Recent Updates
Recently added Catalysts

Plant cell expressed recombinant glucocerebrosidase

Phase 3

Gaucher Disease | Small molecule | Rare Disease |Pfizer, Inc.|Last Updated: Oct 4, 2018

Target and mechanism

ModalitySmall molecule

Also known as Plant cell expressed recombinant glucocerebrosidase (prGCD)

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials1
Total Enrollment32

FDA Designations

No designations recorded

Clinical trial landscape

Plant cell expressed recombinant glucocerebrosidase · 1 trial · 1 indication

Phase 3 1
NCT00376168A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher DiseaseGaucher Disease
COMPLETED32 Analytics
PHASE3COMPLETED
A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Gaucher Disease
NCT0037616832
Unlock trial analytics

Study Endpoints

Primary Endpoints

Change From Baseline in Spleen Volume Measured by MRI.
Baseline and 9 months

Calculated as percent change in spleen volume from Baseline to 9 months

Secondary Endpoints

Change From Baseline in Liver Volume
Baseline and 9 months
Change in Hemoglobin
Baseline and Month 9
Change in Platelet Count
Baseline and Month 9
Unlock Study Endpoints

Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
prGCD 30 Units/kgEXPERIMENTAL -
prGCD 60 Units/kgEXPERIMENTAL -

Interventions

NameTypeDescription
Plant cell expressed recombinant glucocerebrosidase (prGCD)DRUGIntravenous infusion every two weeks for 9 months
Unlock Study Design Details

Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites11

Inclusion Criteria: * Males and females, 18 years or older * Confirmed enzymatic diagnosis of Gaucher disease * Splenomegaly defined as greater than eight times the expected volume (measured volume divided by estimated volume (0.2% of body weight)\] as determined by MRI volumetric analysis * Female...

Countries:United StatesCanadaChileIsraelItalySouth AfricaSpainUnited Kingdom
Unlock Eligibility Criteria