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Trial record 28 of 102 for:    Gaucher Disease

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

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ClinicalTrials.gov Identifier: NCT01842841
Recruitment Status : Completed
First Posted : April 30, 2013
Results First Posted : December 14, 2015
Last Update Posted : December 14, 2015
Sponsor:
Collaborator:
Quintiles, Inc.
Information provided by (Responsible Party):
Shire

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Gaucher Disease
Intervention: Drug: velaglucerase alfa

  Participant Flow

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
No text entered.

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

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]
 5 
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   1 
Male   4 


  Outcome Measures

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) ]

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) ]

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) ]

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

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

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

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

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

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

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

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

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

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

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

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

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


  Serious Adverse Events


  Other Adverse Events


  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.


  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:
unchecked 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.
unchecked 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.


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):

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01842841     History of Changes
Other Study ID Numbers: HGT-GCB-091
First Submitted: April 11, 2013
First Posted: April 30, 2013
Results First Submitted: September 28, 2015
Results First Posted: December 14, 2015
Last Update Posted: December 14, 2015