- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00662675
A Study of the Efficacy and Tolerability of Pancrelipase Microtablet (MT) Capsules for the Treatment of Cystic Fibrosis-dependent Exocrine Pancreatic Insufficiency
April 24, 2014 updated by: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
A Randomized Double-blind (Withdrawal) Phase 3 Study to Evaluate the Efficacy and Tolerability of Pancrelipase MT Capsules Compared With Placebo in the Treatment of Subjects With Cystic Fibrosis-dependent Exocrine Pancreatic Insufficiency
The purpose of this study is to assess the effectiveness and safety of oral pancrelipase MT in the treatment of adult and pediatric/adolescent cystic fibrosis (CF) patients with clinical symptoms of exocrine pancreatic insufficiency (EPI).
Study Overview
Status
Completed
Intervention / Treatment
Detailed Description
This is a randomized, placebo-controlled, double-blind withdrawal, multicenter study to evaluate the effectiveness of pancrelipase MT capsules compared with placebo in the treatment of adult (>18 to 60 years of age) and children/adolescent (7 to <18 years of age) patients with CF and who require pancreatic enzyme replacement therapy (PERT) to control clinical symptoms of EPI and steatorrhea (excess fat in the feces).
The study has 3 phases: a screening phase, an open-label (run-in) phase, and a double-blind withdrawl phase.
The study including the screening phase will be approximately 28 days in length.
In the screening phase, patients will begin a high-fat diet and will take pancrelipase MT10.5 or MT21 capsules (or a combination of both) orally with meals (or snacks) to optimize digestion based on clinical signs and symptoms.
In the open-label phase patients will continue taking their optimal dose of study drug.
After a minimum of 3 days in the open-label treatment phase, an inpatient 72-hour stool collection period for fecal fat determination will be performed.
Patients with a coefficient of fat absorption (COA)-fat of 80% or greater who have completed at least 6 days on a controlled high-fat diet will be eligible for the double-blind withdrawal phase of the study and will be randomly assigned to receive placebo or pancrelipase MT.
After a minimum of 1 day on double-blind treatment and with the presence of deteriorating clinical signs and symptoms, patients will be admitted to the clinic to begin a second 72-hour inpatient stool collection period.
Effectiveness evaluations will be performed throughout the study and consist of stool collection for determination of COA-fat and coefficient of protein absorption (COA-protein), stool diary, nutrition worksheet, and Clinical Global Impression-Severity of illness (CGI-S), Clinical Global Impression-Change (CGI-C), and Global Assessment of Change (GAC) scales.
Signs and symptoms exhibited during the study will be monitored and will include the presence or absence of diarrhea, abdominal pain, nausea, vomiting, bloating, and a description of stool changes.
Safety will be montitored during the study by evaluating adverse events and findings from clinical laboratory tests, vital signs measurements, and physical examinations.
The study hypothesis is that the study drug will be more effective than placebo as measured by the change in the coefficient of fat absorption (COA-fat) in adults and pediatric/adolescent patients with EPI secondary to CF. Pancrelipase MT10.5 or MT21 capsules (or a combination of both) will be taken orally with meals (or snacks) within the recommended ranges of pancreatic enzyme therapy as recommended by the CF Foundation and up to a maximum 10,000 lipase units per kilogram [kg] per day.
All patients will take pancrelipase MT for 6 days in the screening phase and for approximately 6 to 10 days in the open-label phase; patients will take pancrelipase MT or placebo for 4 to 7 days in the double-blind phase.
