Back to resultsPR-018: An Open-Label, Safety Extension of Study PR-011
Primary Purpose
Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Status
Completed
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
EUR-1008 (APT-1008)
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis focused on measuring Exocrine Pancreatic Insufficiency, Infants with CF
Eligibility Criteria
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
Sites / Locations
- Nemours Children's Clinic
- Children's Lung Specialists Ltd.
- Akron Children's Hospital
- Cincinnati Children's Hospital Medical Center
- Penn State Milton S. Hershey Medical Center
- Cystic Fibrosis Care Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
EUR-1008 (APT-1008)
Arm Description
Outcomes
Primary Outcome Measures
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
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
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01131507
Brief Title
PR-018: An Open-Label, Safety Extension of Study PR-011
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)
Study Type
Interventional
2. Study Status
Record Verification Date
February 2017
Overall Recruitment Status
Completed
Study Start Date
July 2010 (undefined)
Primary Completion Date
December 2011 (Actual)
Study Completion Date
December 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Forest Laboratories
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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).
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Keywords
Exocrine Pancreatic Insufficiency, Infants with CF
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)
8. Arms, Groups, and Interventions
Arm Title
EUR-1008 (APT-1008)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
EUR-1008 (APT-1008)
Other Intervention Name(s)
Zenpep® (pancrelipase) 3,000 lipase units delayed release capsules
Intervention Description
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.
Primary Outcome Measure Information:
Title
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Description
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.
Time Frame
Up to Month 12 or early termination
Secondary Outcome Measure Information:
Title
Change From Baseline in Growth Percentiles at Month 3, 6, 9 and 12
Description
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.
Time Frame
Baseline, Month 3, 6, 9 and 12
10. Eligibility
Sex
All
Maximum Age & Unit of Time
12 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Aptalis Medical Information
Organizational Affiliation
Forest Laboratories
Official's Role
Study Director
Facility Information:
Facility Name
Nemours Children's Clinic
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32250
Country
United States
Facility Name
Children's Lung Specialists Ltd.
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89107
Country
United States
Facility Name
Akron Children's Hospital
City
Akron
State/Province
Ohio
ZIP/Postal Code
44308
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Penn State Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Cystic Fibrosis Care Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
12. IPD Sharing Statement
Learn more about this trial
PR-018: An Open-Label, Safety Extension of Study PR-011
We'll reach out to this number within 24 hrs