Safety and Efficacy Study of ULTRASE® MT20 in Participants With Cystic Fibrosis (CF) and Exocrine Pancreatic Insufficiency (PI)

Forest Laboratories

Status and phase

Completed

Phase 3

Conditions

Cystic Fibrosis

Exocrine Pancreatic Insufficiency

Treatments

Drug: Placebo

Drug: Ultrase® MT20

Study type

Interventional

Funder types

Industry

Identifiers

NCT00408317

UMT20CF05-01

Details and patient eligibility

About

The purpose of this study is to assess the safety and efficacy of Ultrase® MT20 compared to placebo for the correction of fat and protein malabsorption in participants with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI). This study is sponsored by Aptalis Pharma (formerly Axcan).

Full description

This is a Phase III, multicenter, randomized, double-blind, two-period cross-over, placebo-controlled study designed to compare the efficacy and safety of Ultrase® MT20 to placebo in participants with CF and pancreatic insufficiency. The study consists of a screening period (up to 11 days) and two treatment periods (6-7 days). During screening period participants will be treated with open-label Ultrase® MT18 or MT20. Each treatment period will be preceded by a stabilization period (4 days) and the two treatment periods are separated by a break period (3-6 days). A safety follow-up visit will be performed 7-10 days after discharge from the last treatment period.

Enrollment

36 patients

Sex

All

Ages

7+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Participants or their legally authorized representative must understand the nature of the study and sign an informed consent or assent form along with a parental form
Participants must have a confirmed diagnosis of CF based on 1 or more clinical features consistent with the CF phenotype, and one of the following:
- A genotype with 2 identifiable mutations consistent with CF
- A sweat chloride test greater than 60 millimole per liter (mmol/L) by quantitative pilocarpine iontophoresis
Participants must have PI as demonstrated by a fecal elastase-1 (FE-1) concentration less than 100 microgram per gram (mcg/g) of stools (ScheBo test) and must require pancreatic enzyme supplementation
Participants must be clinically stable as evidenced by medical and medication history, baseline physical examination including vital signs and laboratory analyses
Participants must be 7 years and older
Participants must have an adequate nutritional status based on the following body mass index (BMI):
- Participants 7 to 20 years old must have a BMI greater than or equal to fifth percentile
- Female participants greater than 20 years old must have a BMI greater than or equal to 16
- Male participants greater 20 years old must have a BMI greater than or equal to 16.5
Participants must be on an optimal clinical dose of pancreatic enzymes (Ultrase® MT18 or MT20 or other pancreatic enzymes preparations including Ultrase® MT12) prior to entry in the study, and must tolerate this medication in the opinion of the investigator
Participants must be able to swallow capsules and must be able to eat a high fat diet calculated as 2 gram (± 15%) fat per kilogram body weight per day
Participants must be, in the opinion of the investigator, able and willing to complete this study
Female participants must be premenarcheal, surgically sterile or postmenopausal for at least 12 consecutive months. Otherwise, the women of childbearing potential (WOCBP) must not be pregnant and must have practiced an acceptable method of contraception for at least one month prior to the study entry

Exclusion criteria

Participants with a known contraindication, sensitivity or hypersensitivity to Ultrase or any porcine protein
Participants with a known allergy to the food drug and cosmetic (FD&C) Blue No. 2 dye indicator (stool marker)
Participants not willing to stop the prohibited medications or products at study entry and throughout the study
Participants who are using narcotics
Participants who are using bowel stimulants and/or laxatives on a regular basis
Participants with acute pancreatitis or acute exacerbation of chronic pancreatic disease
Participants with an acute pulmonary infection
Participants with a history of bowel resection
Participants suffering from any dysmotility disorders
Participants with chronic or severe abdominal pain
Participants receiving enteral tube feeding and not willing to stop during the course of the study
Participants known to have a significant medical disease that would compromise their welfare or confound the study results
Participants with a history of or a current diagnosis of clinically significant portal hypertension
Participants who have a condition known to increase fecal fat loss including celiac's disease, biliary cancer, biliary stricture, cholelithiasis, Crohn's disease, pancreas cancer, radiation enteritis, tropical sprue, Whipple's disease, lactose intolerance, pseudomembranous colitis
Participants with a current diagnosis or a history of complete distal intestinal obstruction syndrome (DIOS) in the past 6 months; or, participants who had 2 or more episodes of DIOS in the past year
Participants with poorly controlled diabetes to the investigator's opinion
Female participants who are pregnant or lactating
Participants who received an investigational drug within 30 days prior to entry into the study