Cystic FibrosisPhase 2NCT00217204
An Effectiveness, Safety, and Palatability Study of Pancrelipase Microtablets in Infants and Toddlers With Cystic Fibrosis and Fat Malabsorption
A Phase 2 study testing Pancrelipase microtablets in people with Cystic Fibrosis and Steatorrhea, run by McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc..
Trial summary
The purpose of this study is to evaluate the effectiveness and safety of PANCREASE MT (pancrelipase microtablets) to improve steatorrhea (excessive excretion of fat in feces) in infants and toddlers with cystic fibrosis who have pancreatic insufficiency, and to assess whether the consistency of the microtablets is… Read more about this study
- Who is it for?
- Ages 6 to 30 months
- Conditions the study is for
- Cystic Fibrosis, Steatorrhea
- What is being tested?
- Pancrelipase microtablets
- How many people
- 18 participants
- Where
- No sites listed yet
- Study dates
- Jul 2005to Feb 2006
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 30 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of cystic fibrosis
- Excessive discharge of fat in feces
- Stable patient requiring pancreatic enzyme therapy
Who cannot join
- No stable antibiotic therapy for small bowel overgrowth
- No hypersensitivity to pork products
- No use of prokinetics eg, metoclopramide or cisapride within the last 30 days
- No nasogastric feeding tube feeding
- No use of steroids
- No use of concomitant H2 blockers or proton pump inhibitors as concomitant therapy
About this study
Official title: A Phase II, Randomized, Investigator-Blinded, Parallel-Group, Pilot Study Evaluating the Safety, Palatability and Effectiveness of Four Doses of Pancrelipase Microtablets in the Treatment of Infants and Toddlers With Cystic Fibrosis-Related Pancreatic Insufficiency and Fat Malabsorption
The purpose of this study is to evaluate the effectiveness and safety of PANCREASE MT (pancrelipase microtablets) to improve steatorrhea (excessive excretion of fat in feces) in infants and toddlers with cystic fibrosis who have pancreatic insufficiency, and to assess whether the consistency of the microtablets is acceptable for swallowing in infants and toddlers
Read the detailed description
The objective of this randomized, Investigator-blinded, parallel group, multicenter, pilot study is to evaluate the preliminary safety, palatability and effectiveness of pancrelipase microtablets to improve fat absorption. The hypothesis is that PANCREASE MT will provide effective, safe and palatable pancreatic enzyme supplementation to be used for the treatment of fat malabsorption in a cohort of infants with cystic fibrosis-related pancreatic insufficiency. On Day 1 of the study, parents will be instructed to administer 500 units lipase/kg/meal for a full five days. Stool will be collected and analyzed during the last 72 hours of this baseline period. On Day 6 of the study, subjects will be randomly assigned to one of four treatment groups. Parents will be instructed to administer the appropriate dose for a full five days and stool will be collected and analyzed during the last 72 hours of this randomized treatment period. Patients will receive PANCREASE MT 500 units lipase/kg/meal by mouth for a maximum of five doses per day for the first 120 hours. Patients will receive PANCREASE MT 500, 1000, 1500 or 2000 units lipase/kg/meal by mouth, for a maximum of five meals per day for the next 120 hours.
- Conditions
- Cystic Fibrosis, Steatorrhea
- Being tested
- Pancrelipase microtablets
- Keywords
- Cystic fibrosis, Pancreatic insufficiency, Steatorrhea, Fat malabsorption, Pancrelipase
- Medical subject headings
- Cystic Fibrosis, Steatorrhea, Exocrine Pancreatic Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pancrelipase microtabletsDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in coefficient of fecal fat absorption from baseline to end of study period. Palatability. Percent carbon dioxide expired by 13C-mixed triglyceride breath test measuring exogenous lipase activity.
