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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Swedish Orphan Biovitrum
ClinicalTrials.gov Identifier:
NCT00743483
First received: August 26, 2008
Last updated: November 17, 2014
Last verified: November 2014
  Purpose

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


Condition Intervention Phase
Cystic Fibrosis
Exocrine Pancreatic Insufficiency
Drug: rhBSSL
Phase 2

Study Type: Interventional
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

Resource links provided by NLM:


Further study details as provided by Swedish Orphan Biovitrum:

Primary Outcome Measures:
  • 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 %



Enrollment: 15
Study Start Date: August 2008
Study Completion Date: June 2009
Primary Completion Date: June 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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
  More Information

No publications provided

Responsible Party: Swedish Orphan Biovitrum
ClinicalTrials.gov Identifier: NCT00743483     History of Changes
Other Study ID Numbers: BVT.BSSL-002, 2007-004063-21
Study First Received: August 26, 2008
Results First Received: November 5, 2014
Last Updated: November 17, 2014
Health Authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Poland: Ministry of Health

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

ClinicalTrials.gov processed this record on November 27, 2014