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This study will assess the effect of pancrelipase delayed release 12,000 unit capsules on fat and nitrogen absorption in subjects 7 - 11 with pancreatic exocrine insufficiency due to Cystic Fibrosis.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| A | Experimental |
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| B | Placebo Comparator |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pancrelipase Delayed Release | Drug | 12,000 unit Capsules, dosed individually based on fat intake. |
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| Measure | Description | Time Frame |
|---|---|---|
| Coefficient of Fat Absorption (%) | This coefficient is calculated from fat intake and fat excretion : 100*[fat intake-fat excretion]/fat intake. Stools were collected on 3 days during the 5 days treatment period. Higher values indicate a better response. | 5 days |
| Measure | Description | Time Frame |
|---|---|---|
| Coefficient of Nitrogen Absorption (%) | This coefficient is calculated from nitrogen intake and nitrogen excretion : 100*[nitrogen intake-nitrogen excretion]/nitrogen intake. Stools were collected on 3 days during the 5 days treatment period. Higher values indicate a better response. | 5 days |
| Total Fat Excretion (Grams) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Global Clinical Director Solvay | Solvay Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Site 2 | Iowa City | Iowa | United States | |||
| Site 5 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 20171415 | Result | Graff GR, Maguiness K, McNamara J, Morton R, Boyd D, Beckmann K, Bennett D. Efficacy and tolerability of a new formulation of pancrelipase delayed-release capsules in children aged 7 to 11 years with exocrine pancreatic insufficiency and cystic fibrosis: a multicenter, randomized, double-blind, placebo-controlled, two-period crossover, superiority study. Clin Ther. 2010 Jan;32(1):89-103. doi: 10.1016/j.clinthera.2010.01.012. | |
| 21681115 |
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Twenty three subjects had given their consent and 17 subjects were randomly allocated to pancrelipase/placebo or placebo/pancrelipase. One subject did not complete the first period of the treatment (consent withdrawal).
Subjects were recruited in 10 centers in US between June 2008 and October 2008. Before the randomization, subjects were evaluated for eligibility. They underwent a short period of up to 14 days on their usual pancreatic enzyme supplementation.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo/Pancrelipase | Placebo period followed by Pancrelipase delayed release 12000 units period |
| FG001 | Pancrelipase/Placebo | Pancrelipase delayed release 12000 units period followed by Placebo period |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Period 1 |
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| Placebo Comparator |
| Drug |
Placebo |
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Total amount of fat excreted during the stool collection period. Stools were collected on 3 days during the 5 days treatment period. Lower values indicate a better response. |
| 5 days |
| Total Stool Weight (Grams) | Total weight of the stools collected during the stool collection period. Stools were collected on 3 days during the 5 days treatment period. Lower values indicate a better response. | 5 days |
| Stool Frequency | Stool frequency is the average of the daily number of stools recorded during the treatment period. Lower values indicate a better response. | 5 days |
| Percentage of Days With no Flatulence. | The percentage of days with no flatulence is calculated from the diary during the treatment period: 100*(number of days with no flatulence/number of days recorded in diary). Higher values indicate a better response. | 5 days |
| Percentage of Days With Formed/Normal Stools. | The percentage of days with formed/normal stools is calculated from the diary during the treatment period: 100*(number of days with formed/normal stools/number of days with any stool). Higher values indicate a better response. | 5 days |
| Percentage of Days With no Abdominal Pain. | The percentage of days with no abdominal pain is calculated from the diary during the treatment period: 100*(number of days with no abdominal pain / number of days recorded in diary). Higher values indicate a better response. | 5 days |
| Louisville |
| Kentucky |
| United States |
| Site 9 | Boston | Massachusetts | United States |
| Site 6 | Ann Arbor | Michigan | United States |
| Site 4 | Minneapolis | Minnesota | United States |
| Site 8 | Albuquerque | New Mexico | United States |
| Site 1 | Cincinnati | Ohio | United States |
| Site 10 | Oklahoma City | Oklahoma | United States |
| Site 7 | Oklahoma City | Oklahoma | United States |
| Site 3 | Hershey | Pennsylvania | United States |
| Derived |
| Caras S, Boyd D, Zipfel L, Sander-Struckmeier S. Evaluation of stool collections to measure efficacy of PERT in subjects with exocrine pancreatic insufficiency. J Pediatr Gastroenterol Nutr. 2011 Dec;53(6):634-40. doi: 10.1097/MPG.0b013e3182281c38. |
| COMPLETED |
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| NOT COMPLETED |
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| Period 2 |
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo/Pancrelipase | Placebo period followed by Pancrelipase delayed release 12000 units period |
| BG001 | Pancrelipase/Placebo | Pancrelipase delayed release 12000 units period followed by Placebo period |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Coefficient of Fat Absorption (%) | This coefficient is calculated from fat intake and fat excretion : 100*[fat intake-fat excretion]/fat intake. Stools were collected on 3 days during the 5 days treatment period. Higher values indicate a better response. | The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement. | Posted | Mean | Standard Deviation | Percentage | 5 days |
