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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00705939




Registration number
NCT00705939
Ethics application status
Date submitted
25/06/2008
Date registered
27/06/2008
Date last updated
4/10/2018

Titles & IDs
Public title
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Scientific title
A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
Secondary ID [1] 0 0
PB-06-003
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(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - Taliglucerase alfa

Experimental: Naive 30 Units/kg - Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)

Experimental: Naive 60 Units/kg - Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)

Experimental: Switchover - Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348)


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
Spleen Volume
Timepoint [1] 0 0
Spleen Volume at Baseline and Months 12, 24, and 36
Secondary outcome [1] 0 0
Liver Volume
Timepoint [1] 0 0
Liver volume at Baseline and Months 12, 24 and 36
Secondary outcome [2] 0 0
Hemoglobin
Timepoint [2] 0 0
Hemoglobin at Baseline and Months 12, 24 and 36
Secondary outcome [3] 0 0
Platelet Count
Timepoint [3] 0 0
Platelet count at Baseline and Months 12, 24 and 36

Eligibility
Key inclusion criteria
* Successful completion of Protocol PB-06-001
* The patient signs informed consent
Minimum age
18 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
* Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease
* Pregnant or nursing
* 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
Non-randomised trial
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
Parallel
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
1/06/2008
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/08/2013
Sample size
Target
Accrual to date
Final
45
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
Georgia
Country [2] 0 0
United States of America
State/province [2] 0 0
New York
Country [3] 0 0
Canada
State/province [3] 0 0
Ontario
Country [4] 0 0
Chile
State/province [4] 0 0
Santiago
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
South Africa
State/province [7] 0 0
Morningside
Country [8] 0 0
Spain
State/province [8] 0 0
Zaragoza
Country [9] 0 0
United Kingdom
State/province [9] 0 0
Cambridge
Country [10] 0 0
United Kingdom
State/province [10] 0 0
London

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

Ethics approval
Ethics application status

Summary
Brief summary
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system.

This is an extension trial to Study NCT00376168 and NCT00712348.
Trial website
https://clinicaltrials.gov/study/NCT00705939
Trial related presentations / publications
Zimran A, Duran G, Mehta A, Giraldo P, Rosenbaum H, Giona F, Amato DJ, Petakov M, Munoz ET, Solorio-Meza SE, Cooper PA, Varughese S, Chertkoff R, Brill-Almon E. Long-term efficacy and safety results of taliglucerase alfa up to 36 months in adult treatment-naive patients with Gaucher disease. Am J Hematol. 2016 Jul;91(7):656-60. doi: 10.1002/ajh.24369. Epub 2016 Apr 24.
Pastores GM, Shankar SP, Petakov M, Giraldo P, Rosenbaum H, Amato DJ, Szer J, Chertkoff R, Brill-Almon E, Zimran A. Enzyme replacement therapy with taliglucerase alfa: 36-month safety and efficacy results in adult patients with Gaucher disease previously treated with imiglucerase. Am J Hematol. 2016 Jul;91(7):661-5. doi: 10.1002/ajh.24399. Epub 2016 May 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/NCT00705939