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

NCT ID: NCT00743483

Last Updated: 2014-11-18

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-08-31

Study Completion Date

2009-06-30

Brief Summary

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

Detailed Description

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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.

Conditions

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Cystic Fibrosis Exocrine Pancreatic Insufficiency

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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rhBSSL

170 mg rhBSSL three times daily for 5 to 6 consecutive days

Group Type EXPERIMENTAL

rhBSSL

Intervention Type DRUG

oral suspension, 170 mg BSSL, 3 times daily for 5-6 days

Interventions

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rhBSSL

oral suspension, 170 mg BSSL, 3 times daily for 5-6 days

Intervention Type DRUG

Other Intervention Names

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BSSL, Bucelipase alfa, rhBSSL

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Swedish Orphan Biovitrum

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Jean-Pierre Isal, MD, PhD

Role: STUDY_DIRECTOR

Swedish Orphan Biovitrum

Janneke Langendonk, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Erasmus Medical Center, Rotterdam, Netherlands

Locations

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Erasmus Medical Center

Rotterdam, , Netherlands

Site Status

Specjalistyczny Zespól Opieki Zdrowotnej nad Matka i Dzieckiem w Gdansku

Gdansk, , Poland

Site Status

Uniwersytet Medyczny im. K. Marcinkowskiego

Poznan, , Poland

Site Status

Oddzialu Terenowego Instytutu, Gruzlicy i Chorob Pluc w Rabce-Zdroju

Rabka-Zdrój, , Poland

Site Status

Countries

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Netherlands Poland

Other Identifiers

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2007-004063-21

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

BVT.BSSL-002

Identifier Type: -

Identifier Source: org_study_id