Gaucher Disease
Conditions
Brief summary
The objective of this post-marketing survey study is to collect data to determine the safety and efficacy of velaglucerase alfa (VPRIV) in participants with Gaucher disease who are new to therapy or have been switched from another therapeutic agent for Gaucher disease.
Interventions
None listed
Sponsors
Takeda
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Male or female participants with a confirmed diagnosis of Gaucher disease * Participants who are either naïve to treatment or participants that have been treated with another therapeutic agent for Gaucher disease * Participants who start VPRIV treatment or transition from VPRIV clinical studies during the enrollment period
Exclusion criteria
\-
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa | Baseline up to end of the study (8 years) | An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalisation or results in prolongation of existing hospitalisation; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration | Baseline, Every 12 weeks up to 8 years | Number of participants of efficacy categories in assessment of change from baseline in hemoglobin concentration during the study was reported. The efficacy categories were classified as Good: increased by at least 1.5g/dL, Moderate response: increase of at least 0.5 g/dL and less than 1.5 g/dL, and Ineffective: increase less than 0.5 g/dL. |
| Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts | Baseline, Every 12 weeks up to 8 years | Number of participants of efficacy categories in assessment of change from baseline in platelet counts during the study was reported. The efficacy categories were classified as Good: increase of at least 30 × 10\^9/L, Moderate response: increase of at least 15 × 10\^9/L and less than 30 × 10\^9/L, and Ineffective: less than 15 × 10\^9/L. |
| Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes | Baseline, Every 24 weeks up to 8 years | Number of participants of efficacy categories in assessment of change from baseline in liver volumes during the study was reported. The efficacy categories were classified as Good: reduction of at least 30%, Moderate response: reduction of at least 10% and less than 30%, and Ineffective: less than 10% reduction. |
| Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes | Baseline, Every 24 weeks up to 8 years | Number of participants of efficacy categories in assessment of change from baseline in spleen volumes during the study was reported. The efficacy categories were classified as Good: reduction of at least 30%, Moderate response: reduction of at least 10% and less than 30%, and Ineffective: less than 10% reduction. |
| Lumbar Spine Bone Mineral Density (BMD) T-scores and Femur Neck BMD T-scores at Baseline and the Last Data Collection | Baseline, at the time of last data collection (up to 8 years) | T-scores were the number of standard deviations (SDs) above or below the average for a young adult at peak BMD. A T-score of 0 was equal to the mean. Negative numbers indicated values lower than the mean and positive numbers indicated values higher than the mean. The reported data in this outcome measure was lumbar spine BMD T-scores and femur neck BMD T-scores related to bone density at baseline and the last data collection. |
| Lumbar Spine BMD Z-scores and Femur Neck BMD Z-scores at Baseline and the Last Data Collection | Baseline, at the time of last data collection (up to 8 years) | Z-scores express the BMD as the number of SDs above or below the average BMD of a healthy participant of the same age and gender. A Z-score of 0 was equal to the mean. Negative numbers indicated values lower than the mean and positive numbers indicated values higher than the mean. The reported data in this outcome measure was lumbar spine BMD Z-scores and femur neck BMD Z-scores related to bone density at baseline and the last data collection. |
| Lumbar Spine BMD and Femur Neck BMD at Baseline and the Last Data Collection | Baseline, at the time of last data collection (up to 8 years) | The reported data in this outcome measure was lumbar spine BMD and femur neck BMD related to bone density at baseline and the last data collection. |
| Number of Participants With Positive Anti-velaglucerase Alfa Antibody Test Results | Baseline up to end of the study (8 years) | Number of participants with positive anti-velaglucerase alfa antibody test results was reported. |
| Number of Participants With Adverse Drug Reaction and Serious Adverse Drug Reaction Following Initiation of Treatment With Velaglucerase Alfa | Baseline up to end of the study (8 years) | An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalisation or results in prolongation of existing hospitalisation; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event. Adverse drug reaction refers to AE related to administered drug. |
| Number of Participants With Adverse Reactions Categorized as Infusion Reactions Following Initiation of Treatment With Velaglucerase Alfa | Baseline up to end of the study (8 years) | An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Adverse drug reaction refers to AE related to administered drug. Infusion reactions were defined as reactions occurring up to 24 hours after the start of the infusion. |
Countries
Japan
Contacts
STUDY_DIRECTORStudy Director
Takeda
Participant flow
Recruitment details
Participants took part in the survey at 45 investigative sites in Japan, from 2 Sep 2014 to 14 May 2024.
