Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease
Study Details
Study Description
Brief Summary
Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. Type 1 Gaucher disease affects an estimated 30,000 persons worldwide and is the most common. Type 1 Gaucher disease does not involve the central nervous system. Patients with Type 2 Gaucher disease present with acute neurological deterioration, which leads to early death. Those with Type 3 disease typically display a more sub-acute neurological course, with later onset and slower progression.
The primary objective of this study is to evaluate the long-term safety of every other week (EOW) dosing of velaglucerase alfa in Japanese patients with Gaucher disease who completed study HGT-GCB-087 and elected to continue treatment with velaglucerase alfa.
Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type 1 Gaucher disease.
Condition or Disease | Intervention/Treatment | Phase |
---|---|---|
|
Phase 3 |
Detailed Description
Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain.
Gaucher disease has been designated in the list of Specified Rare and Intractable Diseases by Specified Disease Treatment Research Program of Ministry of Health, Labor and Welfare (MHLW) as one of "lysosomal storage diseases" since 2001. Gaucher disease is also designated in the Medical Aid Program for Specified Categories of Chronic Pediatric Diseases.
The prevalence of mutations and the phenotype of patients with Gaucher disease in Japan differs from that in non-Japanese populations. Some patients with type 1 Gaucher disease in Japan have more severe and progressive disease compared to non-Japanese patients and the disease is characterized by an earlier onset of symptoms.
Velaglucerase alfa, a highly-purified form of the naturally occurring enzyme glucocerebrosidase, has been developed as an enzyme replacement therapy for Gaucher disease for the symptoms (anemia, thrombocytopenia, hepatomegaly, splenomegaly, and bone manifestation).
The primary objective of this study is to evaluate the long-term safety of every other week (EOW) dosing of velaglucerase alfa in Japanese patients with Gaucher disease who completed study HGT-GCB-087 and elected to continue treatment with velaglucerase alfa.
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
---|---|
Experimental: velaglucerase alfa 15 to 60 U/kg, EOW via intravenous infusion |
Drug: velaglucerase alfa
15-60 U/kg, EOW
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Number of Participants With Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs) [From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks)]
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.
- Number of Participants Using Concomitant Medication [From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks)]
- Number of Participants With Abnormal and Clinically Significant Laboratory Test Results [From Week 65 until the end of study (Week 155)]
Laboratory test results were considered abnormal and clinically significant at the discretion of the investigator.
- Number of Participants With Positive Anti-Velaglucerase Alfa Antibodies [From Week 65 until the end of study (Week 155)]
Serum samples were collected for all participants for determination of anti-velaglucerase alfa antibodies every 12 weeks.
Secondary Outcome Measures
- Change From Baseline in Hemoglobin Concentration at Week 101 [Baseline, Week 101]
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).
- Change From Baseline in Platelet Count at Week 101 [Baseline, Week 101]
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).
- Change From Baseline in Liver Volume Normalized to Body Weight at Week 103 [Baseline, Week 103]
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.
- Change From Baseline in Spleen Volume Normalized to Body Weight at Week 103 [Baseline, Week 103]
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.
- Change From Baseline in Bone Mineral Density (BMD) at Week 103: Z Score [Baseline, Week 103]
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.
- Change From Baseline in Bone Mineral Density (BMD) at Week 103: T-Score [Baseline, Week 103]
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.
- Change From Baseline in Bone Marrow Burden (BMB) Score at Week 103 [Baseline, Week 103]
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.
- Change From Baseline in Growth Velocity at Week 101 : Height Z-Score [Baseline, Week 101]
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.
- Change From Baseline in Skeletal Age at Week 103: Z-Score [Baseline, Week 103]
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.
- Change From Baseline in Plasma Chitotriosidase Levels at Week 101 [Baseline, Week 101]
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.
- Number of Participants With Change From Baseline in Neurological Status at Week 103 [Baseline, Week 103]
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.
- Change From Baseline in Chemokine [C-C Motif] Ligand 18 (CCL18) Levels at Week 101 [Baseline, Week 101]
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.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
The patient has completed treatment with EOW velaglucerase alfa through Week 51 of study HGT-GCB-087.
-
Female patients of child bearing potential must agree to use a medically acceptable method of contraception at all times during the study.
-
The patient, the patient's parent(s)or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee(IRB/IEC)
-
The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator.
Exclusion Criteria:
-
The patient has received treatment with any investigational drug, other than velaglucerase alfa, or investigational device within 30 days prior to study entry; such use during the study is not permitted.
-
The patient, patient's parent(s), or patient's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study.
-
The patient has a significant comorbidity, as determined by the Investigator that might affect study data or confound the study results.
-
The patient is unable to comply with the protocol as determined by the Investigator.