Study Type
Interventional
Enrollment (Actual)
40
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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British Columbia
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Vancouver N/A, British Columbia, Canada
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California
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Long Beach, California, United States
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Los Angeles, California, United States
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Florida
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Orlando, Florida, United States
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Kentucky
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Louisville, Kentucky, United States
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Nevada
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Las Vegas, Nevada, United States
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New Jersey
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Long Branch, New Jersey, United States
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Ohio
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Cincinnati, Ohio, United States
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Cleveland, Ohio, United States
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Oklahoma
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Oklahoma City, Oklahoma, United States
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Pennsylvania
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Pittsburgh, Pennsylvania, United States
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
7 years to 60 years (ADULT, CHILD)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Have a diagnosis of CF documented by sweat chloride results (>60 mmol/L) and require pancreatic enzyme replacement therapy (PERT) to control clinical symptoms of EPI (nausea, vomiting, bloating, diarrhea, and abdominal pain) with a history of excess fat in the feces
- Have documentation of an abnormal COA-fat and a fecal elastase result of <100 micrograms fecal elastase/gram stool
- Must be on a stable diet and dose of pancreatic enzyme supplementation that has provided satisfactory symptom control for at least the past 1 month
Exclusion Criteria:
- No extreme physical wasting with loss of weight and muscle mass
- No severe, acute, or chronic pulmonary disease unrelated to complications of CF
- No worsening of pulmonary disease in past 30 days
- No use of drugs known to affect blood uric acid concentrations (e.g., aspirin, diflunisal, allopurinol, probenecid, thiazide diuretics, phenylbutazone, sulfinpyrazone)
- No known congenital (present at birth) abnormalities of the gastrointestinal tract, heart, or liver
- No distal intestinal obstruction syndrome (DIOS)
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: RANDOMIZED
- Interventional Model: PARALLEL
- Masking: QUADRUPLE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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EXPERIMENTAL: 001
Pancrease MT 10.5 or MT 21 Pancrease MT capsules for maximum dose of 10 000 lipase units / Kg / day
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Pancrease MT capsules for maximum dose of 10,000 lipase units / Kg / day
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EXPERIMENTAL: 002
Placebo for Pancrease MT 10.5 or MT 21 Capsules with Pancrease MT excipients without the active enzymes
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Capsules with Pancrease MT excipients without the active enzymes
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in the Coefficient of Fat Absorption (COA-fat Percent)
Time Frame: 72-hours stool collection in the open-label phase to the end of 72-hours stool collection in the double-blind withdrawal phase.
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Change in the coefficient of fat absorption (percent COA-fat) from the 72-hour inpatient period in the open-label phase to the 72-hour period inpatient period in the double-blind (withdrawal) phase.
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72-hours stool collection in the open-label phase to the end of 72-hours stool collection in the double-blind withdrawal phase.
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in Percent COA-Protein (Nitrogen)
Time Frame: 72-hours stool collection in the open-label phase to the end of 72-hours stool collection in the double-blind withdrawal phase.
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The change in percent COA-protein from the stool collection period in double-blind phase to open-label phase
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72-hours stool collection in the open-label phase to the end of 72-hours stool collection in the double-blind withdrawal phase.
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Percent of Patients Reporting Clinical Signs and Symptoms of Exocrine Pancreatic Insufficiency (EPI) During the Double-Blind Phase
Time Frame: Entire 7 days double-blind phase
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Percent of patients reporting nausea, vomiting, bloating, diarrhea, oily/greasy stools, and abdominal pain signs and symptoms reported as Adverse events during the double-blind phase.
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Entire 7 days double-blind phase
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
August 1, 2008
Primary Completion (ACTUAL)
February 1, 2009
Study Completion (ACTUAL)
February 1, 2009
Study Registration Dates
First Submitted
April 17, 2008
First Submitted That Met QC Criteria
April 17, 2008
First Posted (ESTIMATE)
April 21, 2008
Study Record Updates
Last Update Posted (ESTIMATE)
May 9, 2014
Last Update Submitted That Met QC Criteria
April 24, 2014
Last Verified
April 1, 2014
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Digestive System Diseases
- Pathologic Processes
- Metabolic Diseases
- Respiratory Tract Diseases
- Lung Diseases
- Gastrointestinal Diseases
- Infant, Newborn, Diseases
- Genetic Diseases, Inborn
- Intestinal Diseases
- Pancreatic Diseases
- Fibrosis
- Cystic Fibrosis
- Malabsorption Syndromes
- Exocrine Pancreatic Insufficiency
- Steatorrhea
- Gastrointestinal Agents
- Pancrelipase
- Pancreatin
Other Study ID Numbers
- CR014719
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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