Back to resultsEfficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency
Primary Purpose
Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
rhBSSL
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis focused on measuring cystic fibrosis, pancreatic insufficiency, bucelipase alfa, bile salt stimulating lipase, BSSL, coefficient of fat absorption
Eligibility Criteria
Inclusion Criteria:
- Patients with cystic fibrosis and pancreatic insufficiency who are able to refrain from their ongoing pancreatic enzyme treatment for a period of 7 days and are able to produce stools >= 5 times per week
Sites / Locations
- Erasmus Medical Center
- Specjalistyczny Zespól Opieki Zdrowotnej nad Matka i Dzieckiem w Gdansku
- Uniwersytet Medyczny im. K. Marcinkowskiego
- Oddzialu Terenowego Instytutu, Gruzlicy i Chorob Pluc w Rabce-Zdroju
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
rhBSSL
Arm Description
170 mg rhBSSL three times daily for 5 to 6 consecutive days
Outcomes
Primary Outcome Measures
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 %
Secondary Outcome Measures
Full Information
NCT ID
NCT00743483
First Posted
August 26, 2008
Last Updated
November 17, 2014
Sponsor
Swedish Orphan Biovitrum
1. Study Identification
Unique Protocol Identification Number
NCT00743483
Brief Title
Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency
Official Title
An Open Label, Exploratory Study on the Effect of rhBSSL on the Fat Absorption in Patients With Cystic Fibrosis and Pancreatic Insufficiency
Study Type
Interventional
2. Study Status
Record Verification Date
November 2014
Overall Recruitment Status
Completed
Study Start Date
August 2008 (undefined)
Primary Completion Date
June 2009 (Actual)
Study Completion Date
June 2009 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Swedish Orphan Biovitrum
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to evaluate the efficacy in patients with cystic fibrosis and pancreatic insufficiency following treatment with BSSL
Detailed Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Keywords
cystic fibrosis, pancreatic insufficiency, bucelipase alfa, bile salt stimulating lipase, BSSL, coefficient of fat absorption
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)
8. Arms, Groups, and Interventions
Arm Title
rhBSSL
Arm Type
Experimental
Arm Description
170 mg rhBSSL three times daily for 5 to 6 consecutive days
Intervention Type
Drug
Intervention Name(s)
rhBSSL
Other Intervention Name(s)
BSSL, Bucelipase alfa, rhBSSL
Intervention Description
oral suspension, 170 mg BSSL, 3 times daily for 5-6 days
Primary Outcome Measure Information:
Title
The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA)
Description
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 %
Time Frame
Final 3 days of baseline and treatment period
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patients with cystic fibrosis and pancreatic insufficiency who are able to refrain from their ongoing pancreatic enzyme treatment for a period of 7 days and are able to produce stools >= 5 times per week
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jean-Pierre Isal, MD, PhD
Organizational Affiliation
Swedish Orphan Biovitrum
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Janneke Langendonk, MD, PhD
Organizational Affiliation
Erasmus Medical Center, Rotterdam, Netherlands
Official's Role
Principal Investigator
Facility Information:
Facility Name
Erasmus Medical Center
City
Rotterdam
Country
Netherlands
Facility Name
Specjalistyczny Zespól Opieki Zdrowotnej nad Matka i Dzieckiem w Gdansku
City
Gdansk
Country
Poland
Facility Name
Uniwersytet Medyczny im. K. Marcinkowskiego
City
Poznan
Country
Poland
Facility Name
Oddzialu Terenowego Instytutu, Gruzlicy i Chorob Pluc w Rabce-Zdroju
City
Rabka-Zdroj
Country
Poland
12. IPD Sharing Statement
Learn more about this trial
Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency
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