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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ultrase® MT12 | Experimental |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ultrase® MT12 | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Patients With Control of Steatorrhea | Control of steatorrhea was defined as a less than 30 percent (%) of fat in stools as measured by nuclear magnetic resonance (NMR) spectroscopy in all stool samples which are collected at baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and during the 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12. | A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Patients With Normal Stool Frequency | Normal stool frequency was defined as having less than 4 bowel movements per day in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the Treatment Phase during which the PEP was replaced with Ultrase MT12. | A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) |
| Measure | Description | Time Frame |
|---|---|---|
| Total Weight of Stools | The total weight of stools in grams (g) is the total weight obtained during the stool collection period regardless of the number of stools that had been collected during this same collection period. Mean total weight of stools in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Aptalis Medical Information | Forest Laboratories | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Children's Hospital | Aurora | Colorado | 80045 | United States | ||
| University of Michigan Health System Cystic Fibrosis Center |
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The enrollment started in April 2009 and was completed in November 2009. Cystic fibrosis (CF) patients with pancreatic insufficiency (PI), aged 2 to 6 years old inclusively, were enrolled from 14 CF centers located in United States of America.
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| ID | Title | Description |
|---|---|---|
| FG000 | Ultrase® MT12 | Patients received usual pancreatic enzymes therapy for 9 to 14 days during baseline phase followed by Ultrase® MT12 capsules 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 was not to exceed 10,000 lipase units/kg/day. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Baseline Phase-Usual Pancreatic Enzymes |
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| Percentage of Stools With Normal Consistency | Normal consistency of stool was defined as hard and formed or soft and formed consistency. Abnormal consistency was defined as loose and unformed stool or liquid stools and diarrhea. Percentage of stools with normal consistency of each patient was calculated from normal consistency of stools by the patient per day. Mean percentage of stools with normal consistency in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized. | A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) |
| Percentage of Stools With Abnormal Characteristics | Stools of abnormal characteristics were defined as bulky/large, foul-smelling and/or oily stools. Mean percentage of stools with abnormal characteristics in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized. | A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) |
| Mean Number of Days Without Abdominal Complaints | Abdominal complaints were defined as the reporting of abdominal pain and/or unusual and excessive flatulence/gas production. Mean number of days without abdominal complaints in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized. | A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) |
| A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) |
| Percentage of Days With Abdominal Pain and Excessive Flatulence | Mean percentage of days with abdominal complaints during baseline phase (BP) and the 5-day collection period of the treatment phase for total patients was summarized. Abdominal complaints were defined as the reporting of abdominal pain and/or unusual and excessive flatulence/gas production. Mean number of days abdominal pain (AP) and excessive flatulence (EF) in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized. | A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) |
| Ann Arbor |
| Michigan |
| 48109-0212 |
| United States |
| Helen DeVos Children's Hospital-Spectrum Health Research Department | Grand Rapids | Michigan | 40503 | United States |
| SUNY Upstate Medical University | Syracuse | New York | 13203 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Rainbow Babies and Children's Hospital - Cystic Fibrosis Center | Cleveland | Ohio | 44106 | United States |
| Children's Medical Center of Dayton | Dayton | Ohio | 45404 | United States |
| University of Oklahoma Health Sciences Center | Oklahoma City | Oklahoma | 73104 | United States |
| Respiratory Diseases of Children and Adolescents | Oklahoma City | Oklahoma | 73112 | United States |
| Pennsylvania State University and the Milton S. Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15213 | United States |
| Sanford Children's Specialty Clinic | Sioux Falls | South Dakota | 57117-5039 | United States |
| University of Utah | Salt Lake City | Utah | 84108 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23298 | United States |
| UW Hospital and Clinics | Madison | Wisconsin | 53792 | United States |
| COMPLETED |
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| NOT COMPLETED |
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| Treatment Phase With Ultrase® MT12 |
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Safety population included patients who signed an informed consent form (ICF), completed the baseline phase and started the treatment phase by taking at least one dose of the study treatment.