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
---|---|---|---|---|---|
1 | Hamamatsu University School of Medicine | Hamamatsu | Shizuoka | Japan | 431-3192 |
2 | Iwata City Hospital | Okubo | Shizuoka | Japan | 438-8550 |
3 | The Jikei University School of Medicine | Minato-ku | Tokyo | Japan | 105-8471 |
4 | Chiba Children's Hospital | Chiba | Japan | 266-0007 | |
5 | Osaka City University Hospital | Osaka | Japan | 545-8586 |
Sponsors and Collaborators
- Shire
- Quintiles, Inc.
Investigators
- Study Director: Study Director, Takeda
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- HGT-GCB-091
Study Results
Participant Flow
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 Detail |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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 |
Baseline Characteristics
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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 Participants | 5 |
Age (years) [Mean (Standard Deviation) ] | |
Mean (Standard Deviation) [years] |
22.20
(11.946)
|
Sex: Female, Male (Count of Participants) | |
Female |
1
20%
|
Male |
4
80%
|
Outcome Measures
Title | Number of Participants With Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs) |
---|---|
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) |
Outcome Measure Data
Analysis Population Description |
---|
Safety population. |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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. |
Measure Participants | 5 |
Study drug-related AEs |
2
40%
|
Infusion-related AEs |
0
0%
|
Serious AEs |
2
40%
|
Title | Number of Participants Using Concomitant Medication |
---|---|
Description | |
Time Frame | From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks) |
Outcome Measure Data
Analysis Population Description |
---|
Safety population. |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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. |
Measure Participants | 5 |
Number [participants] |
5
100%
|
Title | Number of Participants With Abnormal and Clinically Significant Laboratory Test Results |
---|---|
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) |
Outcome Measure Data
Analysis Population Description |
---|
Safety population. |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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. |
Measure Participants | 5 |
Number [participants] |
1
20%
|
Title | Number of Participants With Positive Anti-Velaglucerase Alfa Antibodies |
---|---|
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) |
Outcome Measure Data
Analysis Population Description |
---|
Safety population. |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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. |
Measure Participants | 5 |
Number [participants] |
0
0%
|
Title | Change From Baseline in Hemoglobin Concentration at Week 101 |
---|---|
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 |
Outcome Measure Data
Analysis Population Description |
---|
Safety population |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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. |
Measure Participants | 5 |
Mean (Standard Deviation) [gram per deciliter (g/dL)] |
0.22
(0.817)
|
Title | Change From Baseline in Platelet Count at Week 101 |
---|---|
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 |
Outcome Measure Data
Analysis Population Description |
---|
Safety population |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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. |
Measure Participants | 5 |
Mean (Standard Deviation) [*10^9 platelets per liter] |
9.8
(13.14)
|
Title | Change From Baseline in Liver Volume Normalized to Body Weight at Week 103 |
---|---|
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 |
Outcome Measure Data
Analysis Population Description |
---|
Safety population |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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. |
Measure Participants | 5 |
Mean (Standard Deviation) [Percentage of body weight] |
0.01
(0.207)
|
Title | Change From Baseline in Spleen Volume Normalized to Body Weight at Week 103 |
---|---|
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 |
Outcome Measure Data
Analysis Population Description |
---|
Safety population |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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. |
Measure Participants | 5 |
Mean (Standard Deviation) [Percentage of body weight] |
0.04
(0.089)
|
Title | Change From Baseline in Bone Mineral Density (BMD) at Week 103: Z Score |
---|---|
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 |
Outcome Measure Data
Analysis Population Description |
---|
Data was not reported as there were lesser than (<) 50% of participants with evaluable data. |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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. |
Measure Participants | 0 |
Title | Change From Baseline in Bone Mineral Density (BMD) at Week 103: T-Score |
---|---|
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 |
Outcome Measure Data
Analysis Population Description |
---|
Data was not reported as there were <50% of participants with evaluable data. |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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. |