Pre-assignment details
Participants with Gaucher disease who received Recombinant Velaglucerase Alfa were enrolled. Participants received VPRIV intravenous infusion as part of a routine medical care.
Participants by arm
| Arm | Count |
|---|---|
| Recombinant Velaglucerase Alfa Intravenous Infusion Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care. | 60 |
| Total | 60 |
Baseline characteristics
| Characteristic | Recombinant Velaglucerase Alfa Intravenous Infusion |
|---|---|
| Age, Continuous | 19.8 Years STANDARD_DEVIATION 21.77 |
| Sex: Female, Male Female | 30 Participants |
| Sex: Female, Male Male | 30 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 14 / 60 |
| other Total, other adverse events | 31 / 60 |
| serious Total, serious adverse events | 27 / 60 |
Outcome results
Primary
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa
An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalisation or results in prolongation of existing hospitalisation; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event.
Time frame: Baseline up to end of the study (8 years)
Population: Safety Analysis Set, The safety analysis set was defined as all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa | Adverse Events | 42 Participants |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa | Serious Adverse Events | 27 Participants |
Secondary
Lumbar Spine BMD and Femur Neck BMD at Baseline and the Last Data Collection
The reported data in this outcome measure was lumbar spine BMD and femur neck BMD related to bone density at baseline and the last data collection.
Time frame: Baseline, at the time of last data collection (up to 8 years)
Population: Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Recombinant Velaglucerase Alfa Intravenous Infusion | Lumbar Spine BMD and Femur Neck BMD at Baseline and the Last Data Collection | Lumbar Spine BMD: Baseline | 0.7215 gram per square centimeter | Standard Deviation 0.19304 |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Lumbar Spine BMD and Femur Neck BMD at Baseline and the Last Data Collection | Lumbar Spine BMD: At the last data collection | 0.3550 gram per square centimeter | — |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Lumbar Spine BMD and Femur Neck BMD at Baseline and the Last Data Collection | Femur Neck BMD: Baseline | 0.6335 gram per square centimeter | Standard Deviation 0.11809 |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Lumbar Spine BMD and Femur Neck BMD at Baseline and the Last Data Collection | Femur Neck BMD: At the last data collection | 0.3400 gram per square centimeter | — |
Secondary
Lumbar Spine BMD Z-scores and Femur Neck BMD Z-scores at Baseline and the Last Data Collection
Z-scores express the BMD as the number of SDs above or below the average BMD of a healthy participant of the same age and gender. A Z-score of 0 was equal to the mean. Negative numbers indicated values lower than the mean and positive numbers indicated values higher than the mean. The reported data in this outcome measure was lumbar spine BMD Z-scores and femur neck BMD Z-scores related to bone density at baseline and the last data collection.
Time frame: Baseline, at the time of last data collection (up to 8 years)
Population: Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Recombinant Velaglucerase Alfa Intravenous Infusion | Lumbar Spine BMD Z-scores and Femur Neck BMD Z-scores at Baseline and the Last Data Collection | Lumbar Spine BMD Z-scores: Baseline | -2.20 Z-score | — |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Lumbar Spine BMD Z-scores and Femur Neck BMD Z-scores at Baseline and the Last Data Collection | Lumbar Spine BMD Z-scores: At the last data collection | -1.15 Z-score | Standard Deviation 1.202 |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Lumbar Spine BMD Z-scores and Femur Neck BMD Z-scores at Baseline and the Last Data Collection | Femur Neck BMD Z-scores: Baseline | -0.60 Z-score | — |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Lumbar Spine BMD Z-scores and Femur Neck BMD Z-scores at Baseline and the Last Data Collection | Femur Neck BMD Z-scores: At the last data collection | -0.50 Z-score | — |
Secondary
Lumbar Spine Bone Mineral Density (BMD) T-scores and Femur Neck BMD T-scores at Baseline and the Last Data Collection
T-scores were the number of standard deviations (SDs) above or below the average for a young adult at peak BMD. A T-score of 0 was equal to the mean. Negative numbers indicated values lower than the mean and positive numbers indicated values higher than the mean. The reported data in this outcome measure was lumbar spine BMD T-scores and femur neck BMD T-scores related to bone density at baseline and the last data collection.
