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

NCT00981214 | Completed Phase 3 Results DMC

• 1 other identifier: EUR-1009-M
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 14

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).

Conditions

Cystic Fibrosis; Exocrine Pancreatic Insufficiency

Keywords

Cystic fibrosis; Exocrine Pancreatic Insufficiency; Zenpep-1009

Outcome Measures

Primary Outcomes (2)

• Percentage of Participants Who Were Responders After 1 Week of Treatment With Study Medication

  Responders were defined as those participants without steatorrhea (defined as less than 30 percent (%) fecal fat content) and without signs and symptoms of malabsorption after 1 week of treatment with study medication.

  Day 11

• Percentage of Participants Who Were Responders After 2 Weeks of Treatment With Study Medication

  Responders were defined as those participants without steatorrhea (defined as less than 30% fecal fat content) and without signs and symptoms of malabsorption after 2 weeks of treatment with study medication.

  Day 18 (end of treatment)

Secondary Outcomes (9)

• Change From Baseline in Weight at Day 12, 19

  Baseline, Day 12, 19

• Mean Daily Number of Stools

  Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period)

• Percentage of Stool Categorized by Consistency

  Baseline, Day 5 up to Day 11 (dose stabilization period) and Day 12 up to Day 18 (treatment period)

• Mean Number of Abdominal Symptoms: Bloating

  Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period)

• Mean Number of Abdominal Symptoms: Flatulence

  Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period)

Study Arms (1)

EUR-1008 (APT-1008)

EXPERIMENTAL

Drug: EUR-1008 (APT-1008)

Interventions

EUR-1008 (APT-1008) 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).

Also known as: ZENPEP®, Pancrelipase

EUR-1008 (APT-1008)

Eligibility Criteria

• Age: Up to 7 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Forest Laboratories: lead

Study Sites (14)

University of Alabama

Birmingham, Alabama, 35294, United States

Location

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Location

Children's Hospital - Oakland

Oakland, California, 94609, United States

Location

Stanford University Medical Center

Palo Alto, California, 94304, United States

Location

Children's Hospital of San Diego

San Diego, California, 92123, United States

Location

University of Florida College of Medicine

Gainsville, Florida, 32610-0296, United States

Location

Nemours Childrens Clinic

Jacksonville, Florida, 32250, United States

Location

Childrens Memorial Hospital

Chicago, Illinois, 60614, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

University of Michigan, Cystic Fibrosis Center

Ann Arbor, Michigan, 48109, United States

Location

Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

University of Texas

Tyler, Texas, 75708, United States

Location

University of Utah

Salt Lake City, Utah, 84108, United States

Location

West Virginia Health Sciences Center

Morgantown, West Virginia, 26506, 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

Limitations and Caveats

Protocol amendment 4 extended screening period to 14 days, but this period not included in result as all participants started treatment prior to site Institutional Review Board approval of protocol amendment 4, this did not affect study procedures.

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 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 21, 2009
• First Posted: September 22, 2009
• Study Start: May 1, 2006
• Primary Completion: September 1, 2006
• Study Completion: September 1, 2006
• Last Updated: March 16, 2017
• Results First Posted: April 4, 2014

Record last verified: 2017-02

Locations

US (14)