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

Sobi

Status and phase: Completed; Phase 2

Conditions: Cystic Fibrosis; Exocrine Pancreatic Insufficiency

Treatments: Drug: rhBSSL

Study type: Interventional

Funder types: Industry

Identifiers

NCT00743483

2007-004063-21 (EudraCT Number)

BVT.BSSL-002

Details and patient eligibility

About

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

Full 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.

Enrollment: 15 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 1 patient group

rhBSSL

Experimental group

Description:

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

Treatment:

Drug: rhBSSL