PR-018: An Open-Label, Safety Extension of Study PR-011

This study has been completed.
Information provided by (Responsible Party):
Aptalis Pharma Identifier:
First received: May 25, 2010
Last updated: January 27, 2014
Last verified: January 2014

A study to evaluate long term safety and effect on ability to thrive of EUR-1008 (APT-1008) 3,000 lipase units (Zenpep® [pancrelipase] delayed release capsules) in infants with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF).

Condition Intervention Phase
Cystic Fibrosis
Exocrine Pancreatic Insufficiency
Drug: EUR-1008 (APT-1008)
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multicenter, Open-Label, Safety Extension of Study PR-011 Titled: 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)

Resource links provided by NLM:

Further study details as provided by Aptalis Pharma:

Primary Outcome Measures:
  • Frequency and Severity of Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Up to Month 12 or early termination ] [ Designated as safety issue: Yes ]
    TEAE was any event not present prior to exposure to study drug or any event already present that worsened in either intensity or frequency following exposure to test drug. Serious AE (SAE) was any event that resulted in death, immediately life threatening, hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or a congenital anomaly/birth defect. Number of participants with TEAEs, SAEs, TEAE's relationship to study drug (unrelated, possible and probable) and on the basis of severity (mild [minimal/no treatment and did not interfere with daily activities], moderate [resulted in a low level of inconvenience or concern with the therapeutic measures and may have caused some interference with functioning] and severe [interrupted participant's usual daily activity, may have required systemic drug therapy or other treatment and were usually incapacitating]) with a frequency threshold of above 5% were reported.

Secondary Outcome Measures:
  • Change From Baseline in Growth Percentiles at Month 3, 6, 9 and 12 [ Time Frame: Baseline, Month 3, 6, 9 and 12 ] [ Designated as safety issue: No ]
    Participant's ability to thrive was evaluated through growth percentiles for weight-for-age, length-for-age and weight-for-length recorded on Center for Disease Control and Prevention (CDC) growth charts during each treatment visit.

Enrollment: 15
Study Start Date: July 2010
Study Completion Date: December 2011
Primary Completion Date: December 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: EUR-1008 (APT-1008) Drug: EUR-1008 (APT-1008)
EUR-1008 (APT-1008) (Zenpep® [pancrelipase] 3,000 lipase units delayed release capsules) from open capsules mixed with a small amount of apple juice or apple sauce will be administered orally starting at the same dose as administered at the end of study PR-011 (NCT01100606), with dose increments of 3,000 lipase units. The dose will be adjusted based on participants' age and body weight. Total dose will not exceed 10,000 lipase units per kilogram (kg) of body weight per day unless clinically indicated. Total duration of study treatment will be up to 12 months.
Other Name: Zenpep® (pancrelipase) 3,000 lipase units delayed release capsules

Detailed Description:

This is a multi-center, open-label, safety extension of Aptalis (formerly Eurand) study PR-011 (NCT01100606) in pediatric participants with EPI due to CF. The study will be carried out in participants who completed the PR-011 study.

The study is comprised of 5 visits: an enrollment visit, treatment visit 1 (3 months), treatment visit 2 (6 months), treatment visit 3 (9 months) and treatment visit 4 (12 months). Once determined eligible for participation, participants will be enrolled into the study.


Ages Eligible for Study:   up to 12 Months
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Participant's parent or guardian signed informed consent form (ICF)
  • Participants who have completed study PR-011 (NCT01100606)

Exclusion Criteria:

  • Participant having any condition that would, in the investigator's opinion, limit the participant's ability to complete the study or will result in excess risk to the participant that is above the standard of care
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01131507

United States, Florida
Nemours Children's Clinic
Jacksonville, Florida, United States, 32250
United States, Nevada
Children's Lung Specialists Ltd.
Las Vegas, Nevada, United States, 89107
United States, Ohio
Akron Children's Hospital
Akron, Ohio, United States, 44308
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Pennsylvania
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
United States, Texas
Cystic Fibrosis Care Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
Aptalis Pharma
Study Director: Aptalis Medical Information Aptalis Pharma
  More Information

No publications provided

Responsible Party: Aptalis Pharma Identifier: NCT01131507     History of Changes
Other Study ID Numbers: PR-018
Study First Received: May 25, 2010
Results First Received: January 27, 2014
Last Updated: January 27, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Aptalis Pharma:
Exocrine Pancreatic Insufficiency
Infants with CF

Additional relevant MeSH terms:
Cystic Fibrosis
Exocrine Pancreatic Insufficiency
Digestive System Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Lung Diseases
Pancreatic Diseases
Pathologic Processes
Respiratory Tract Diseases
Gastrointestinal Agents
Pharmacologic Actions
Therapeutic Uses processed this record on October 20, 2014