Recent Updates
Recently added Catalysts

Taliglucerase alfa

Phase 3

Gaucher Disease | Small molecule | Rare Disease |Pfizer, Inc.|Last Updated: Apr 19, 2023

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials4
Total Enrollment110

FDA Designations

No designations recorded

Clinical trial landscape

Taliglucerase alfa · 4 trials · 1 indication

Phase 3 4
NCT01411228A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher DiseaseGaucher Disease
COMPLETED15 Analytics
NCT01422187A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher DiseaseGaucher Disease
COMPLETED19 Analytics
NCT00712348Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human GlucocerebrosidaseGaucher Disease
COMPLETED31 Analytics
NCT00705939Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension TrialGaucher Disease
COMPLETED45 Analytics
PHASE3COMPLETED
A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Gaucher DiseaseUnlock trial analytics
PHASE3COMPLETED
A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
Gaucher DiseaseUnlock trial analytics
PHASE3COMPLETED
Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase
Gaucher DiseaseUnlock trial analytics
PHASE3COMPLETED
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Gaucher DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Hemoglobin
Baseline, months 9, 12 and 24

Median and interquartile range. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.

Spleen Volume
60 months

Spleen volume measured by MRI

Secondary Endpoints

Chitotriosidase
Baseline, months 9, 12 and 24
Spleen Volume
Baseline, months 12 and 24
Platelet Count
Baseline, months 9, 12, 24 and 33-36
Unlock Study Endpoints

Study Design & Arms

AllocationNON_RANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
60 Units/kgEXPERIMENTAL -
30 Units/kgEXPERIMENTAL -
Taliglucerase alfa 30 units/kgEXPERIMENTALSubjects randomized to receive 30 units/kg
Taliglucerase alfa 60 units/kgEXPERIMENTALSubjects randomized to 60 units/kg
Taliglucerase alfaEXPERIMENTALOpen label taliglucerase alfa treatment
Naive 30 Units/kgEXPERIMENTALContinue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
Naive 60 Units/kgEXPERIMENTALContinue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
SwitchoverEXPERIMENTALContinue taliglucerase alfa treatment from PB-06-002 (NCT00712348)

Interventions

NameTypeDescription
Taliglucerase alfaDRUGTaliglucerase alfa for infusion every two weeks for 24 months
Unlock Study Design Details

Eligibility Criteria

Age Range2 Years to 18 Years
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: * Successful completion of Protocol PB-06-002 or PB-06-005 * The subject, parent(s) or legal guardian(s) signs an informed consent and/or assent Exclusion Criteria: * Currently taking another investigational drug for any condition. * Presence of neurological signs and symptoms...

Countries:IsraelParaguaySouth AfricaUnited StatesAustraliaCanadaSpainUnited KingdomChile
Unlock Eligibility Criteria

Frequently asked questions about Taliglucerase alfa

What is Taliglucerase alfa used for?

Taliglucerase alfa is an investigational enzyme replacement therapy being developed for the treatment of Gaucher Disease, a rare genetic disorder. It is currently in Phase 3 clinical development and has not yet been approved by regulatory authorities.

Who makes Taliglucerase alfa?

Taliglucerase alfa is being developed by Pfizer, Inc. (NYSE: PFE). The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Gaucher Disease.

What phase is Taliglucerase alfa in?

Taliglucerase alfa is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA or other regulatory agencies. All four clinical trials for the drug have been completed.

What clinical trials is Taliglucerase alfa in?

Taliglucerase alfa has been studied in four completed Phase 3 clinical trials. These include NCT00705939, NCT00712348, NCT01411228, and NCT01422187, which evaluated the drug in adult and pediatric patients with Gaucher Disease.

Is Taliglucerase alfa the same as imiglucerase?

Taliglucerase alfa is a distinct enzyme replacement therapy, though one clinical trial (NCT00712348) involved switching patients from imiglucerase to taliglucerase alfa. The drug is being developed as a potential treatment option for Gaucher Disease.