Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

This study has been completed.

Study NCT00478647 Information provided by Shire Human Genetic Therapies, Inc.

First Received on May 23, 2007. Last Updated on September 20, 2010 History of Changes

Results First Received: August 4, 2010

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Treatment
Condition:	Gaucher Disease, Type 1
Intervention:	Biological: GA-GCB (velaglucerase alfa)

Participant Flow

Hide Participant Flow

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
The first patient consented to participate in the study on 13 July 2007 and the last patient enrolled on 10 June 2008.Patients received the same dose of GA-GCB (velaglucerase alfa) as their previous dose of imiglucerase (range-</= 60 Ukg->/=15 U/kg) every other week via intravenous infusion.

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Patients at least 2 years old with documented diagnosis of type 1 Gaucher disease.Consistent treatment(every other week at a dose ≤/= 60 U/kg and ≥/= 15 U/kg) with imiglucerase for a minimum of 30 consecutive months; same dose during the 6 months prior to study enrollment.Minor dosing interval variance was allowed per standard clinical practice.

Reporting Groups

	Description
GA-GCB (Velaglucerase Alfa)	15-60 U/kg, every other week via intravenous infusion

Participant Flow: Overall Study

	GA-GCB (Velaglucerase Alfa)
STARTED	40 ^[1]
COMPLETED	38 ^[2]
NOT COMPLETED	2

[1]	Reasons not completed-1 patient withdrew due to serious adverse event and 1 pts withdrew consent
[2]	ITT patient population

Baseline Characteristics

Hide Baseline Characteristics

Reporting Groups

	Description
GA-GCB (Velaglucerase Alfa)	15-60 U/kg, every other week via intravenous infusion

Baseline Measures

	GA-GCB (Velaglucerase Alfa)
Number of Participants [units: participants]	40
Age [units: participants]
<=18 years	9
Between 18 and 65 years	31
>=65 years	0
Age [units: years] Mean ± Standard Deviation	35.6 ± 18.37
Gender [units: participants]
Female	22
Male	18
Region of Enrollment [units: participants]
United States	22
Spain	1
Poland	5
Israel	9
United Kingdom	3
Baseline hemoglobin concentration [units: g/dL] Median ( Full Range )	13.775 ( 10.40 to 16.45 )
Baseline liver volume [units: Percent (%) body weight] Median ( Full Range )	1.90 ( 1.4 to 3.9 )
Baseline platelet count [units: *10^9/L] Median ( Full Range )	162.00 ( 29.0 to 399.0 )
Baseline spleen volume [units: Percent (%) body weight] Median ( Full Range )	0.50 ( 0.2 to 3.2 )

Outcome Measures

Show All Outcome Measures

1. Primary:

Patients Which Experienced at Least One Adverse Event [ Time Frame: Week 53 ]

Show Outcome Measure 1

2. Secondary:

Change From Baseline to Week 53 in Hemoglobin Concentration [ Time Frame: Week 53 ]

Show Outcome Measure 2

3. Secondary:

Percent Change From Baseline to Week 53 in Platelet Count [ Time Frame: Week 53 ]

Show Outcome Measure 3

4. Secondary:

Percent Change From Baseline to Week 51 in Normalized Liver Volume [ Time Frame: Week 51 ]

Show Outcome Measure 4

5. Secondary:

Percent Change From Baseline to Week 51 in Normalized Spleen Volume [ Time Frame: Week 51 ]

Show Outcome Measure 5

Serious Adverse Events

Show Serious Adverse Events

Other Adverse Events

Show Other Adverse Events

More Information

Hide More Information

Certain Agreements:

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

There IS 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.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: Shire's agreements with investigators vary. All agreements provide Shire the right to embargo communications regarding trial results prior to public release for a period ≤180 days from the time submitted to Shire for review. Shire does not prohibit publication, but can require the removal of confidential information (excluding trial results) and can request postponement of a single-center publication until after disclosure of the trial's multi-center publication.

Limitations and Caveats

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
No text entered.

Results Point of Contact:

Name/Title: Tiffany Crump
Organization: Shire HGT
phone: 484-595-8850
e-mail: tcrump@shire.com

No publications provided

Responsible Party:	Tiffany Crump, Senior Medical Affairs Associate, Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier:	NCT00478647 History of Changes
Other Study ID Numbers:	TKT034
Study First Received:	May 23, 2007
Results First Received:	August 4, 2010
Last Updated:	September 20, 2010
Health Authority:	United States: Food and Drug Administration; Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products; Israel: Ministry of Health; United Kingdom: Medicines and Healthcare Products Regulatory Agency; Spain: Spanish Agency of Medicines