A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01422187

Enrollment

Registered

2011-08-23

Start date

2011-08-31

Completion date

2014-09-30

Last updated

2023-04-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Gaucher Disease

Keywords

enzyme replacement therapy

Brief summary

This is a multi-center trial to further extend the assessment of the safety and efficacy of taliglucerase alfa in adult subjects (≥18 years old) with Gaucher disease who have enrolled in Protocol PB-06-003. Subjects will continue to receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The duration of treatment will be a maximum of 21 months or until taliglucerase alfa is commercially available to the subject at the discretion of the Sponsor.

Interventions

DRUGTaliglucerase alfa

Taliglucerase infusion every two weeks for 21 months

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

* Successfully completed Protocol PB-06-001 and enrolled in Protocol PB-06-003 * The subject signs an informed consent

Exclusion criteria

* Currently taking another investigational drug for any condition. * Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.

Design outcomes

Primary

Measure	Time frame	Description
Spleen Volume	60 months	Spleen volume measured by MRI

Secondary

Measure	Time frame	Description
Liver Volume	60 months	Liver volume by MRI
Platelet Count	60 months	Platelet count measure annually
Hemoglobin	60 months	Hemoglobin measure yearly

Participant flow

Participants by arm

Arm	Count
Taliglucerase Alfa 30 Units/kg Subjects randomized to receive 30 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	8
Taliglucerase Alfa 60 Units/kg Subjects randomized to 60 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	9
Dose Adjusted Subjects randomized to 30 units/kg but with dose increased Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	2
Total	19

Withdrawals & dropouts

Period	Reason	FG000	FG001	FG002
Overall Study	Lost to Follow-up	0	1	0
Overall Study	Personal reason	1	0	0

Baseline characteristics

Characteristic	Taliglucerase Alfa 30 Units/kg	Taliglucerase Alfa 60 Units/kg	Dose Adjusted	Total
Age, Continuous	44.8 years STANDARD_DEVIATION 13.4	39.9 years STANDARD_DEVIATION 12	38.5 years STANDARD_DEVIATION 0.7	41.8 years STANDARD_DEVIATION 11.8
Race (NIH/OMB) American Indian or Alaska Native	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Asian	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Black or African American	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) More than one race	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) White	8 Participants	9 Participants	2 Participants	19 Participants
Region of Enrollment Chile	0 participants	1 participants	1 participants	2 participants
Region of Enrollment Israel	2 participants	2 participants	0 participants	4 participants
Region of Enrollment Italy	0 participants	1 participants	0 participants	1 participants
Region of Enrollment Mexico	3 participants	2 participants	0 participants	5 participants
Region of Enrollment Serbia	2 participants	2 participants	0 participants	4 participants
Region of Enrollment South Africa	0 participants	0 participants	1 participants	1 participants
Region of Enrollment Spain	1 participants	1 participants	0 participants	2 participants
Sex: Female, Male Female	4 Participants	3 Participants	1 Participants	8 Participants
Sex: Female, Male Male	4 Participants	6 Participants	1 Participants	11 Participants

Adverse events

Event type	EG000 affected / at risk	EG001 affected / at risk	EG002 affected / at risk
deaths Total, all-cause mortality	— / —	— / —	— / —
other Total, other adverse events	6 / 8	8 / 9	1 / 2
serious Total, serious adverse events	0 / 8	2 / 9	0 / 2