Measure Participants | 0 |
Title | Change From Baseline in Bone Marrow Burden (BMB) Score at Week 103 |
---|---|
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 |
Outcome Measure Data
Analysis Population Description |
---|
Safety population. |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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. |
Measure Participants | 5 |
Lumbar spine scores |
-1.2
(1.79)
|
Femur scores |
0.0
(2.12)
|
Total scores |
-1.2
(2.95)
|
Title | Change From Baseline in Growth Velocity at Week 101 : Height Z-Score |
---|---|
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 |
Outcome Measure Data
Analysis Population Description |
---|
Data was not reported as there was <50% of participants had evaluable data. |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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. |
Measure Participants | 0 |
Title | Change From Baseline in Skeletal Age at Week 103: Z-Score |
---|---|
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 |
Outcome Measure Data
Analysis Population Description |
---|
Data was not reported as there was <50% of participants had evaluable data. |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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. |
Measure Participants | 0 |
Title | Change From Baseline in Plasma Chitotriosidase Levels at Week 101 |
---|---|
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 |
Outcome Measure Data
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®) |
---|---|
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. |
Measure Participants | 2 |
Mean (Standard Deviation) [nanomole/milliliter/hour (nmol/mL/h)] |
-181.0
(268.70)
|
Title | Number of Participants With Change From Baseline in Neurological Status at Week 103 |
---|---|
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 |
Outcome Measure Data
Analysis Population Description |
---|
Safety population. Number of participants analyzed signifies participants evaluable for this outcome. |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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. |
Measure Participants | 3 |
Number [participants] |
0
0%
|
Title | Change From Baseline in Chemokine [C-C Motif] Ligand 18 (CCL18) Levels at Week 101 |
---|---|
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 |
Outcome Measure Data
Analysis Population Description |
---|
Safety population |
Arm/Group Title | Velaglucerase Alfa (VPRIV®) |
---|---|
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. |
Measure Participants | 5 |
Mean (Standard Deviation) [nanogram per milliliter (ng/mL)] |
-11.4
(50.97)
|
Adverse Events
Time Frame | From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks) | |
---|---|---|
Adverse Event Reporting Description | ||
Arm/Group Title | Velaglucerase Alfa (VPRIV®) | |
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 (%) | # Events | |
Total | / (NaN) | |
Serious Adverse Events |
||
Velaglucerase Alfa (VPRIV®) | ||
Affected / at Risk (%) | # Events | |
Total | 2/5 (40%) | |
Ear and labyrinth disorders | ||
Hypoacusis | 1/5 (20%) | 2 |
Eye disorders | ||
Retinal detachment | 1/5 (20%) | 2 |
Vitreous opacities | 1/5 (20%) | 2 |
Other (Not Including Serious) Adverse Events |
||
Velaglucerase Alfa (VPRIV®) | ||
Affected / at Risk (%) | # Events | |
Total | 5/5 (100%) | |
Ear and labyrinth disorders | ||
Hypoacusis | 1/5 (20%) | 2 |
Eye disorders | ||
Retinal detachment | 1/5 (20%) | 2 |
Vitreous opacities | 1/5 (20%) | 2 |
Gastrointestinal disorders | ||
Abdominal pain | 1/5 (20%) | 1 |
Abdominal pain upper | 2/5 (40%) | 2 |
Constipation | 1/5 (20%) | 1 |
Enteritis | 1/5 (20%) | 1 |
General disorders | ||
Chest pain | 1/5 (20%) | 1 |
Infections and infestations | ||
Adenoiditis | 1/5 (20%) | 1 |
Gastroenteritis | 2/5 (40%) | 2 |
Hordeolum | 2/5 (40%) | 2 |
Nasopharyngitis | 2/5 (40%) | 7 |
Otitis media acute | 1/5 (20%) | 1 |
Subcutaneous abscess | 1/5 (20%) | 1 |
Upper respiratory tract infection | 1/5 (20%) | 1 |
Injury, poisoning and procedural complications | ||
Concussion | 1/5 (20%) | 1 |
Contusion | 2/5 (40%) | 3 |
Excoriation | 2/5 (40%) | 2 |
Fall | 1/5 (20%) | 1 |
Heat stroke | 1/5 (20%) | 1 |
Road traffic accident | 1/5 (20%) | 1 |
Wound | 1/5 (20%) | 1 |
Investigations | ||
Blood creatine phosphokinase increased | 1/5 (20%) | 1 |
C-Reactive protein increased | 1/5 (20%) | 1 |
Intraocular pressure increased | 1/5 (20%) | 2 |
Musculoskeletal and connective tissue disorders | ||
Back pain | 1/5 (20%) | 1 |
Intervertebral disc protrusion | 1/5 (20%) | 1 |
Muscle fatigue | 1/5 (20%) | 1 |
Neck pain | 1/5 (20%) | 1 |
Pain in extremity | 1/5 (20%) | 1 |
Trigger finger | 1/5 (20%) | 1 |
Nervous system disorders | ||
Headache | 3/5 (60%) | 4 |
Psychiatric disorders | ||
Sleep disorder | 1/5 (20%) | 2 |
Somatoform disorder | 1/5 (20%) | 1 |
Skin and subcutaneous tissue disorders | ||
Alopecia | 1/5 (20%) | 1 |
Eczema | 1/5 (20%) | 1 |
Pruritus | 1/5 (20%) | 2 |
Limitations/Caveats
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
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 Director |
---|---|
Organization | Shire |
Phone | +1 866 842 5335 |
ClinicalTransparency@shire.com |
- HGT-GCB-091