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

Cystic Fibrosis Clinical Trial

Official title:

An Open Label, Exploratory Study on the Effect of rhBSSL on the Fat Absorption in Patients With Cystic Fibrosis and Pancreatic Insufficiency

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00743483
• Other study ID #: BVT.BSSL-002
• Secondary ID: 2007-004063-21
• Status: Completed
• Phase: Phase 2
• First received: August 26, 2008
• Last updated: November 17, 2014
• Start date: August 2008
• Est. completion date: June 2009

Study information

• Verified date: November 2014
• Source: Swedish Orphan Biovitrum
• Contact: n/a
• Is FDA regulated: No
• Health authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)Poland: Ministry of Health
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the efficacy in patients with cystic fibrosis and pancreatic insufficiency following treatment with BSSL

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: June 2009
• Est. primary completion date: June 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Exocrine Pancreatic Insufficiency
• Fibrosis

Intervention

Drug:
• rhBSSL
oral suspension, 170 mg BSSL, 3 times daily for 5-6 days

Locations

Country	Name	City	State
Netherlands	Erasmus Medical Center	Rotterdam
Poland	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

Sponsors (1)

Lead Sponsor	Collaborator
Swedish Orphan Biovitrum

Countries where clinical trial is conducted

Netherlands,  Poland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
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 %	Final 3 days of baseline and treatment period	No