CompletedNCT00705939Updated Oct 4, 2018Results posted

Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

A Phase 3 interventional study of Taliglucerase alfa in Gaucher Disease, sponsored by Pfizer. Completed at 11 sites in 8 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2018-10-04.

Sponsored by Pfizer · Phase 3, Interventional, and Treatment

Phase: Phase 3
Study type: Interventional
Enrollment: 45
Allocation: Non-randomized
Ages: 18 Years and older
Sex: All

Study 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.

Read the detailed description

This will be a multi-center, double-blind, parallel group, extension trial to assess the safety and efficacy of prGCD in patients completing NCT00376168. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the extension study will be fifteen months. There will be two treatment groups: 30 units/kg every 2 weeks or 60 units/kg every 2 weeks.

Conditions studied

Gaucher Disease

Browse trials for

Gaucher Disease 171

Keywords

Gaucher Disease
Enzyme replacement therapy

In context

Gaucher Disease

171 studies on the registry are indexed under Gaucher Disease; 37 are open to participants now.

This study's enrollment of 45 is above the median of 20 across 98 interventional studies indexed under Gaucher Disease.

Browse Gaucher Disease studies →

Lead sponsor

Pfizer is the lead sponsor of 3,244 studies on the registry; 139 are open to participants now.

Of its 582 completed or terminated interventional studies of FDA-regulated products, 381 (65%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

Successful completion of Protocol PB-06-001
The patient signs informed consent

Exclusion criteria

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

Phase: Phase 3
Primary purpose: Treatment
Allocation: Non-randomized
Intervention model: Parallel assignment
Masking: None (open label)
Enrollment: 45 participants (actual)

Study arms

Experimental
Naive 30 Units/kg
Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
Drug: Taliglucerase alfa
Experimental
Naive 60 Units/kg
Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
Drug: Taliglucerase alfa
Experimental
Switchover
Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348)
Drug: Taliglucerase alfa

Interventions

DrugTaliglucerase alfa
Intravenous infusion every 2 weeks
Also known as: Plant Cell Expressed Recombinant Human Glucocerebrosidase, prGCD

What researchers measure

Primary outcomes

Spleen Volume
Spleen volume measured by MRI
Time frame: Spleen Volume at Baseline and Months 12, 24, and 36

Secondary outcomes

Liver Volume
Liver volume measured by MRI
Time frame: Liver volume at Baseline and Months 12, 24 and 36
Hemoglobin
Time frame: Hemoglobin at Baseline and Months 12, 24 and 36
Platelet Count
Time frame: Platelet count at Baseline and Months 12, 24 and 36

Other outcomes

Spleen Volume Multiples of Normal (MN)
Spleen volume measured by MRI. Normal spleen volume is 2 mL/kg × body weight (kg)
Time frame: Baseline and Months 12, 24, and 36
Liver Volume Multiples of Normal (MN)
Liver volume measured by MRI. Normal liver volume is 25 mL/kg × body weight (kg).
Time frame: Baseline and Months 12, 24 and 36

Results

Posted Jul 15, 2014

Participant flow

Patients completing Studies PB-06-001 (NCT00376168) or PB-06-002 (NCT00712348) were offered continued treatment in this study.

Participant flow — Overall Study
Milestone	Naive 30 Units/kg	Naive 60 Units/kg	Switchover
Started	12	14	18
Completed	12	12	14
Not completed	0	2	4
Withdrew: Protocol violation	0	0	1
Withdrew: Withdrawal by subject	0	1	3
Withdrew: Physician decision	0	1	0

Outcome measures

PrimarySpleen Volume

Spleen volume measured by MRI

Time frame:: Spleen Volume at Baseline and Months 12, 24, and 36
Reported as:: Mean · mL

Spleen Volume
mL	Naive 30 Units/kg	Naive 60 Units/kg	Switchover
Baseline	2324.0 ± 1209.0	2120.0 ± 1426.5	778.0 ± 666.3
Month 12	1707.7 ± 1069.5	1267.9 ± 1114.1	883.7 ± 760.0
Month 24	1420.4 ± 852.3	946.7 ± 699.6	609.1 ± 442.7
Month 36	1237.2 ± 695.7	761.0 ± 556.0	548.2 ± 433.7

SecondaryLiver Volume

Liver volume measured by MRI

Time frame:: Liver volume at Baseline and Months 12, 24 and 36
Reported as:: Mean · mL

