capsule) from open capsule, mixed with a small amount of apple juice, will be given orally daily using a syringe nurser at dose increments of 3,000 lipase units, for 10 days in either first treatment period or second treatment period. Total dose will not exceed 10,000 lipase units per kilogram (kg) of body weight per day.

Also known as: Zenpep® (pancrelipase) delayed release capsules

EUR-1008 (APT-1008) in Apple Juice

Eligibility Criteria

• Age: 1 Month - 12 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Forest Laboratories: lead

Study Sites (6)

Nemours Children's Clinic

Jacksonville, Florida, 32250, United States

Location

Children's Lung Specialists Ltd.

Las Vegas, Nevada, 89107, United States

Location

Akron Children's Hospital

Akron, Ohio, 44308, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Cystic Fibrosis Care Center

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis; Exocrine Pancreatic Insufficiency

Interventions

Pancrelipase

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Lipase; Carboxylic Ester Hydrolases; Esterases; Hydrolases; Enzymes; Enzymes and Coenzymes; Pancreatic Extracts; Tissue Extracts; Complex Mixtures

Results Point of Contact

• Title: Robert Winkler, MD, VP, Clinical Development and Operations
• Organization: Aptalis Pharma US, Inc.

1-800-472-2634

Study Officials

• Aptalis Medical Information

  Forest Laboratories

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 31, 2010
• First Posted: April 9, 2010
• Study Start: June 1, 2010
• Primary Completion: December 1, 2010
• Study Completion: December 1, 2010
• Last Updated: April 10, 2014
• Results First Posted: April 10, 2014

Record last verified: 2014-03

Locations

US (6)