ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
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
Enrollment 5
Recruitment Details 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  
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 5
Completed 5
Not Completed 0
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 5
Hide Baseline Analysis Population Description
Safety population included all enrolled participants who received at least 1 investigational product infusion (full or partial).
Age, Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 5 participants
22.20  (11.946)
[1]
Measure Description: Age at the time of first dose in the current study (Week 53)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
Female
1
  20.0%
Male
4
  80.0%
1.Primary Outcome
Title Number of Participants With Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs)
Hide Description An AE was any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered related to investigational product. A SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. An infusion-related AE was defined as an AE that started either during or within 12 hours after the start of the infusion and that was judged as possibly or probably related to investigational product.
Time Frame From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety population.
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: participants
Study drug-related AEs 2
Infusion-related AEs 0
Serious AEs 2
2.Primary Outcome
Title Number of Participants Using Concomitant Medication
Hide Description [Not Specified]
Time Frame From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety population.
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: participants
5
3.Primary Outcome
Title Number of Participants With Abnormal and Clinically Significant Laboratory Test Results
Hide Description Laboratory test results were considered abnormal and clinically significant at the discretion of the investigator.
Time Frame From Week 65 until the end of study (Week 155)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety population.
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: participants
1
4.Primary Outcome
Title Number of Participants With Positive Anti-Velaglucerase Alfa Antibodies
Hide Description Serum samples were collected for all participants for determination of anti-velaglucerase alfa antibodies every 12 weeks.
Time Frame From Week 65 until the end of study (Week 155)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety population.
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: participants
0
5.Secondary Outcome
Title Change From Baseline in Hemoglobin Concentration at Week 101
Hide Description Baseline was the modified baseline hemoglobin concentration, the average of the values from screening, baseline, and Week 1 Day 1 from Study HGT-GCB-087 (NCT01614574).
Time Frame Baseline, Week 101
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety population
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: gram per deciliter (g/dL)
0.22  (0.817)
6.Secondary Outcome
Title Change From Baseline in Platelet Count at Week 101
Hide Description Baseline was the modified baseline platelet count, the average of the values from screening, baseline, and Week 1 Day 1 from Study HGT-GCB-087 (NCT01614574).
Time Frame Baseline, Week 101
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety population
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: *10^9 platelets per liter
9.8  (13.14)
7.Secondary Outcome
Title Change From Baseline in Liver Volume Normalized to Body Weight at Week 103
Hide Description Liver volume was measured using magnetic resonance imaging (MRI). Liver volume measurements were normalized to the percentage of body weight. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint.
Time Frame Baseline, Week 103
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety population
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Percentage of body weight
0.01  (0.207)
8.Secondary Outcome
Title Change From Baseline in Spleen Volume Normalized to Body Weight at Week 103
Hide Description Spleen volume was measured using MRI. Spleen volume measurements were normalized to the percentage of body weight. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint.
Time Frame Baseline, Week 103
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety population
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: Percentage of body weight
0.04  (0.089)
9.Secondary Outcome
Title Change From Baseline in Bone Mineral Density (BMD) at Week 103: Z Score
Hide Description BMD was measured by dual energy x-ray absorptiometry (DXA) for lumbar spine and femurs. To ensure standardization and allow for comparisons of BMD, results were converted to standardized Z-scores (matched for age and gender). Z-scores express the BMD as the number of standard deviations (SDs) above or below the average BMD of a healthy participant of the same age and gender. Statistical analysis plan only required summarization if greater than (>) 50 percent (%) of participants had evaluable data. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint.
Time Frame Baseline, Week 103
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data was not reported as there were lesser than (<) 50% of participants with evaluable data.
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
10.Secondary Outcome
Title Change From Baseline in Bone Mineral Density (BMD) at Week 103: T-Score
Hide Description BMD was measured by DXA for lumbar spine and femurs. To ensure standardization and allow for comparisons of BMD, results were converted to standardized T-scores; normal values were used from databases from Hologic based on standard criteria. T-scores are the number of SDs above or below the average for a young adult at peak BMD. Statistical analysis plan only required summarization if >50% of participants had evaluable data. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint.
Time Frame Baseline, Week 103
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data was not reported as there were <50% of participants with evaluable data.
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
11.Secondary Outcome
Title Change From Baseline in Bone Marrow Burden (BMB) Score at Week 103
Hide Description BMB Score was measured using MRI, range from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0-16 points. A higher BMB score signified more severe bone marrow involvement. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint.
Time Frame Baseline, Week 103
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety population.
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: units on a scale
Lumbar spine scores -1.2  (1.79)
Femur scores 0.0  (2.12)
Total scores -1.2  (2.95)
12.Secondary Outcome
Title Change From Baseline in Growth Velocity at Week 101 : Height Z-Score
Hide Description The Z-score, or Standard Deviation Score, is a measure of number of SDs above or below the average BMD of a healthy participant of the same age and gender. World Health Organization 2007 growth reference data were used for Z-score calculation. Statistical analysis plan only required summarization if >50% of participants had evaluable data. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint.
Time Frame Baseline, Week 101
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data was not reported as there was <50% of participants had evaluable data.
