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

November 17, 2014 updated by: Swedish Orphan Biovitrum

An Open Label, Exploratory Study on the Effect of rhBSSL on the Fat Absorption 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 Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Netherlands
      • Rotterdam, Netherlands
        • Erasmus Medical Center
  • Poland
      • Gdansk, Poland
        • Specjalistyczny Zespól Opieki Zdrowotnej nad Matka i Dzieckiem w Gdansku
      • Poznan, Poland
        • Uniwersytet Medyczny im. K. Marcinkowskiego
      • Rabka-Zdroj, Poland
        • Oddzialu Terenowego Instytutu, Gruzlicy i Chorob Pluc w Rabce-Zdroju

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: rhBSSL
170 mg rhBSSL three times daily for 5 to 6 consecutive days
Drug: rhBSSL
oral suspension, 170 mg BSSL, 3 times daily for 5-6 days
Other Names:
  • BSSL, Bucelipase alfa, rhBSSL

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA)
Time Frame: 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 %
Final 3 days of baseline and treatment period

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Swedish Orphan Biovitrum

Investigators

  • Study Director: Jean-Pierre Isal, MD, PhD, Swedish Orphan Biovitrum
  • Principal Investigator: Janneke Langendonk, MD, PhD, Erasmus Medical Center, Rotterdam, Netherlands

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2008

Primary Completion (Actual)

June 1, 2009

Study Completion (Actual)

June 1, 2009

Study Registration Dates

First Submitted

August 26, 2008

First Submitted That Met QC Criteria

August 27, 2008

First Posted (Estimate)

August 28, 2008

Study Record Updates

Last Update Posted (Estimate)

November 18, 2014

Last Update Submitted That Met QC Criteria

November 17, 2014

Last Verified

November 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BVT.BSSL-002
  • 2007-004063-21 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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