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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00712348




Registration number
NCT00712348
Ethics application status
Date submitted
7/07/2008
Date registered
10/07/2008
Date last updated
4/10/2018

Titles & IDs
Public title
Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase
Scientific title
A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase in Patients With Gaucher Disease Treated With Imiglucerase
Secondary ID [1] 0 0
PB-06-002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Gaucher Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Taliglucerase alfa

Experimental: Taliglucerase alfa - Open label taliglucerase alfa treatment


Treatment: Drugs: Taliglucerase alfa
Intravenous infusion every 2 weeks
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Hemoglobin
Timepoint [1] 0 0
Every 3 months from Baseline to Month 9

Eligibility
Key inclusion criteria
* Males and females, 2 years or older
* Confirmed diagnosis of Gaucher disease by the enzymatic activity assay
* Stable Gaucher disease
* Treatment with imiglucerase (Cerezyme®) for at least 2 years and on a stable maintenance regimen (dose and regimen unchanged, except for situation of drug shortage) for at least the last six months
* Able to provide written informed consent
Minimum age
2 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Currently taking another experimental drug for any condition
* History of allergy to carrots
* History of allergy to beta lactam antibiotics
* Previous infusion reaction suspected to be allergic in nature to Cerezyme® or Ceredase® or receiving premedication to prevent infusion reactions
* Presence of HIV and/or HBsAg and/or hepatitis C infection
* Presence of unresolved anemia due to iron, folic acid or vitamin B12 deficiency
* Presence of any significant comorbidity that could confound the interpretation of the clinical response to taliglucerase alfa
* Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the requirements of the study

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/12/2008
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/05/2013
Sample size
Target
Accrual to date
Final
31
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Bone Marrow Transplant Service, The Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
- Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
New York
Country [4] 0 0
Canada
State/province [4] 0 0
Ontario
Country [5] 0 0
Israel
State/province [5] 0 0
Haifa
Country [6] 0 0
Israel
State/province [6] 0 0
Jerusalem
Country [7] 0 0
Spain
State/province [7] 0 0
Zaragoza
Country [8] 0 0
United Kingdom
State/province [8] 0 0
Cambridge
Country [9] 0 0
United Kingdom
State/province [9] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Pfizer
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a multi-center, open-label, switchover trial to assess the safety of taliglucerase alfa in 30 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) enzyme replacement therapy.
Trial website
https://clinicaltrials.gov/study/NCT00712348
Trial related presentations / publications
Pastores GM, Petakov M, Giraldo P, Rosenbaum H, Szer J, Deegan PB, Amato DJ, Mengel E, Tan ES, Chertkoff R, Brill-Almon E, Zimran A. A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imiglucerase. Blood Cells Mol Dis. 2014 Dec;53(4):253-60. doi: 10.1016/j.bcmd.2014.05.004. Epub 2014 Jun 18.
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT00712348