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
13.Secondary Outcome
Title Change From Baseline in Skeletal Age at Week 103: Z-Score
Hide Description Skeletal age was measured via radiography (X-ray) of the left hand and wrist by the method of Greulich and Pyle. The Z-score, or Standard Deviation Score, is a measure of number of SDs above or below the average BMD of a healthy participant of the same age and gender. Statistical analysis plan only required summarization if >50% of participants had evaluable data. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint.
Time Frame Baseline, Week 103
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data was not reported as there was <50% of participants had evaluable data.
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
14.Secondary Outcome
Title Change From Baseline in Plasma Chitotriosidase Levels at Week 101
Hide Description Plasma chitotriosidase activity levels were measured using an enzymatic assay with 4-methylumbelliferyl-deoxychitobiose as a substrate. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint.
Time Frame Baseline, Week 101
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety population. Number of participants analyzed signifies participants who were not deficient at baseline in chitotriosidase activity or who did not have a 24 base pair duplication in either copy of the chitotriosidase gene.
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 2
Mean (Standard Deviation)
Unit of Measure: nanomole/milliliter/hour (nmol/mL/h)
-181.0  (268.70)
15.Secondary Outcome
Title Number of Participants With Change From Baseline in Neurological Status at Week 103
Hide Description Neurological status was considered normal or abnormal based on investigator’s discretion. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint.
Time Frame Baseline, Week 103
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety population. Number of participants analyzed signifies participants evaluable for this outcome.
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: participants
0
16.Secondary Outcome
Title Change From Baseline in Chemokine [C-C Motif] Ligand 18 (CCL18) Levels at Week 101
Hide Description Plasma CCL18 concentrations were measured using a time-resolved fluorescence assay. Week 51 of Study HGT-GCB-087 (NCT01614574) was considered as baseline for this endpoint.
Time Frame Baseline, Week 101
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety population
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 5
Mean (Standard Deviation)
Unit of Measure: nanogram per milliliter (ng/mL)
-11.4  (50.97)
Time Frame From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Velaglucerase Alfa (VPRIV®)
Hide Arm/Group Description 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.
All-Cause Mortality
Velaglucerase Alfa (VPRIV®)
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Velaglucerase Alfa (VPRIV®)
Affected / at Risk (%) # Events
Total   2/5 (40.00%)    
Ear and labyrinth disorders   
Hypoacusis  1  1/5 (20.00%)  2
Eye disorders   
Retinal detachment  1  1/5 (20.00%)  2
Vitreous opacities  1  1/5 (20.00%)  2
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (9.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Velaglucerase Alfa (VPRIV®)
Affected / at Risk (%) # Events
Total   5/5 (100.00%)    
Ear and labyrinth disorders   
Hypoacusis  1  1/5 (20.00%)  2
Eye disorders   
Retinal detachment  1  1/5 (20.00%)  2
Vitreous opacities  1  1/5 (20.00%)  2
Gastrointestinal disorders   
Abdominal pain  1  1/5 (20.00%)  1
Abdominal pain upper  1  2/5 (40.00%)  2
Constipation  1  1/5 (20.00%)  1
Enteritis  1  1/5 (20.00%)  1
General disorders   
Chest pain  1  1/5 (20.00%)  1
Infections and infestations   
Adenoiditis  1  1/5 (20.00%)  1
Gastroenteritis  1  2/5 (40.00%)  2
Hordeolum  1  2/5 (40.00%)  2
Nasopharyngitis  1  2/5 (40.00%)  7
Otitis media acute  1  1/5 (20.00%)  1
Subcutaneous abscess  1  1/5 (20.00%)  1
Upper respiratory tract infection  1  1/5 (20.00%)  1
Injury, poisoning and procedural complications   
Concussion  1  1/5 (20.00%)  1
Contusion  1  2/5 (40.00%)  3
Excoriation  1  2/5 (40.00%)  2
Fall  1  1/5 (20.00%)  1
Heat stroke  1  1/5 (20.00%)  1
Road traffic accident  1  1/5 (20.00%)  1
Wound  1  1/5 (20.00%)  1
Investigations   
Blood creatine phosphokinase increased  1  1/5 (20.00%)  1
C-Reactive protein increased  1  1/5 (20.00%)  1
Intraocular pressure increased  1  1/5 (20.00%)  2
Musculoskeletal and connective tissue disorders   
Back pain  1  1/5 (20.00%)  1
Intervertebral disc protrusion  1  1/5 (20.00%)  1
Muscle fatigue  1  1/5 (20.00%)  1
Neck pain  1  1/5 (20.00%)  1
Pain in extremity  1  1/5 (20.00%)  1
Trigger finger  1  1/5 (20.00%)  1
Nervous system disorders   
Headache  1  3/5 (60.00%)  4
Psychiatric disorders   
Sleep disorder  1  1/5 (20.00%)  2
Somatoform disorder  1  1/5 (20.00%)  1
Skin and subcutaneous tissue disorders   
Alopecia  1  1/5 (20.00%)  1
Eczema  1  1/5 (20.00%)  1
Pruritus  1  1/5 (20.00%)  2
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (9.0)
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.
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
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