A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Protalix

ClinicalTrials.gov Identifier:

NCT00376168

First received: September 12, 2006

Last updated: July 25, 2012

Last verified: July 2012

History of Changes

Results First Received: May 11, 2012

Study Type:	Interventional
Study Design:	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition:	Gaucher Disease
Intervention:	Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD)

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
ELELYSO 30 Units/kg	Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks
ELELYSO 60 Units/kg	Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks

Participant Flow: Overall Study

	ELELYSO 30 Units/kg	ELELYSO 60 Units/kg
STARTED	16	16 ^[1]
COMPLETED	14	15
NOT COMPLETED	2	1
Adverse Event	1	1
Pregnancy	1	0

^[1]	One patient randomized but not treated

Baseline Characteristics

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Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups

	Description
ELELYSO 30 Units/kg	Taliglucerase alfa 30 Units/kg by intravenous infusion every two weeks
ELELYSO 60 Units/kg	Taliglucerase alfa 60 Units/kg by intravenous infusion every two weeks
Total	Total of all reporting groups

Baseline Measures

ELELYSO 30 Units/kg

ELELYSO 60 Units/kg

Total

Overall Participants Analyzed
[Units: Participants]

Age
[Units: Participants]

<=18 years

Between 18 and 65 years

>=65 years

Age
[Units: Years]
Mean (Standard Deviation)

36.3 (11.8)

36.0 (12.2)

36.2 (11.8)

Gender
[Units: Participants]

Female

Male

Region of Enrollment
[Units: Participants]

Canada

Spain

Chile

South Africa

Israel

Italy

United Kingdom

Mexico

Serbia

Spleen Volume ^[1]
[Units: mL]
Mean (Standard Deviation)

2130.94 (1154.72)

2117.38 (1356.17)

2120 (1200)

^[1]	Measured by MRI

Liver Volume ^[1]
[Units: mL]
Mean (Standard Deviation)

2880.60 (736.12)

2481.31 (452.74)

2600 (600)

^[1]	Measured by MRI

Hemoglobin ^[1]
[Units: g/dL]
Mean (Standard Deviation)

12.2 (1.7)

11.4 (2.6)

12 (2)

^[1]	Measured in central laboratory

Platelet Count ^[1]
[Units: Count/mm^3]
Mean (Standard Deviation)

75,320 (40,861)

65,038 (28,668)

70,000 (32,000)

^[1]	Measured in local laboratory

Chitotriosidase ^[1]
[Units: Nmol/ml/hr]
Mean (Standard Deviation)

28158 (11686)

24702 (17428)

26000 (14000)

^[1]	Gaucher disease biomarker

Outcome Measures

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1. Primary:

Change From Baseline in Spleen Volume Measured by MRI. [ Time Frame: Baseline and 9 months ]

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2. Secondary:

Change From Baseline in Liver Volume [ Time Frame: Baseline and 9 months ]

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3. Secondary:

Change in Hemoglobin [ Time Frame: Baseline and Month 9 ]

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4. Secondary:

Change in Platelet Count [ Time Frame: Baseline and Month 9 ]

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5. Other Pre-specified:

Change in Chitotriosidase [ Time Frame: Baseline and Month 9 ]

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Serious Adverse Events

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Other Adverse Events

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Limitations and Caveats

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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact:

Name/Title: Einat Almon
Organization: Protalix Ltd.
phone: 972-4-9889488 ext 137
e-mail: einata@protalix.com

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Abbas R, Park G, Damle B, Chertkoff R, Alon S. Pharmacokinetics of Novel Plant Cell-Expressed Taliglucerase Alfa in Adult and Pediatric Patients with Gaucher Disease. PLoS One. 2015 Jun 8;10(6):e0128986. doi: 10.1371/journal.pone.0128986. eCollection 2015.

Zimran A, Brill-Almon E, Chertkoff R, Petakov M, Blanco-Favela F, Muñoz ET, Solorio-Meza SE, Amato D, Duran G, Giona F, Heitner R, Rosenbaum H, Giraldo P, Mehta A, Park G, Phillips M, Elstein D, Altarescu G, Szleifer M, Hashmueli S, Aviezer D. Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease. Blood. 2011 Nov 24;118(22):5767-73. doi: 10.1182/blood-2011-07-366955. Epub 2011 Sep 6. Erratum in: Blood. 2012 May 10;119(19):4577.

Winckler T. [Enzyme replacement therapy for Gaucher's Disease]. Pharm Unserer Zeit. 2008;37(5):352-3. doi: 10.1002/pauz.200890067. German.

Responsible Party:	Protalix
ClinicalTrials.gov Identifier:	NCT00376168 History of Changes
Other Study ID Numbers:	PB-06-001
Study First Received:	September 12, 2006
Results First Received:	May 11, 2012
Last Updated:	July 25, 2012

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