An Effectiveness, Safety, and Palatability Study of Pancrelipase Microtablets in Infants and Toddlers With Cystic Fibrosis and Fat Malabsorption

McNeil-PPC

Status and phase

Completed

Phase 2

Conditions

Steatorrhea

Cystic Fibrosis

Treatments

Drug: Pancrelipase microtablets

Study type

Interventional

Funder types

Industry

Identifiers

NCT00217204

CR002665

Details and patient eligibility

About

The purpose of this study is to evaluate the effectiveness and safety of PANCREASE MT (pancrelipase microtablets) to improve steatorrhea (excessive excretion of fat in feces) in infants and toddlers with cystic fibrosis who have pancreatic insufficiency, and to assess whether the consistency of the microtablets is acceptable for swallowing in infants and toddlers

Full description

The objective of this randomized, Investigator-blinded, parallel group, multicenter, pilot study is to evaluate the preliminary safety, palatability and effectiveness of pancrelipase microtablets to improve fat absorption. The hypothesis is that PANCREASE MT will provide effective, safe and palatable pancreatic enzyme supplementation to be used for the treatment of fat malabsorption in a cohort of infants with cystic fibrosis-related pancreatic insufficiency. On Day 1 of the study, parents will be instructed to administer 500 units lipase/kg/meal for a full five days. Stool will be collected and analyzed during the last 72 hours of this baseline period. On Day 6 of the study, subjects will be randomly assigned to one of four treatment groups. Parents will be instructed to administer the appropriate dose for a full five days and stool will be collected and analyzed during the last 72 hours of this randomized treatment period. Patients will receive PANCREASE MT 500 units lipase/kg/meal by mouth for a maximum of five doses per day for the first 120 hours. Patients will receive PANCREASE MT 500, 1000, 1500 or 2000 units lipase/kg/meal by mouth, for a maximum of five meals per day for the next 120 hours.

Enrollment

18 patients

Sex

All

Ages

6 to 30 months old

Volunteers

No Healthy Volunteers

Inclusion criteria

Diagnosis of cystic fibrosis
Excessive discharge of fat in feces
Stable patient requiring pancreatic enzyme therapy

Exclusion criteria

No stable antibiotic therapy for small bowel overgrowth
No hypersensitivity to pork products
No use of prokinetics eg, metoclopramide or cisapride within the last 30 days
No nasogastric feeding tube feeding
No use of steroids
No use of concomitant H2 blockers or proton pump inhibitors as concomitant therapy