Secondary outcomes (1)
Clinical Global Impression Severity Subscale. Global Change Subscale. Weight gain/loss. Global Assessment effectiveness. Nitrogen excretion/24 hr. Mean dose of pancrease assessed/day & weight. Mean daily calories, fat intake. Coefficient fat absorption
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2005 (estimated)
- Study startJul 2005
- Study completionFeb 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- McNeil Consumer & Specialty Pharmaceuticals, a Division of Mc Neil-PPC, Inc. Clinical Trial, Study director, McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00217204
- Sponsor's protocol ID
- CR002665
- Status last verified
- Apr 2010
- First submitted
- 13 Sep 2005
- Last updated
- 19 May 2011
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 May 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Randomized, Investigator-Blinded, Parallel-Group, Pilot Study Evaluating the Safety, Palatability and Effectiveness of Four Doses of Pancrelipase Microtablets in the Treatment of Infants and Toddlers With Cystic Fibrosis-Related Pancreatic Insufficiency and Fat Malabsorption
- ClinicalTrials.gov ID
- NCT00217204
- Acronym / sponsor ID
- CR002665
- Conditions
- Cystic Fibrosis, Steatorrhea
- Keywords
- Cystic fibrosis, Pancreatic insufficiency, Steatorrhea, Fat malabsorption, Pancrelipase
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.Industry (pharma, biotech, devices)
- Study start
- Jul 2005
- Study completion
- Feb 2006 (actual)
- First posted
- 22 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the effectiveness and safety of PANCREASE MT (pancrelipase microtablets) to improve steatorrhea (excessive excretion of fat in feces) in infants and toddlers with cystic fibrosis who have pancreatic insufficiency, and to assess whether the consistency of the microtablets is acceptable for swallowing in infants and toddlers
Detailed description
The objective of this randomized, Investigator-blinded, parallel group, multicenter, pilot study is to evaluate the preliminary safety, palatability and effectiveness of pancrelipase microtablets to improve fat absorption. The hypothesis is that PANCREASE MT will provide effective, safe and palatable pancreatic enzyme supplementation to be used for the treatment of fat malabsorption in a cohort of infants with cystic fibrosis-related pancreatic insufficiency. On Day 1 of the study, parents will be instructed to administer 500 units lipase/kg/meal for a full five days. Stool will be collected and analyzed during the last 72 hours of this baseline period. On Day 6 of the study, subjects will be randomly assigned to one of four treatment groups. Parents will be instructed to administer the appropriate dose for a full five days and stool will be collected and analyzed during the last 72 hours of this randomized treatment period. Patients will receive PANCREASE MT 500 units lipase/kg/meal by mouth for a maximum of five doses per day for the first 120 hours. Patients will receive PANCREASE MT 500, 1000, 1500 or 2000 units lipase/kg/meal by mouth, for a maximum of five meals per day for the next 120 hours.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
Who may qualify
- Ages
- 6 to 30 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Diagnosis of cystic fibrosis
- Excessive discharge of fat in feces
- Stable patient requiring pancreatic enzyme therapy
Key exclusion criteria (6 listed)
- No stable antibiotic therapy for small bowel overgrowth
- No hypersensitivity to pork products
- No use of prokinetics eg, metoclopramide or cisapride within the last 30 days
- No nasogastric feeding tube feeding
- No use of steroids
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Pancrelipase microtablets | Drug | – |
Primary outcome
Change in coefficient of fecal fat absorption from baseline to end of study period. Palatability. Percent carbon dioxide expired by 13C-mixed triglyceride breath test measuring exogenous lipase activity.
1 secondary outcome
Clinical Global Impression Severity Subscale. Global Change Subscale. Weight gain/loss. Global Assessment effectiveness. Nitrogen excretion/24 hr. Mean dose of pancrease assessed/day & weight. Mean daily calories, fat intake. Coefficient fat absorption
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 May 2011.
- An Effectiveness, Safety, and Palatability Study of Pancrelipase Microtablets in Infants and Toddlers With Cystic Fibrosis and Fat MalabsorptionLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
McNeil Consumer & Specialty Pharmaceuticals, a Division of Mc Neil-PPC, Inc. Clinical Trial study director
McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pancrelipase microtablets in people with Cystic Fibrosis and Steatorrhea, run by McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 May 2011. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in coefficient of fecal fat absorption from baseline to end of study period. Palatability. Percent carbon dioxide expired by 13C-mixed triglyceride breath test measuring exogenous lipase activity.. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 May 2011.