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

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ClinicalTrials.gov Identifier: NCT00743483
Recruitment Status : Completed
First Posted : August 28, 2008
Results First Posted : November 18, 2014
Last Update Posted : November 18, 2014
Sponsor:
Information provided by (Responsible Party):
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

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Exocrine Pancreatic Insufficiency Drug: rhBSSL Phase 2

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  (Clinical Trial)
Actual Enrollment : 15 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : August 2008
Actual Primary Completion Date : June 2009
Actual Study Completion Date : June 2009

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
U.S. FDA Resources

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 Name: BSSL, Bucelipase alfa, rhBSSL



Primary Outcome Measures :
  1. 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 %




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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00743483


Locations
Netherlands
Erasmus Medical Center
Rotterdam, Netherlands
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, Poland
Sponsors and Collaborators
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

Responsible Party: Swedish Orphan Biovitrum
ClinicalTrials.gov Identifier: NCT00743483     History of Changes
Other Study ID Numbers: BVT.BSSL-002
2007-004063-21 ( EudraCT Number )
First Posted: August 28, 2008    Key Record Dates
Results First Posted: November 18, 2014
Last Update Posted: November 18, 2014
Last Verified: November 2014

Keywords provided by Swedish Orphan Biovitrum:
cystic fibrosis
pancreatic insufficiency
bucelipase alfa
bile salt stimulating lipase
BSSL
coefficient of fat absorption

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Exocrine Pancreatic Insufficiency
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases