Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency

Phase 2

Completed

• Conditions: Cystic Fibrosis; Exocrine Pancreatic Insufficiency
• Interventions: Drug: rhBSSL

• Registration Number: NCT00743483
• Lead Sponsor: Swedish Orphan Biovitrum

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.

Study Timeline

• Study Start: 2008-08
• Study First Submitted: 2008-08-26
• Study First Submitted QC: 2008-08-27
• Study First Posted: 2008-08-28
• Primary Completion: 2009-06
• Study Completion: 2009-06
• Status Verified: 2014-11
• Results First Submitted: 2014-11-05
• Results First Submitted QC: 2014-11-17
• Last Update Submitted: 2014-11-17
• Results First Posted: 2014-11-18
• Last Update Posted: 2014-11-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

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

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
rhBSSL	rhBSSL	170 mg rhBSSL three times daily for 5 to 6 consecutive days

Primary Outcome Measures

Name	Time	Method
The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA)	Final 3 days of baseline and treatment period	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 %

Trial Locations

Locations (4)

Erasmus Medical Center; 🇳🇱 Rotterdam, Netherlands

Specjalistyczny Zespól Opieki Zdrowotnej nad Matka i Dzieckiem w Gdansku; 🇵🇱 Gdansk, Poland

Uniwersytet Medyczny im. K. Marcinkowskiego; 🇵🇱 Poznan, Poland

Oddzialu Terenowego Instytutu, Gruzlicy i Chorob Pluc w Rabce-Zdroju; 🇵🇱 Rabka-Zdroj, Poland