Efficacy and Safety of Ultrase® MT20 in Improving the Coefficient of Fat Absorption (CFA) in Children With Cystic Fibrosis (CF) and Pancreatic Insufficiency (PI)

NCT00513682 | Completed Phase 3 Results DMC

• 1 other identifier: UMT20CF07-01
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 3

Brief Summary

This is a Phase III, multicenter, open-label study, that will evaluate the improvement of nutrient absorption when participants will receive Ultrase® MT20. This study is sponsored by Aptalis (formerly Axcan). This study is performed in children from 7 to 11 years old.

Conditions

Cystic Fibrosis; Pancreatic Insufficiency

Keywords

Cystic Fibrosis; Pancreatic insufficiency; Steatorrhea; Children 7 to 11 years; Pancreatic enzyme

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 in either washout phase or treatment phase. Mean percent CFA was calculated for Day 3 to Day 5 or Day 6 of the respective phase.

  Day 3 to Day 5 or Day 6 during washout phase and treatment phase

Secondary Outcomes (1)

• Percent Coefficient of Nitrogen Absorption (CNA)

  Day 3 to Day 5 or Day 6 during washout phase and treatment phase

Study Arms (1)

Ultrase® MT20

EXPERIMENTAL

Drug: Ultrase® MT20

Interventions

Ultrase® MT20 DRUG

Ultrase® MT20 capsules will be administered orally with each meal during Day 1 to 15 in screening phase at a dose based on investigator's discretion. During Day 12 to 15, participants will receive high-fat diet and Ultrase® MT20 dose will be adjusted depending on symptoms of steatorrhea. This will be followed by a washout phase of 6 to 7 days, in which participants will receive only high-fat diet; then stabilized dose of Ultrase® MT20 capsule (as identified during screening phase) will be administered orally for 7 to 11 days during treatment phase. The stabilized dose should not to exceed 2500 lipase units per kilogram body weight per meal (lipase units/kg/meal).

Ultrase® MT20

Eligibility Criteria

• Age: 7 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• After a full explanation of the study, participants or their legally authorized representative must have signed the informed consent or assent form along with a parental form
• Participants must have a confirmed diagnosis of CF based on one or more clinical features consistent with the CF phenotype, and one of the following:
• A genotype with two identifiable mutations consistent with CF
• A sweat chloride test greater that 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 requiring pancreatic enzyme supplementation
• Participants must be clinically stable according to the physician's judgment by:
• Medical and medication history
• Baseline physical examination including vital signs and laboratory analyses
• Participants must be 7 to 11 years of age
• Participants must have an adequate nutritional status based on body mass index (BMI) greater than or equal to fifth percentile
• Participants must be on an optimal clinical dose of pancreatic enzymes (Ultrase® MT12 , MT18 or MT20 or other pancreatic enzyme preparations) prior to entry in the study and must tolerate this medication in the opinion of the investigator
• Participants must be able to be able to swallow capsules and eat a high-fat diet calculated as 2 gram fat per kilogram (g fat/kg) of body weight per day
• Participants must be, in the opinion of the investigator, able and willing to complete the study
• Female participants should be premenarcheal. Otherwise, a female participant of childbearing potential (WOCBP) must not be pregnant and must have practiced an acceptable method of contraception for at least 1 month prior to the study entry

You may not qualify if:

• Participants with a known contraindication, sensitivity or hypersensitivity to Ultrase® MT20 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 who are not willing to stop the prohibited medications or products at study entry and throughout the study
• Participants who use narcotics chronically and 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 with 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, ulcerative colitis, 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 according to the investigator's opinion

Sponsors & Collaborators

• Forest Laboratories: lead

Study Sites (3)

University of Michigan Health System Cystic Fibrosis Center

Ann Arbor, Michigan, 48109-0212, United States

Location

Cystic Fibrosis Center Rainbow Babies and Children's HospitalDivision

Cleveland, Ohio, 44106, United States

Location

Pennsylvania State University And the Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis; Exocrine Pancreatic Insufficiency; Steatorrhea

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; Malabsorption Syndromes; Intestinal Diseases; Gastrointestinal Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Limitations and Caveats

No limitations

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: August 7, 2007
• First Posted: August 9, 2007
• Study Start: July 1, 2007
• Primary Completion: March 1, 2008
• Study Completion: April 1, 2008
• Last Updated: March 16, 2017
• Results First Posted: April 10, 2014

Record last verified: 2017-02

Locations

US (3)