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

Forest Laboratories15 enrolled

Overview

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).

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Cystic Fibrosis Exocrine Pancreatic Insufficiency

Interventions

EUR-1008 (APT-1008)DRUG

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.

Eligibility

Sex: ALLMax age: 12 Months

Medical Language ↔ Plain English

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

Locations (6)

Nemours Children's Clinic

Jacksonville, Florida, United States

Children's Lung Specialists Ltd.

Las Vegas, Nevada, United States

Akron Children's Hospital

Akron, Ohio, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Outcomes

Primary Outcomes

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.

Time frame: Up to Month 12 or early termination

Secondary Outcomes

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.

Time frame: Baseline, Month 3, 6, 9 and 12

Data from ClinicalTrials.gov

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