Use of Ultrase® MT12 in Young Cystic Fibrosis Children (CF)

NCT ID: NCT00880100

Last Updated: 2017-03-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 49 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-04-30
• Study Completion Date: 2009-11-30

Brief Summary

Multicenter, explorative, phase IIIb, open-label study to assess the efficacy and safety of Ultrase® MT12, in the control of steatorrhea and clinical signs and symptoms of malabsorption in CF children with pancreatic insufficiency (PI). This study is sponsored by Aptalis Pharma (formerly Axcan).

Detailed Description

This is a multicenter, explorative, phase IIIb, open-label study in patients with CF and PI. The study consists of a screening visit (visit 1), followed by a baseline phase of 9 days (plus a 5-day window if necessary) during which the regular pancreatic enzyme will be maintained and 10 stool samples will be collected over 5 days, for baseline evaluation of steatorrhea. Afterward, a treatment phase of 19 days (plus a 5-day window if necessary) with Ultrase® MT12 will follow (the usual pancreatic enzyme will be replaced by Ultrase® MT12). Over the last 5 days of the treatment phase, 10 additional stool samples will be collected, for evaluation of steatorrhea.

Conditions

Cystic Fibrosis; Pancreatic Insufficiency

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Ultrase® MT12

Group Type: EXPERIMENTAL

Ultrase® MT12

Intervention Type: DRUG

Ultrase® MT12 capsules will be given orally daily based on investigator's discretion to a maximum dose of 2,500 lipase units per kilogram (kg) body weight per meal or snack for 19 to 24 days during the treatment phase. Total maximum dose not to exceed 10,000 lipase units/kg/day.

Interventions

Ultrase® MT12

Ultrase® MT12 capsules will be given orally daily based on investigator's discretion to a maximum dose of 2,500 lipase units per kilogram (kg) body weight per meal or snack for 19 to 24 days during the treatment phase. Total maximum dose not to exceed 10,000 lipase units/kg/day.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Male or female patients aged 2 to 6 years inclusively
• Patients with current diagnosis of CF based on one or more typical clinical features of CF or a sibling with CF or a positive newborn screening and at least either with sweat chloride test greater than or equal to 60 millimoles/liter (mmol/L) by quantitative pilocarpine iontophoresis on two separate occasions or two identifiable CF-causing mutations
• Patients with presence of PI as demonstrated by fecal elastase (FE-1) less than 100 microgram/gram (mcg/g) of stools (performed by ScheBo test) and requiring pancreatic enzyme supplementation
• Patients who are able to eat a high-fat diet calculated at a value between 2g to 4g fat/kg of body weight per day during the whole study and having a current adequate nutritional status based on the body mass index (BMI) greater than or equal to fifth percentile
• Patients receiving current treatment of PI with pancreatic enzymes
• The parent or legal guardian signed informed consent form (ICF) and is mentally able to understand and comply with the study procedures

Exclusion Criteria

• Patients currently receiving or received an Ultrase® MT product (MT12, MT18, MT20) for PI in the last 30 days
• Patients having known contraindication, sensitivity or hypersensitivity to Ultrase® or to any porcine protein
• Patients with presence of a medical condition known to increase fecal fat loss or that could compromise study results or the study patient safety
• Patients with current diagnosis or history of complete distal intestinal obstruction syndrome (DIOS) in the past 6 months or who had 2 or more episodes of incomplete DIOS in the past year
• Patients with use of any prohibited medication or product at study entry and during the course of the study
• Patients with chronic use of narcotics
• Patients with use of bowel stimulants and/or laxatives more than once a week
• Patients with presence of acute pancreatitis or exacerbation of chronic pancreatic disease
• Patients with presence of an acute infection that needed to be treated with oral or intravenous (IV) broad-spectrum antibiotics
• Patients having history of significant bowel resection; small bowel resection for meconium ileus at birth and appendectomy were accepted. Patients with Presence of dysmotility disorders
• Patients with presence of chronic or severe abdominal pain
• Patients unable to comply with diet requirement
• Patients receiving enteral tube feeding overnight at study entry or who will need to receive enteral tube feeding overnight during the course of the study
• Patients with history of or a current diagnosis of clinically significant portal hypertension
• Patients with presence of poorly controlled diabetes according to the Investigator's clinical judgment
• Patients having any condition or pre-study laboratory abnormality or history of any illness which, in the opinion of the Investigator, might have put the patient at risk, prevented the patient from completing the study, or otherwise affect the outcome of the study
• Patient with use of any investigational drug within 30 days prior to the date of signature of the ICF

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Forest Laboratories

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Aptalis Medical Information

Role: STUDY_DIRECTOR

Forest Laboratories

Locations

The Children's Hospital

Aurora, Colorado, United States

University of Michigan Health System Cystic Fibrosis Center

Ann Arbor, Michigan, United States

Helen DeVos Children's Hospital-Spectrum Health Research Department

Grand Rapids, Michigan, United States

SUNY Upstate Medical University

Syracuse, New York, United States

Duke University Medical Center

Durham, North Carolina, United States

Rainbow Babies and Children's Hospital - Cystic Fibrosis Center

Cleveland, Ohio, United States

Children's Medical Center of Dayton

Dayton, Ohio, United States

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, United States

Respiratory Diseases of Children and Adolescents

Oklahoma City, Oklahoma, United States

Pennsylvania State University and the Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Sanford Children's Specialty Clinic

Sioux Falls, South Dakota, United States

University of Utah

Salt Lake City, Utah, United States

Virginia Commonwealth University

Richmond, Virginia, United States

UW Hospital and Clinics

Madison, Wisconsin, United States

Countries

United States

Other Identifiers

UMT12CF08-01

Identifier Type: -

Identifier Source: org_study_id