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

Primary Purpose

Cystic Fibrosis, Exocrine Pancreatic Insufficiency

Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Ultrase® MT20
Placebo
Sponsored by
Forest Laboratories
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis focused on measuring Cystic Fibrosis, Exocrine Pancreatic Insufficiency, Ultrase® MT20

Eligibility Criteria

7 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • DeVos Children's Hospital
  • Rainbow Babies & Children's Hospital
  • Pennsylvania State University, The Milton S. Hershey Medical Center
  • University of Utah Health Sciences Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Ultrase® MT20

Placebo

Arm Description

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Percent Coefficient of Nitrogen Absorption (CNA)
Percent (%) CNA was calculated as [(nitrogen intake-nitrogen excretion)/nitrogen intake]*100, determined by the stools collected during the 72-hour period which could extend to 96 hours during both intervention periods. Nitrogen intake was calculated as protein intake/6.25. Mean percent CNA was calculated for 72-hour/96-hour period during Day 3 to Day 7 in the first and second intervention periods.

Full Information

First Posted
December 4, 2006
Last Updated
February 7, 2017
Sponsor
Forest Laboratories
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1. Study Identification

Unique Protocol Identification Number
NCT00408317
Brief Title
Safety and Efficacy Study of ULTRASE® MT20 in Participants With Cystic Fibrosis (CF) and Exocrine Pancreatic Insufficiency (PI)
Official Title
A Multicenter, Randomized, Double-Blind, Crossover Study to Compare the Safety and Efficacy of Ultrase® MT20 to Placebo for the Correction of Steatorrhea in Patients With Cystic Fibrosis (CF)
Study Type
Interventional

2. Study Status

Record Verification Date
February 2017
Overall Recruitment Status
Completed
Study Start Date
November 2006 (undefined)
Primary Completion Date
April 2007 (Actual)
Study Completion Date
April 2007 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Forest Laboratories

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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).
Detailed 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Keywords
Cystic Fibrosis, Exocrine Pancreatic Insufficiency, Ultrase® MT20

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
36 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Ultrase® MT20
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Ultrase® MT20
Other Intervention Name(s)
Pancreatic enzyme product
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo matched to Ultrase® MT 20 capsules orally daily for 6 to 7 days in either first intervention period or second intervention period.
Primary Outcome Measure Information:
Title
Percent Coefficient of Fat Absorption (CFA)
Description
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.
Time Frame
Day 3 to Day 7 in first intervention period and second intervention period
Secondary Outcome Measure Information:
Title
Percent Coefficient of Nitrogen Absorption (CNA)
Description
Percent (%) CNA was calculated as [(nitrogen intake-nitrogen excretion)/nitrogen intake]*100, determined by the stools collected during the 72-hour period which could extend to 96 hours during both intervention periods. Nitrogen intake was calculated as protein intake/6.25. Mean percent CNA was calculated for 72-hour/96-hour period during Day 3 to Day 7 in the first and second intervention periods.
Time Frame
Day 3 to Day 7 in first intervention period and second intervention period
Other Pre-specified Outcome Measures:
Title
Number of Bowel Movements
Description
Number of bowel movements of each participant was calculated from frequency of stools by the participant per day. Mean daily number of bowel movements on Day 3 for the first treatment period and second treatment period was summarized.
Time Frame
Day 3 on first intervention period and second intervention period
Title
Percentage of Stool Categorized by Consistency
Description
Stool consistency was categorized as hard, formed/normal, soft or watery stool. Percentage of stools of a specific consistency of each participant was calculated as the number of stools with a specific consistency relative to the total number of stools during the collection period. Mean percentage of stool with specific consistency on Day 4 for the first treatment period and second treatment period period for total participants was summarized.
Time Frame
Day 4 on first intervention period and second intervention period

10. Eligibility

Sex
All
Minimum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Aptalis Medical Information
Organizational Affiliation
Forest Laboratories
Official's Role
Study Director
Facility Information:
Facility Name
DeVos Children's Hospital
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
49503
Country
United States
Facility Name
Rainbow Babies & Children's Hospital
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
Pennsylvania State University, The Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
University of Utah Health Sciences Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States

12. IPD Sharing Statement

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

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