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| ID | Type | Description | Link |
|---|---|---|---|
| 2007-004063-21 | EudraCT Number |
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The purpose of this study is to evaluate the efficacy in patients with cystic fibrosis and pancreatic insufficiency following treatment with BSSL
In this open study, patients will enter a baseline period of 6 days where the pancreatic enzyme therapy will be discontinued and a standard diet given. After the baseline period, patients will enter a treatment period of 6 days where a fixed dose of BSSL will be administered. The primary efficacy measurements will be made by collecting stool during the last three days of each period.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| rhBSSL | Experimental | 170 mg rhBSSL three times daily for 5 to 6 consecutive days |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| rhBSSL | Drug | oral suspension, 170 mg BSSL, 3 times daily for 5-6 days |
|
| Measure | Description | Time Frame |
|---|---|---|
| The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA) | The absolute difference between baseline and treatment CFA, i.e. the change from the baseline level. CFA was calculated as follows 100 x ((fat consumed - fat excreted)/fat consumed). Fat consumed was determined from the weight of fat of the dietary intake during a 72 hour period during the final 3 days of the baseline and treatment period. Fat excreted was determined from stool collected during the 72-hour periods and analyzed for fat using the Van de Kamer method. The unit of CFA is % | Final 3 days of baseline and treatment period |
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Inclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Jean-Pierre Isal, MD, PhD | Swedish Orphan Biovitrum | Study Director |
| Janneke Langendonk, MD, PhD | Erasmus Medical Center, Rotterdam, Netherlands | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Erasmus Medical Center | Rotterdam | Netherlands | ||||
| Specjalistyczny Zespól Opieki Zdrowotnej nad Matka i Dzieckiem w Gdansku |
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| ID | Title | Description |
|---|---|---|
| FG000 | rhBSSL | Oral suspension, 170 mg rhBSSL, 3 times daily for 5-6 days |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Baseline Period |
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| ||||||||||||||||||
| Treatment Period |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | BSSL | Bucelipase alfa (INN): oral suspension, 170 mg BSSL, 3 times daily for 5-6 days |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA) | The absolute difference between baseline and treatment CFA, i.e. the change from the baseline level. CFA was calculated as follows 100 x ((fat consumed - fat excreted)/fat consumed). Fat consumed was determined from the weight of fat of the dietary intake during a 72 hour period during the final 3 days of the baseline and treatment period. Fat excreted was determined from stool collected during the 72-hour periods and analyzed for fat using the Van de Kamer method. The unit of CFA is % | Per protocol | Posted | Mean | Standard Deviation | % CFA | Final 3 days of baseline and treatment period |
|
Treatment period (5-6 days)
Only treatment emergent events (events that started post-rhBSSL administration) is 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 | BSSL | Oral suspension, 170 mg, 3 times daily for 5-6 days |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cystic Fibrosis Exacerbation | Congenital, familial and genetic disorders | MedDRA (11.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA (11.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Maria Öhman, TA Clinical Head Neonatology | Swedish Orphan Biovitrum | +46 70 694 81 61 | maria.ohman@sobi.com |
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| 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 |
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| Gdansk |
| Poland |
| Uniwersytet Medyczny im. K. Marcinkowskiego | Poznan | Poland |
| Oddzialu Terenowego Instytutu, Gruzlicy i Chorob Pluc w Rabce-Zdroju | Rabka-Zdrój | Poland |
|
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
| 1 |
| 14 |
| 6 |
| 14 |
| Viral Infection | Infections and infestations | MedDRA (11.0) | Systematic Assessment |
|
| C-reactive protein increased | Investigations | MedDRA (11.0) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (11.0) | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA (11.0) | Systematic Assessment |
|
| Cystic fibrosis lung | Congenital, familial and genetic disorders | MedDRA (11.0) | Systematic Assessment |
|
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| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |