Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency
This study has been completed.
Sponsor:
Swedish Orphan Biovitrum
Information provided by:
Swedish Orphan Biovitrum
ClinicalTrials.gov Identifier:
NCT00743483
First received: August 26, 2008
Last updated: August 4, 2009
Last verified: August 2009
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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: Bucelipase alfa (INN) |
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:
Genetics Home Reference related topics:
cystic fibrosis
MedlinePlus related topics:
Cystic Fibrosis
U.S. FDA Resources
Further study details as provided by Swedish Orphan Biovitrum:
Primary Outcome Measures:
- Coefficient of fat absorption measured in stool collected for a 72-hour period during the final 3 days of each period [ Time Frame: Final 3 days of each 6-day treatment period ] [ Designated as safety issue: No ]
| 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: BSSL |
Drug: Bucelipase alfa (INN)
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
Exclusion Criteria:
Contacts and Locations
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: | Maria Öhman, MSc Pharm, Clinical Program Leader, Clinical Development, Biovitrum AB |
| ClinicalTrials.gov Identifier: | NCT00743483 History of Changes |
| Other Study ID Numbers: | BVT.BSSL-002, EudraCT Number: 2007-004063-21 |
| Study First Received: | August 26, 2008 |
| Last Updated: | August 4, 2009 |
| 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 Fibrosis Exocrine Pancreatic Insufficiency Pancreatic Diseases Digestive System Diseases |
Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Pathologic Processes |
ClinicalTrials.gov processed this record on May 21, 2013