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

NCT00408317 | Completed Phase 3 Results DMC

• 1 other identifier: UMT20CF05-01
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 4

Brief Summary

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).

Conditions

Cystic Fibrosis; Exocrine Pancreatic Insufficiency

Keywords

Cystic Fibrosis; Exocrine Pancreatic Insufficiency; Ultrase® MT20

Outcome Measures

Primary Outcomes (1)

• Percent Coefficient of Fat Absorption (CFA)

  Percent (%) CFA was calculated as (\[fat intake - fat excretion\]/fat intake)\*100, determined by the stools collected during the 72-hour period which could extend to 96 hours during both intervention periods. Mean CFA percent was calculated for 72-hour/96-hour period during Day 3 to Day 7 in the first and second intervention periods.

  Day 3 to Day 7 in first intervention period and second intervention period

Secondary Outcomes (1)

• Percent Coefficient of Nitrogen Absorption (CNA)

  Day 3 to Day 7 in first intervention period and second intervention period

Other Outcomes (2)

• Number of Bowel Movements

  Day 3 on first intervention period and second intervention period

• Percentage of Stool Categorized by Consistency

  Day 4 on first intervention period and second intervention period

Study Arms (2)

Ultrase® MT20

EXPERIMENTAL

Drug: Ultrase® MT20

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Ultrase® MT20 DRUG

Ultrase® MT 20 capsules containing enteric-coated minitablets orally daily at a dose stabilized during the first stabilization period (4 days), as per investigator's discretion, for 6 to 7 days in either first intervention period or second intervention period.

Also known as: Pancreatic enzyme product

Ultrase® MT20

Placebo DRUG

Placebo matched to Ultrase® MT 20 capsules orally daily for 6 to 7 days in either first intervention period or second intervention period.

Placebo

Eligibility Criteria

• Age: 7 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Forest Laboratories: lead

Study Sites (4)

DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

Location

Rainbow Babies & Children's Hospital

Cleveland, Ohio, 44106, United States

Location

Pennsylvania State University, The Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

University of Utah Health Sciences Center

Salt Lake City, Utah, 84112, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis; Exocrine Pancreatic Insufficiency

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

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: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 4, 2006
• First Posted: December 6, 2006
• Study Start: November 1, 2006
• Primary Completion: April 1, 2007
• Study Completion: April 1, 2007
• Last Updated: March 16, 2017
• Results First Posted: March 12, 2014

Record last verified: 2017-02

Locations

US (4)