Outcome results

Primary

Spleen Volume

Spleen volume measured by MRI

Time frame: 60 months

Arm	Measure	Group	Value (MEAN)	Dispersion
Taliglucerase Alfa 30 Units/kg	Spleen Volume	Baseline	2032.0 Milliliters	Standard Error 454.9
Taliglucerase Alfa 30 Units/kg	Spleen Volume	Month 12	1522.6 Milliliters	Standard Error 429.6
Taliglucerase Alfa 30 Units/kg	Spleen Volume	Month 24	1193.0 Milliliters	Standard Error 320.2
Taliglucerase Alfa 30 Units/kg	Spleen Volume	Month 36	1068.7 Milliliters	Standard Error 264.6
Taliglucerase Alfa 30 Units/kg	Spleen Volume	Month 48	1137.4 Milliliters	Standard Error 314.6
Taliglucerase Alfa 30 Units/kg	Spleen Volume	Month 60	1019.2 Milliliters	Standard Error 285.3
Taliglucerase Alfa 60 Units/kg	Spleen Volume	Month 60	516.1 Milliliters	Standard Error 54.5
Taliglucerase Alfa 60 Units/kg	Spleen Volume	Baseline	1960.0 Milliliters	Standard Error 451.7
Taliglucerase Alfa 60 Units/kg	Spleen Volume	Month 36	634.0 Milliliters	Standard Error 71
Taliglucerase Alfa 60 Units/kg	Spleen Volume	Month 48	526.8 Milliliters	Standard Error 48.5
Taliglucerase Alfa 60 Units/kg	Spleen Volume	Month 12	1063.7 Milliliters	Standard Error 280.7
Taliglucerase Alfa 60 Units/kg	Spleen Volume	Month 24	744.9 Milliliters	Standard Error 130.6
Dose Adjusted	Spleen Volume	Month 12	1677.9 Milliliters	Standard Error 609.3
Dose Adjusted	Spleen Volume	Month 24	1536.3 Milliliters	Standard Error 506.7
Dose Adjusted	Spleen Volume	Month 60	919.9 Milliliters	Standard Error 180.9
Dose Adjusted	Spleen Volume	Month 36	1256.6 Milliliters	Standard Error 385
Dose Adjusted	Spleen Volume	Baseline	2321.7 Milliliters	Standard Error 679.6
Dose Adjusted	Spleen Volume	Month 48	1070.0 Milliliters	Standard Error 280.6

Secondary

Hemoglobin

Hemoglobin measure yearly

Time frame: 60 months

Arm	Measure	Group	Value (MEAN)	Dispersion
Taliglucerase Alfa 30 Units/kg	Hemoglobin	Month 36	14.0 mg/dL	Standard Error 0.5
Taliglucerase Alfa 30 Units/kg	Hemoglobin	Month 12	13.9 mg/dL	Standard Error 0.7
Taliglucerase Alfa 30 Units/kg	Hemoglobin	Month 60	14.1 mg/dL	Standard Error 0.9
Taliglucerase Alfa 30 Units/kg	Hemoglobin	Month 48	13.7 mg/dL	Standard Error 0.6
Taliglucerase Alfa 30 Units/kg	Hemoglobin	Month 24	13.5 mg/dL	Standard Error 0.8
Taliglucerase Alfa 30 Units/kg	Hemoglobin	Baseline	12.3 mg/dL	Standard Error 0.7
Taliglucerase Alfa 60 Units/kg	Hemoglobin	Month 24	14.1 mg/dL	Standard Error 0.7
Taliglucerase Alfa 60 Units/kg	Hemoglobin	Month 36	14.2 mg/dL	Standard Error 0.8
Taliglucerase Alfa 60 Units/kg	Hemoglobin	Month 60	13.9 mg/dL	Standard Error 0.6
Taliglucerase Alfa 60 Units/kg	Hemoglobin	Baseline	11.6 mg/dL	Standard Error 0.9
Taliglucerase Alfa 60 Units/kg	Hemoglobin	Month 48	14.0 mg/dL	Standard Error 0.8
Taliglucerase Alfa 60 Units/kg	Hemoglobin	Month 12	14.2 mg/dL	Standard Error 0.7
Dose Adjusted	Hemoglobin	Month 60	14.3 mg/dL	Standard Error 0.1
Dose Adjusted	Hemoglobin	Month 48	14.5 mg/dL	Standard Error 0.1
Dose Adjusted	Hemoglobin	Baseline	12.5 mg/dL	Standard Error 0.9
Dose Adjusted	Hemoglobin	Month 12	14.3 mg/dL	Standard Error 0.9
Dose Adjusted	Hemoglobin	Month 24	14.2 mg/dL	Standard Error 0.2
Dose Adjusted	Hemoglobin	Month 36	14.4 mg/dL	Standard Error 0.1