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| Secondary | Coefficient of Nitrogen Absorption (%) | This coefficient is calculated from nitrogen intake and nitrogen excretion : 100*[nitrogen intake-nitrogen excretion]/nitrogen intake. Stools were collected on 3 days during the 5 days treatment period. Higher values indicate a better response. | The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement. | Posted | Mean | Standard Deviation | Percentage | 5 days |
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| Secondary | Total Fat Excretion (Grams) | Total amount of fat excreted during the stool collection period. Stools were collected on 3 days during the 5 days treatment period. Lower values indicate a better response. | The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement. | Posted | Mean | Standard Deviation | Grams | 5 days |
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| Secondary | Total Stool Weight (Grams) | Total weight of the stools collected during the stool collection period. Stools were collected on 3 days during the 5 days treatment period. Lower values indicate a better response. | The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement. | Posted | Mean | Standard Deviation | Grams | 5 days |
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| Secondary | Stool Frequency | Stool frequency is the average of the daily number of stools recorded during the treatment period. Lower values indicate a better response. | The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement. | Posted | Mean | Standard Deviation | Number per day | 5 days |
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| Secondary | Percentage of Days With no Flatulence. | The percentage of days with no flatulence is calculated from the diary during the treatment period: 100*(number of days with no flatulence/number of days recorded in diary). Higher values indicate a better response. | The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement. | Posted | Mean | Standard Deviation | Percentage of days | 5 days |
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| Secondary | Percentage of Days With Formed/Normal Stools. | The percentage of days with formed/normal stools is calculated from the diary during the treatment period: 100*(number of days with formed/normal stools/number of days with any stool). Higher values indicate a better response. | The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement. | Posted | Mean | Standard Deviation | Percentage of days | 5 days |
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| Secondary | Percentage of Days With no Abdominal Pain. | The percentage of days with no abdominal pain is calculated from the diary during the treatment period: 100*(number of days with no abdominal pain / number of days recorded in diary). Higher values indicate a better response. | The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement. | Posted | Mean | Standard Deviation | Percentage of days | 5 days |
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The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic.
Only Treatment Emergent Adverse events are presented.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Placebo treatment | 0 | 16 | 9 | 16 | ||
| EG001 | Pancrelipase | Pancrelipase delayed release 12000 units treatment | 0 | 17 | 5 | 17 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Rectal Discharge | Gastrointestinal disorders | MedDRA 8.1 | Non-systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 8.1 | Non-systematic Assessment |
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| Faecal Volume Increased | Gastrointestinal disorders | MedDRA 8.1 | Non-systematic Assessment |
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| Flatulence | Gastrointestinal disorders | MedDRA 8.1 | Non-systematic Assessment |
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| Abdominal Pain | Gastrointestinal disorders | MedDRA 8.1 | Non-systematic Assessment |
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| Frequent Bowel Movements | Gastrointestinal disorders | MedDRA 8.1 | Non-systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 8.1 | Non-systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 8.1 | Non-systematic Assessment |
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| Blood glucose increased | Investigations | MedDRA 8.1 | Non-systematic Assessment |
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| Weight Decrease | Investigations | MedDRA 8.1 | Non-systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 8.1 | Non-systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA 8.1 | Non-systematic Assessment |
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| Cold sweat | Skin and subcutaneous tissue disorders | MedDRA 8.1 | Non-systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 8.1 | Non-systematic Assessment |
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| Rash Macular | Skin and subcutaneous tissue disorders | MedDRA 8.1 | Non-systematic Assessment |
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| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 8.1 | Non-systematic Assessment |
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At 60 days prior to submitting or presenting a manuscript or other materials relating to the study to a publisher, reviewer or outside persons, the Site shall provide to Sponsor a copy and allow Sponsor 60 days to review and comment.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Sven Voet - Global Communication | Solvay Pharmaceuticals | +32(0)2 509 69 77 | sven.voet@solvay.com |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| D010188 | Exocrine Pancreatic Insufficiency |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
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