Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease

This study has been completed.

Sponsor:

Collaborator:

Quintiles, Inc.

Information provided by (Responsible Party):

Shire

ClinicalTrials.gov Identifier:

NCT01842841

First received: April 11, 2013

Last updated: November 9, 2015

Last verified: September 2015

History of Changes

Results First Received: September 28, 2015

Study Type:	Interventional
Study Design:	Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Treatment
Condition:	Gaucher Disease
Intervention:	Drug: velaglucerase alfa

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
Participants from study HGT-GCB-087 (NCT01614574) were enrolled in this study except one participant who was not enrolled due to personal reasons.

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
Velaglucerase Alfa (VPRIV®)	Velaglucerase alfa (VPRIV®) 15 to 60 units per kilogram (U/kg), every other week (EOW) administered as an intravenous (IV) infusion over 60 minutes from Week 53 (2 weeks after the last infusion [Week 51] in Study HGT-GCB-087 [NCT01614574]) to Week 155.

Participant Flow: Overall Study

	Velaglucerase Alfa (VPRIV®)
STARTED	5
COMPLETED	5
NOT COMPLETED	0

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.
Safety population included all enrolled participants who received at least 1 investigational product infusion (full or partial).

Reporting Groups

	Description
Velaglucerase Alfa (VPRIV®)	Velaglucerase alfa (VPRIV®) 15 to 60 U/kg, EOW, administered as an IV infusion over 60 minutes from Week 53 (2 weeks after the last infusion [Week 51] in Study HGT-GCB-087 [NCT01614574]) to Week 155.

Baseline Measures

Velaglucerase Alfa (VPRIV®)

Overall Participants Analyzed
[Units: Participants]

Age ^[1]
[Units: Years]
Mean (Standard Deviation)

22.20 (11.946)

^[1]	Age at the time of first dose in the current study (Week 53)

Gender
[Units: Participants]

Female

Male

Outcome Measures

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1. Primary:

Number of Participants With Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs) [ Time Frame: From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks) ]

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2. Primary:

Number of Participants Using Concomitant Medication [ Time Frame: From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks) ]

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3. Primary:

Number of Participants With Abnormal and Clinically Significant Laboratory Test Results [ Time Frame: From Week 65 until the end of study (Week 155) ]

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4. Primary:

Number of Participants With Positive Anti-Velaglucerase Alfa Antibodies [ Time Frame: From Week 65 until the end of study (Week 155) ]

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5. Secondary:

Change From Baseline in Hemoglobin Concentration at Week 101 [ Time Frame: Baseline, Week 101 ]

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6. Secondary:

Change From Baseline in Platelet Count at Week 101 [ Time Frame: Baseline, Week 101 ]

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7. Secondary:

Change From Baseline in Liver Volume Normalized to Body Weight at Week 103 [ Time Frame: Baseline, Week 103 ]

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8. Secondary:

Change From Baseline in Spleen Volume Normalized to Body Weight at Week 103 [ Time Frame: Baseline, Week 103 ]

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9. Secondary:

Change From Baseline in Bone Mineral Density (BMD) at Week 103: Z Score [ Time Frame: Baseline, Week 103 ]

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10. Secondary:

Change From Baseline in Bone Mineral Density (BMD) at Week 103: T-Score [ Time Frame: Baseline, Week 103 ]

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11. Secondary:

Change From Baseline in Bone Marrow Burden (BMB) Score at Week 103 [ Time Frame: Baseline, Week 103 ]

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12. Secondary:

Change From Baseline in Growth Velocity at Week 101 : Height Z-Score [ Time Frame: Baseline, Week 101 ]

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13. Secondary:

Change From Baseline in Skeletal Age at Week 103: Z-Score [ Time Frame: Baseline, Week 103 ]

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14. Secondary:

Change From Baseline in Plasma Chitotriosidase Levels at Week 101 [ Time Frame: Baseline, Week 101 ]

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15. Secondary:

Number of Participants With Change From Baseline in Neurological Status at Week 103 [ Time Frame: Baseline, Week 103 ]

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16. Secondary:

Change From Baseline in Chemokine [C-C Motif] Ligand 18 (CCL18) Levels at Week 101 [ Time Frame: Baseline, Week 101 ]

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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 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: If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.

Results Point of Contact:

Name/Title: Study Physician
Organization: Shire
phone: 1 866-842-5335

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

Ida H, Tanaka A, Matsubayashi T, Murayama K, Hongo T, Lee HM, Mellgard B. A multicenter, open-label extension study of velaglucerase alfa in Japanese patients with Gaucher disease: Results after a cumulative treatment period of 24months. Blood Cells Mol Dis. 2016 Jul;59:140-7. doi: 10.1016/j.bcmd.2015.10.002. Epub 2015 Oct 16.

Responsible Party:	Shire
ClinicalTrials.gov Identifier:	NCT01842841 History of Changes
Other Study ID Numbers:	HGT-GCB-091
Study First Received:	April 11, 2013
Results First Received:	September 28, 2015
Last Updated:	November 9, 2015

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