Secondary

Liver Volume

Liver volume by MRI

Time frame: 60 months

Arm	Measure	Group	Value (MEAN)	Dispersion
Taliglucerase Alfa 30 Units/kg	Liver Volume	Month 12	2619.3 Milliliters	Standard Error 266.4
Taliglucerase Alfa 30 Units/kg	Liver Volume	Baseline	3103.4 Milliliters	Standard Error 324.7
Taliglucerase Alfa 30 Units/kg	Liver Volume	Month 36	2429.6 Milliliters	Standard Error 229.6
Taliglucerase Alfa 30 Units/kg	Liver Volume	Month 60	2290.9 Milliliters	Standard Error 233.6
Taliglucerase Alfa 30 Units/kg	Liver Volume	Month 48	2429.1 Milliliters	Standard Error 283.2
Taliglucerase Alfa 30 Units/kg	Liver Volume	Month 24	2367.9 Milliliters	Standard Error 212.8
Taliglucerase Alfa 60 Units/kg	Liver Volume	Baseline	2536.1 Milliliters	Standard Error 170
Taliglucerase Alfa 60 Units/kg	Liver Volume	Month 12	2155.2 Milliliters	Standard Error 104.8
Taliglucerase Alfa 60 Units/kg	Liver Volume	Month 24	1939.5 Milliliters	Standard Error 101.5
Taliglucerase Alfa 60 Units/kg	Liver Volume	Month 36	2010.6 Milliliters	Standard Error 143.5
Taliglucerase Alfa 60 Units/kg	Liver Volume	Month 48	1971.2 Milliliters	Standard Error 179.3
Taliglucerase Alfa 60 Units/kg	Liver Volume	Month 60	2067.7 Milliliters	Standard Error 188.9
Dose Adjusted	Liver Volume	Month 12	2048.0 Milliliters	Standard Error 87.8
Dose Adjusted	Liver Volume	Month 60	1820.8 Milliliters	Standard Error 196.5
Dose Adjusted	Liver Volume	Month 48	1792.2 Milliliters	Standard Error 143.6
Dose Adjusted	Liver Volume	Baseline	2488.6 Milliliters	Standard Error 206.2
Dose Adjusted	Liver Volume	Month 24	2064.0 Milliliters	Standard Error 202.5
Dose Adjusted	Liver Volume	Month 36	1959.0 Milliliters	Standard Error 140.5

Secondary

Platelet Count

Platelet count measure annually

Time frame: 60 months

Arm	Measure	Group	Value (MEAN)	Dispersion
Taliglucerase Alfa 30 Units/kg	Platelet Count	Baseline	70975 platelets/mm^3	Standard Error 8700.6
Taliglucerase Alfa 30 Units/kg	Platelet Count	Month 12	86613 platelets/mm^3	Standard Error 11894
Taliglucerase Alfa 30 Units/kg	Platelet Count	Month 24	103250 platelets/mm^3	Standard Error 17008
Taliglucerase Alfa 30 Units/kg	Platelet Count	Month 36	106775 platelets/mm^3	Standard Error 13517
Taliglucerase Alfa 30 Units/kg	Platelet Count	Month 48	100643 platelets/mm^3	Standard Error 15314
Taliglucerase Alfa 30 Units/kg	Platelet Count	Month 60	104986 platelets/mm^3	Standard Error 18961
Taliglucerase Alfa 60 Units/kg	Platelet Count	Month 60	180625 platelets/mm^3	Standard Error 17062
Taliglucerase Alfa 60 Units/kg	Platelet Count	Baseline	70067 platelets/mm^3	Standard Error 7695.5
Taliglucerase Alfa 60 Units/kg	Platelet Count	Month 36	155667 platelets/mm^3	Standard Error 16060
Taliglucerase Alfa 60 Units/kg	Platelet Count	Month 48	184000 platelets/mm^3	Standard Error 16688
Taliglucerase Alfa 60 Units/kg	Platelet Count	Month 12	137556 platelets/mm^3	Standard Error 18358
Taliglucerase Alfa 60 Units/kg	Platelet Count	Month 24	168778 platelets/mm^3	Standard Error 23717
Dose Adjusted	Platelet Count	Month 12	88000 platelets/mm^3	Standard Error 65000
Dose Adjusted	Platelet Count	Month 24	101000 platelets/mm^3	Standard Error 68000
Dose Adjusted	Platelet Count	Month 60	103500 platelets/mm^3	Standard Error 60500
Dose Adjusted	Platelet Count	Month 36	94500 platelets/mm^3	Standard Error 64500
Dose Adjusted	Platelet Count	Baseline	69500 platelets/mm^3	Standard Error 42500
Dose Adjusted	Platelet Count	Month 48	103000 platelets/mm^3	Standard Error 53000

Source: ClinicalTrials.gov · Data processed: Mar 13, 2026

A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease

Conditions

Keywords

Brief summary

Related papers

Interventions

Sponsors

Study design

Eligibility

Inclusion criteria

Exclusion criteria

Design outcomes

Primary

Secondary

Participant flow

Participants by arm

Withdrawals & dropouts

Baseline characteristics

Adverse events

Outcome results

Spleen Volume

Hemoglobin

Liver Volume

Platelet Count