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

NCT00743483 | Completed Phase 2 Results

• 2 other identifiers: BVT.BSSL-0022007-004063-21
• Study Type: interventional
• Target: 15
• Locations: 2 countries
• Sites: 4

Brief Summary

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

Conditions

Cystic Fibrosis; Exocrine Pancreatic Insufficiency

Keywords

cystic fibrosis; pancreatic insufficiency; bucelipase alfa; bile salt stimulating lipase; BSSL; coefficient of fat absorption

Outcome Measures

Primary Outcomes (1)

• The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA)

  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 %

  Final 3 days of baseline and treatment period

Study Arms (1)

rhBSSL

EXPERIMENTAL

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

Drug: rhBSSL

Interventions

rhBSSL DRUG

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

Also known as: BSSL, Bucelipase alfa, rhBSSL

rhBSSL

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead

Study Sites (4)

Erasmus Medical Center

Rotterdam, Netherlands

Location

Specjalistyczny Zespól Opieki Zdrowotnej nad Matka i Dzieckiem w Gdansku

Gdansk, Poland

Location

Uniwersytet Medyczny im. K. Marcinkowskiego

Poznan, Poland

Location

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

Rabka-Zdrój, Poland

Location

MeSH Terms

Conditions

Cystic Fibrosis; Exocrine Pancreatic Insufficiency

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Results Point of Contact

• Title: Maria Öhman, TA Clinical Head Neonatology
• Organization: Swedish Orphan Biovitrum

maria.ohman@sobi.com

+46 70 694 81 61

Study Officials

• Jean-Pierre Isal, MD, PhD

  Swedish Orphan Biovitrum

  STUDY DIRECTOR

• Janneke Langendonk, MD, PhD

  Erasmus Medical Center, Rotterdam, Netherlands

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 26, 2008
• First Posted: August 28, 2008
• Study Start: August 1, 2008
• Primary Completion: June 1, 2009
• Study Completion: June 1, 2009
• Last Updated: November 18, 2014
• Results First Posted: November 18, 2014

Record last verified: 2014-11

Locations

PL (3); NL (1)