Liver Volume
mL	Naive 30 Units/kg	Naive 60 Units/kg	Switchover
Baseline	2999.7 ± 779.4	2470.5 ± 484.9	1775.7 ± 434.4
Month 12	2515.6 ± 642.1	2118.7 ± 318.1	1788.9 ± 378.0
Month 24	2362.8 ± 518.7	1998.2 ± 291.9	1757.1 ± 357.0
Month 36	2341.1 ± 553.4	1971.8 ± 404.7	1821.2 ± 523.6

Other pre-specifiedSpleen Volume Multiples of Normal (MN)

Spleen volume measured by MRI. Normal spleen volume is 2 mL/kg × body weight (kg)

Time frame:: Baseline and Months 12, 24, and 36
Reported as:: Mean · Multiples of Normal Spleen Volume

Spleen Volume Multiples of Normal (MN)
Multiples of Normal Spleen Volume	Naive 30 Units/kg	Naive 60 Units/kg	Switchover
Baseline	16.4 ± 8.3	16.8 ± 14.2	5.5 ± 5.4
Month 12	11.7 ± 7.2	9.3 ± 9.4	7.0 ± 6.8
Month 24	9.6 ± 1.6	6.6 ± 5.3	4.0 ± 2.7
Month 36	8.2 ± 4.4	5.6 ± 4.5	3.7 ± 2.9

Other pre-specifiedLiver Volume Multiples of Normal (MN)

Liver volume measured by MRI. Normal liver volume is 25 mL/kg × body weight (kg).

Time frame:: Baseline and Months 12, 24 and 36
Reported as:: Mean · Multiples of Normal Liver Volume

Liver Volume Multiples of Normal (MN)
Multiples of Normal Liver Volume	Naive 30 Units/kg	Naive 60 Units/kg	Switchover
Baseline	1.7 ± 0.4	1.5 ± 0.4	1.0 ± 0.1
Month 12	1.4 ± 0.3	1.2 ± 0.2	1.1 ± 0.2
Month 24	1.3 ± 0.2	1.1 ± 0.2	0.9 ± 0.2
Month 36	1.3 ± 0.2	1.1 ± 0.2	1.0 ± 0.3

SecondaryHemoglobin

Time frame:: Hemoglobin at Baseline and Months 12, 24 and 36
Reported as:: Mean · mg/dL

Hemoglobin
mg/dL	Naive 30 Units/kg	Naive 60 Units/kg	Switchover
Baseline	12.5 ± 1.8	11.4 ± 2.7	13.6 ± 1.6
Month 12	14.2 ± 1.7	13.6 ± 2.6	13.6 ± 1.7
Month 24	13.8 ± 1.6	13.8 ± 1.8	13.5 ± 1.2
Month 36	14.3 ± 1.5	14.1 ± 2.2	13.3 ± 1.2

SecondaryPlatelet Count

Time frame:: Platelet count at Baseline and Months 12, 24 and 36
Reported as:: Mean · Platelets per cubic millimeter

Platelet Count
Platelets per cubic millimeter	Naive 30 Units/kg	Naive 60 Units/kg	Switchover
Baseline	64900 ± 30133	69043 ± 28242	163833 ± 88882
Month 12	80325 ± 41806	122857 ± 53857	145250 ± 97426
Month 24	93333 ± 53328	141071 ± 73896	174200 ± 100483
Month 36	94683 ± 47526	144417 ± 55190	172467 ± 89340

Adverse events

Collected over 36 months. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Naive 30 Units/kg	—	2/12 (16.7%)	12/12 (100%)
Naive 60 Units/kg	—	2/14 (14.3%)	12/14 (85.7%)
Switchover	—	3/18 (16.7%)	17/18 (94.4%)

