Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency
The purpose of this study is to evaluate the efficacy in patients with cystic fibrosis and pancreatic insufficiency following treatment with BSSL
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||An Open Label, Exploratory Study on the Effect of rhBSSL on the Fat Absorption in Patients With Cystic Fibrosis and Pancreatic Insufficiency|
- The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA) [ Time Frame: Final 3 days of baseline and treatment period ] [ Designated as safety issue: No ]
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 %
|Study Start Date:||August 2008|
|Study Completion Date:||June 2009|
|Primary Completion Date:||June 2009 (Final data collection date for primary outcome measure)|
170 mg rhBSSL three times daily for 5 to 6 consecutive days
oral suspension, 170 mg BSSL, 3 times daily for 5-6 days
Other Name: BSSL, Bucelipase alfa, rhBSSL
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00743483
|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|
|Study Director:||Jean-Pierre Isal, MD, PhD||Swedish Orphan Biovitrum|
|Principal Investigator:||Janneke Langendonk, MD, PhD||Erasmus Medical Center, Rotterdam, Netherlands|