Time frame: Baseline, at the time of last data collection (up to 8 years)
Population: Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Recombinant Velaglucerase Alfa Intravenous Infusion | Lumbar Spine Bone Mineral Density (BMD) T-scores and Femur Neck BMD T-scores at Baseline and the Last Data Collection | Lumbar Spine BMD T-scores: Baseline | -4.30 T-score |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Lumbar Spine Bone Mineral Density (BMD) T-scores and Femur Neck BMD T-scores at Baseline and the Last Data Collection | Lumbar Spine BMD T-scores: At the last data collection | -0.30 T-score |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Lumbar Spine Bone Mineral Density (BMD) T-scores and Femur Neck BMD T-scores at Baseline and the Last Data Collection | Femur Neck BMD T-scores: Baseline | -2.90 T-score |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Lumbar Spine Bone Mineral Density (BMD) T-scores and Femur Neck BMD T-scores at Baseline and the Last Data Collection | Femur Neck BMD T-scores: At the last data collection | -0.60 T-score |
Secondary
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration
Number of participants of efficacy categories in assessment of change from baseline in hemoglobin concentration during the study was reported. The efficacy categories were classified as Good: increased by at least 1.5g/dL, Moderate response: increase of at least 0.5 g/dL and less than 1.5 g/dL, and Ineffective: increase less than 0.5 g/dL.
Time frame: Baseline, Every 12 weeks up to 8 years
Population: Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration | Good | 11 Participants |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration | Moderate response | 4 Participants |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration | Ineffective | 20 Participants |
Secondary
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes
Number of participants of efficacy categories in assessment of change from baseline in liver volumes during the study was reported. The efficacy categories were classified as Good: reduction of at least 30%, Moderate response: reduction of at least 10% and less than 30%, and Ineffective: less than 10% reduction.
Time frame: Baseline, Every 24 weeks up to 8 years
Population: Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes | Good | 0 Participants |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes | Moderate response | 1 Participants |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes | Ineffective | 6 Participants |
Secondary
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts
Number of participants of efficacy categories in assessment of change from baseline in platelet counts during the study was reported. The efficacy categories were classified as Good: increase of at least 30 × 10\^9/L, Moderate response: increase of at least 15 × 10\^9/L and less than 30 × 10\^9/L, and Ineffective: less than 15 × 10\^9/L.
Time frame: Baseline, Every 12 weeks up to 8 years
Population: Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts | Good | 9 Participants |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts | Moderate response | 6 Participants |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts | Ineffective | 4 Participants |
Secondary
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes
Number of participants of efficacy categories in assessment of change from baseline in spleen volumes during the study was reported. The efficacy categories were classified as Good: reduction of at least 30%, Moderate response: reduction of at least 10% and less than 30%, and Ineffective: less than 10% reduction.
Time frame: Baseline, Every 24 weeks up to 8 years
Population: Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes | Good | 3 Participants |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes | Moderate response | 2 Participants |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes | Ineffective | 3 Participants |
Secondary
Number of Participants With Adverse Drug Reaction and Serious Adverse Drug Reaction Following Initiation of Treatment With Velaglucerase Alfa
An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalisation or results in prolongation of existing hospitalisation; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event. Adverse drug reaction refers to AE related to administered drug.
Time frame: Baseline up to end of the study (8 years)
Population: Safety Analysis Set, The safety analysis set was defined as all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants With Adverse Drug Reaction and Serious Adverse Drug Reaction Following Initiation of Treatment With Velaglucerase Alfa | Adverse Drug Reaction | 14 Participants |
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants With Adverse Drug Reaction and Serious Adverse Drug Reaction Following Initiation of Treatment With Velaglucerase Alfa | Serious Adverse Drug Reaction | 5 Participants |
Secondary
Number of Participants With Adverse Reactions Categorized as Infusion Reactions Following Initiation of Treatment With Velaglucerase Alfa
An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Adverse drug reaction refers to AE related to administered drug. Infusion reactions were defined as reactions occurring up to 24 hours after the start of the infusion.
Time frame: Baseline up to end of the study (8 years)
Population: Safety Analysis Set, The safety analysis set was defined as all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants With Adverse Reactions Categorized as Infusion Reactions Following Initiation of Treatment With Velaglucerase Alfa | 6 Participants |
Secondary
Number of Participants With Positive Anti-velaglucerase Alfa Antibody Test Results
Number of participants with positive anti-velaglucerase alfa antibody test results was reported.
Time frame: Baseline up to end of the study (8 years)
Population: Efficacy Analysis Set: all participants who received at least one dose of Recombinant Velaglucerase Alfa intravenous infusion. The number analyzed was the number of participants what the data was available in this outcome measure during the study.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Recombinant Velaglucerase Alfa Intravenous Infusion | Number of Participants With Positive Anti-velaglucerase Alfa Antibody Test Results | 2 Participants |