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| ID | Title | Description |
|---|---|---|
| BG000 | Ultrase® MT12 | Ultrase® MT12 capsules were 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 was not to exceed 10,000 lipase units/kg/day. |
| 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, Categorical | Count of Participants | Participants |
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | 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 | Percentage of Patients With Control of Steatorrhea | Control of steatorrhea was defined as a less than 30 percent (%) of fat in stools as measured by nuclear magnetic resonance (NMR) spectroscopy in all stool samples which are collected at baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and during the 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12. | The Intent-to-treat (ITT) population included all patients who signed an informed consent form (ICF) and started the baseline phase. The 50th percentile imputation method was used for missing data. | Posted | Number | 95% Confidence Interval | percentage of patients | A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) |
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| Secondary | Percentage of Patients With Normal Stool Frequency | Normal stool frequency was defined as having less than 4 bowel movements per day in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the Treatment Phase during which the PEP was replaced with Ultrase MT12. | The ITT population included all patients who signed an ICF and started the baseline phase. Here "n" signifies patients who were evaluable for each specified category. | Posted | Number | 95% Confidence Interval | percentage of patients | A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) |
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| Secondary | Percentage of Stools With Normal Consistency | Normal consistency of stool was defined as hard and formed or soft and formed consistency. Abnormal consistency was defined as loose and unformed stool or liquid stools and diarrhea. Percentage of stools with normal consistency of each patient was calculated from normal consistency of stools by the patient per day. Mean percentage of stools with normal consistency in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized. | The ITT population included all patients who signed an ICF and started the baseline phase. Here "n" signifies patients who were evaluable for each specified category. | Posted | Mean | Standard Deviation | percentage of stools | A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) |
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| Secondary | Percentage of Stools With Abnormal Characteristics | Stools of abnormal characteristics were defined as bulky/large, foul-smelling and/or oily stools. Mean percentage of stools with abnormal characteristics in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized. | The ITT population included all patients who signed an ICF and started the baseline phase. Here "n" signifies patients who were evaluable for each specified category. | Posted | Mean | Standard Deviation | percentage of stools | A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) |
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| Secondary | Mean Number of Days Without Abdominal Complaints | Abdominal complaints were defined as the reporting of abdominal pain and/or unusual and excessive flatulence/gas production. Mean number of days without abdominal complaints in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized. | The ITT population included all patients who signed an ICF and started the baseline phase. Here "n" signifies patients who were evaluable for each specified category. | Posted | Mean | Standard Deviation | days | A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) |
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| Other Pre-specified | Total Weight of Stools | The total weight of stools in grams (g) is the total weight obtained during the stool collection period regardless of the number of stools that had been collected during this same collection period. Mean total weight of stools in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized. | The ITT population included all patients who signed an ICF and started the baseline phase. Here "N" (number of patients analyzed) represents number of patients who were evaluable for this outcome measure. Here "n" signifies patients who were evaluable for each specified category. | Posted | Mean | Standard Deviation | gram (g) | A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) |
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| Other Pre-specified | Percentage of Days With Abdominal Pain and Excessive Flatulence | Mean percentage of days with abdominal complaints during baseline phase (BP) and the 5-day collection period of the treatment phase for total patients was summarized. Abdominal complaints were defined as the reporting of abdominal pain and/or unusual and excessive flatulence/gas production. Mean number of days abdominal pain (AP) and excessive flatulence (EF) in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12, for total patients was summarized. | The ITT population included all patients who signed an ICF and started the baseline phase. Here "n" signifies patients who were evaluable for each specific category. | Posted | Mean | Standard Deviation | percentage of days | A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) |
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| Post-Hoc | Percentage of Patients With Control of Steatorrhea Based on Concomitant Use of Proton Pump Inhibitors (PPIs) | Control of steatorrhea was defined as a less than 30 percent (%) of fat in stools as measured by nuclear magnetic resonance (NMR) spectroscopy in all stool samples which are collected in baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12. | The ITT population included all patients who signed an ICF and started the baseline phase. There was no imputation of missing data. Here "n" signifies patients who were evaluable for each specific category. | Posted | Number | 95% Confidence Interval | percentage of patients | A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3) |
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From signing of informed consent up to the study discharge (last study visit on Day 24 of treatment phase or early discontinuation visit)
Safety population included patients who signed an ICF, completed the baseline phase and started the treatment phase by taking at least one dose of the study treatment. Adverse event (AE) was defined as any untoward medical occurrence regardless of causal relationship to study medication.
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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 | Ultrase® MT12 | Ultrase® MT12 capsules were 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 was not to exceed 10,000 lipase units/kg/day. | 0 | 48 | 24 | 48 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Feacal fat increased | Investigations | MedDRA (12.0) | Systematic Assessment |
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| Decreased appetite | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
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Restrictions vary in accordance with each agreement with the individual investigators. Sponsor will allow publication after a multi-center publication has been published or after an agreed period of time if no such multi-center publication is submitted for publication. Sponsor can ask that Sponsor's confidential information be removed from any publication and can defer publication for a period of time to allow for Sponsor to obtain patent or other intellectual property right protection.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Robert Winkler, MD, VP, Clinical Development and Operations | Aptalis Pharma US, Inc. | 1- 800- 472- 263 |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| D010188 | Exocrine Pancreatic Insufficiency |
| D045602 | Steatorrhea |
| D015746 | Abdominal Pain |
| 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 |
| D008286 | Malabsorption Syndromes |
| D007410 | Intestinal Diseases |
| D005767 | Gastrointestinal Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D010146 | Pain |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012817 | Signs and Symptoms, Digestive |
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