Most frequent serious events

Showing 10 of 11

Most frequent serious events
Event	Naive 30 Units/kg	Naive 60 Units/kg	Switchover
HAEMANGIOMANeoplasms benign, malignant and unspecified (incl cysts and polyps)	1/12	0/14	0/18
PULMONARY EMBOLISMRespiratory, thoracic and mediastinal disorders	1/12	0/14	0/18
TONSILLECTOMYSurgical and medical procedures	1/12	0/14	0/18
TOOTH EXTRACTIONSurgical and medical procedures	1/12	0/14	0/18
VOCAL CORD POLYPECTOMYSurgical and medical procedures	1/12	0/14	0/18
AUTOIMMUNE THROMBOCYTOPENIABlood and lymphatic system disorders	0/12	1/14	0/18
HEAD INJURYInjury, poisoning and procedural complications	0/12	1/14	0/18
OSTEONECROSISMusculoskeletal and connective tissue disorders	0/12	1/14	0/18
ARTHRALGIAMusculoskeletal and connective tissue disorders	0/12	0/14	1/18
PNEUMOTHORAX TRAUMATICInjury, poisoning and procedural complications	0/12	0/14	1/18

Most frequent other events

Showing 10 of 25

Most frequent other events
Event	Naive 30 Units/kg	Naive 60 Units/kg	Switchover
NASOPHARYNGITISInfections and infestations	2/12	3/14	7/18
ARTHRALGIAMusculoskeletal and connective tissue disorders	4/12	4/14	4/18
HEADACHENervous system disorders	4/12	3/14	2/18
PAIN IN EXTREMITYMusculoskeletal and connective tissue disorders	2/12	4/14	2/18
DIARRHOEAGastrointestinal disorders	3/12	0/14	3/18
ERYTHEMASkin and subcutaneous tissue disorders	3/12	0/14	0/18
UPPER RESPIRATORY TRACT INFECTIONInfections and infestations	3/12	3/14	3/18
PYREXIAGeneral disorders	0/12	3/14	4/18
HYPERTENSIONVascular disorders	2/12	3/14	1/18
SINUSITISInfections and infestations	0/12	3/14	2/18

Baseline characteristics

All treated patients are included in the analysis

Age, Continuous
Age, Continuous(years)	Naive 30 Units/kg	Naive 60 Units/kg	Switchover	Total
Mean	39.7 ± 11.8	36.6 ± 12.0	46.5 ± 13.7	41.6 ± 13.0

Sex: Female, Male
Sex: Female, Male(Participants)	Naive 30 Units/kg	Naive 60 Units/kg	Switchover	Total
Female	5	8	9	22
Male	7	6	9	22

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	Naive 30 Units/kg	Naive 60 Units/kg	Switchover	Total
American Indian or Alaska Native	0	0	0	0
Asian	0	0	0	0
Native Hawaiian or Other Pacific Islander	0	0	0	0
Black or African American	0	0	0	0
White	12	13	18	43
More than one race	0	0	0	0
Unknown or Not Reported	0	1	0	1

Region of Enrollment
Region of Enrollment(participants)	Naive 30 Units/kg	Naive 60 Units/kg	Switchover	Total
United States	0	0	6	6
Canada	1	1	1	3
Spain	0	1	2	3
Australia	0	0	1	1
Chile	1	1	0	2
South Africa	1	1	0	2
Israel	3	4	6	13
United Kingdom	0	1	0	1
Italy	1	1	0	2
Serbia	2	2	2	6
Mexico	3	2	0	5

Study locations

11 sites

Department of Human Genetics, Emory University School of Medicine
Decatur, Georgia 30033, United States
Neurogenetics, NYU at Rivergate
New York, New York 10016, United States
Bone Marrow Transplant Service, The Royal Melbourne Hospital
Parkville, Victoria, Australia
Mount Sinai Hospital
Toronto, Ontario M5G 1X5, Canada
Pontificia Universidad Catolica de Chile
Santiago, Chile
Rambam Medical Center
Haifa, 31096, Israel
Shaare Zedek Medical Center
Jerusalem, Israel
Morningside Medi-Clinic
Morningside, 2196, South Africa
Hospital Universitario Miguel Servet
Zaragoza, 50009, Spain
Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust
Cambridge, United Kingdom
Royal Free Hospital
London, NW3 2QG, United Kingdom

References and documents

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. PubMed 27174694 ↗
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. PubMed 27102949 ↗

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Oct 4, 2018, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT00705939
Lead sponsor: Pfizer
Responsible party: Sponsor
First posted: Jun 27, 2008
Start date: Jun 2008
Primary completion: May 2012
Completion: Aug 2013
Results posted: Jul 15, 2014
Last update: Oct 4, 2018

Oversight

Data monitoring committee: Yes

View the source record on ClinicalTrials.gov ↗

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