Study of Pancreatic Enzyme Product in Pediatric Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency

NCT ID: NCT00981214

Last Updated: 2017-03-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 19 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2006-05-31
• Study Completion Date: 2006-09-30

Brief Summary

This is an open-label study to evaluate the efficacy and safety of Aptalis' (formerly Eurand) pancreatic enzyme product (PEP) microtabs in pediatric participants under age 7 with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI).

Detailed Description

The study sample will consist of evaluable participants, all of whom will be children younger than 7 years of age. Participants will receive EUR-1008 (APT-1008) Microtabs formulation. The study design involves a 4-day screening period, a 7-day dose stabilization period, and a 7-day treatment period (excluding an end-of-study evaluation).

The optimal dose of EUR-1008 (APT-1008) Microtabs, determined during the dose stabilization period, will be used during the treatment period. Participants are instructed to consume a predefined diet.

Conditions

Cystic Fibrosis; Exocrine Pancreatic Insufficiency

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

EUR-1008 (APT-1008)

Group Type: EXPERIMENTAL

EUR-1008 (APT-1008)

Intervention Type: DRUG

EUR-1008 (APT-1008) Microtabs contained in a capsule will be administered orally from Day 5 to Day 11 at an enzyme dose based on investigator's discretion, in dose stabilization period or the content of the capsule will be allowed to sprinkle on food, where necessary, followed by stabilized dose from Day 12 to Day 18 in treatment period, up to a maximum total dose of 10,000 lipase units per kilogram body weight per day (unit/kg/day).

Interventions

EUR-1008 (APT-1008)

EUR-1008 (APT-1008) Microtabs contained in a capsule will be administered orally from Day 5 to Day 11 at an enzyme dose based on investigator's discretion, in dose stabilization period or the content of the capsule will be allowed to sprinkle on food, where necessary, followed by stabilized dose from Day 12 to Day 18 in treatment period, up to a maximum total dose of 10,000 lipase units per kilogram body weight per day (unit/kg/day).

Intervention Type: DRUG

Other Intervention Names

ZENPEP®; Pancrelipase

Eligibility Criteria

Inclusion Criteria

• Participants less than 7 years of age
• Participants who have pancreatic insufficiency documented by a fecal elastase level less than 100 micrograms per gram (mcg/g), or if not documented, the fecal elastase test must be done at the screening visit
• Participants who have a need of de novo treatment with pancreatic enzymes or be able to be switched from an existing treatment
• Participants who have a body mass index greater than the twenty fifth percentile for children 2 years and older
• Participants with a weight for height index greater than the twenty fifth percentile for children less than 2 years of age
• Participants with diagnosis of CF based upon the following criteria:

• Have 2 clinical features consistent with CF and
• Have either a genotype with 2 identifiable mutations consistent with CF or a sweat chloride concentration that is more than 60 milliequivalent per liter (mEq/L) by quantitative pilocarpine iontophoresis
• Participants who are clinically stable with no evidence of acute upper or lower respiratory tract infection

Exclusion Criteria

• Participants with fibrosing colonopathy
• Participants allergic to pork or other porcine PEPs
• Participants with any respiratory condition that in the investigator's opinion would result in an intervention requiring hospitalization or intensive pulmonary treatment during the trial
• Participants with any acute systemic administration of an antibiotic for any reason in the previous 4 weeks; however, a low stable dose of an antibiotic (such as azithromycin 250 or 500 milligram [mg] up to 3 times per week) is allowed. Moreover, chronic treatment (that is, daily for at least 1 month) with an inhalatory antibiotic (for example, colistin, tobramycin, or ceftazidime) is allowed
• Participants who have hepatic insufficiency as defined by a history or presence of ascites, or a serum albumin level of less than 3.0 milligram per deciliter (mg/dL), or coagulopathy with an international normalized ratio that is greater than 1.7
• Participants with hyperuricemia or hyperuricosuria
• Participants participating in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to screening visit
• Participants with history of or current screening evaluation of hyperglycemia as defined by an 8-hour fasting serum glucose equivalent to 126 mg/dL or more, or of cystic-fibrosis-related diabetes as determined according to the Cystic Fibrosis Foundation (CFF) Consensus Conference of January 1999 (Section IX Part II), that is:

• Fasting Blood Glucose (FBG) greater than126 mg/dl (7.0 milli mole [mM]) on two or more occasions
• FBG greater than 126 mg/dl (7 .0 mM) plus casual (without regard to time of day or last meal consumed) glucose level greater than200 mg/dl (11.1 mM)
• Casual (previously called random) glucose levels greater than 200 mg/dl (11.1 mM) on two or more occasions with symptoms
• Participants with any solid organ transplant or surgery affecting the bowel
• Participants using an enzyme preparation in excess of 10,000 lipase units/kg/day
• Participants with an acute dose of any steroid in the previous 2 weeks; however, low chronic doses of a steroid (less 0.5 mg/kg every other day) will be allowed
• Participants with any condition that would, in the investigator's opinion, limit the patient's ability to complete the study
• Participants with history of or current screening determination of distal ileal obstruction syndrome (DIOS), or any clinical signs and symptoms suggestive of DIOS (that is, constipation, abdominal pain, anorexia, early satiety, recurrent vomiting and palpable fecal mass) on physical examination
• Participants who are unable to discontinue excluded concomitant medications over the course of the study

• Maximum Eligible Age: 7 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Forest Laboratories

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Aptalis Medical Information

Role: STUDY_DIRECTOR

Forest Laboratories

Locations

University of Alabama

Birmingham, Alabama, United States

Children's Hospital of Los Angeles

Los Angeles, California, United States

Children's Hospital - Oakland

Oakland, California, United States

Stanford University Medical Center

Palo Alto, California, United States

Children's Hospital of San Diego

San Diego, California, United States

University of Florida College of Medicine

Gainsville, Florida, United States

Nemours Childrens Clinic

Jacksonville, Florida, United States

Childrens Memorial Hospital

Chicago, Illinois, United States

University of Iowa

Iowa City, Iowa, United States

University of Michigan, Cystic Fibrosis Center

Ann Arbor, Michigan, United States

Children's Hospital Medical Center

Cincinnati, Ohio, United States

University of Texas

Tyler, Texas, United States

University of Utah

Salt Lake City, Utah, United States

West Virginia Health Sciences Center

Morgantown, West Virginia, United States

Countries

United States

Other Identifiers

EUR-1009-M

Identifier Type: -

Identifier Source: org_study_id