Trial Outcomes & Findings for Survey Study for Velaglucerase Alfa (VPRIV) in Japan (NCT NCT03625882)

Last Updated: 2025-01-10

Results Overview

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.

Recruitment status

COMPLETED

Target enrollment

63 participants

Primary outcome timeframe

Baseline up to end of the study (8 years)

Results posted on

2025-01-10

Participant Flow

Participants took part in the survey at 45 investigative sites in Japan, from 2 Sep 2014 to 14 May 2024.

Participants with Gaucher disease who received Recombinant Velaglucerase Alfa were enrolled. Participants received VPRIV intravenous infusion as part of a routine medical care.

Participant milestones

Participant milestones
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
Overall Study STARTED	63
Overall Study COMPLETED	60
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
Overall Study Protocol Violation	3

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion n=60 Participants Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
Age, Continuous	19.8 Years STANDARD_DEVIATION 21.77 • n=60 Participants
Sex: Female, Male Female	30 Participants n=60 Participants
Sex: Female, Male Male	30 Participants n=60 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion n=60 Participants Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa Adverse Events	42 Participants
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 outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion n=35 Participants Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration Good	11 Participants
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration Moderate response	4 Participants
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration Ineffective	20 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion n=19 Participants Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts Good	9 Participants
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts Moderate response	6 Participants
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts Ineffective	4 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion n=7 Participants Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes Good	0 Participants
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes Moderate response	1 Participants
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes Ineffective	6 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion n=8 Participants Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes Good	3 Participants
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes Moderate response	2 Participants
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes Ineffective	3 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion n=1 Participants Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
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 Standard Deviation NA The standard deviation was not evaluable due to low number of participants with events.
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 Standard Deviation NA The standard deviation was not evaluable due to low number of participants with events.
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 Standard Deviation NA The standard deviation was not evaluable due to low number of participants with events.
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 Standard Deviation NA The standard deviation was not evaluable due to low number of participants with events.

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion n=2 Participants Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
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 Standard Deviation NA The standard deviation was not evaluable due to low number of participants with events.
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
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 Standard Deviation NA The standard deviation was not evaluable due to low number of participants with events.
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 Standard Deviation NA The standard deviation was not evaluable due to low number of participants with events.

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion n=2 Participants Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
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
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 Standard Deviation NA The standard deviation was not evaluable due to low number of participants with events.
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
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 Standard Deviation NA The standard deviation was not evaluable due to low number of participants with events.

SECONDARY outcome

Timeframe: Baseline up to end of the study (8 years)

Number of participants with positive anti-velaglucerase alfa antibody test results was reported.

Outcome measures

Outcome measures
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion n=15 Participants Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
Number of Participants With Positive Anti-velaglucerase Alfa Antibody Test Results	2 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion n=60 Participants Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
Number of Participants With Adverse Drug Reaction and Serious Adverse Drug Reaction Following Initiation of Treatment With Velaglucerase Alfa Adverse Drug Reaction	14 Participants
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 outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion n=60 Participants Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
Number of Participants With Adverse Reactions Categorized as Infusion Reactions Following Initiation of Treatment With Velaglucerase Alfa	6 Participants

Adverse Events

Recombinant Velaglucerase Alfa Intravenous Infusion

Serious events: 27 serious events

Other events: 32 other events

Deaths: 14 deaths

Serious adverse events

Serious adverse events
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion n=60 participants at risk Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
Infections and infestations Bronchitis	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Infections and infestations Cellulitis	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Infections and infestations Peritonitis	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Infections and infestations Pneumonia	5.0% 3/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Infections and infestations Pneumonia aspiration	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Infections and infestations Pyelonephritis	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Infections and infestations Sepsis	3.3% 2/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Infections and infestations Urinary tract infection	3.3% 2/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Infections and infestations Staphylococcal sepsis	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Infections and infestations Device related sepsis	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Acute myeloid leukaemia	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Myelodysplastic syndrome	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Blood and lymphatic system disorders Autoimmune neutropenia	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Metabolism and nutrition disorders Dehydration	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Metabolism and nutrition disorders Hyperkalaemia	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Psychiatric disorders Eating disorder	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Psychiatric disorders Tension	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Psychiatric disorders Bipolar disorder	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Nervous system disorders Myoclonus	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Nervous system disorders Seizure	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Nervous system disorders Myoclonic epilepsy	3.3% 2/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Cardiac disorders Arrhythmia	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Cardiac disorders Cardiac arrest	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Cardiac disorders Cardiac failure congestive	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Cardiac disorders Cardio-respiratory arrest	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Cardiac disorders Ventricular fibrillation	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Respiratory, thoracic and mediastinal disorders Asphyxia	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Respiratory, thoracic and mediastinal disorders Central sleep apnoea syndrome	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Respiratory, thoracic and mediastinal disorders Dyspnoea	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Respiratory, thoracic and mediastinal disorders Respiratory disorder	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Respiratory, thoracic and mediastinal disorders Respiratory failure	5.0% 3/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Gastrointestinal disorders Abdominal pain	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Gastrointestinal disorders Ascites	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Gastrointestinal disorders Colitis ischaemic	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Gastrointestinal disorders Diarrhoea	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Gastrointestinal disorders Duodenal ulcer	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Gastrointestinal disorders Gastrooesophageal reflux disease	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Gastrointestinal disorders Gastroenteritis eosinophilic	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Gastrointestinal disorders Vomiting	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Hepatobiliary disorders Bile duct stone	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Hepatobiliary disorders Cholelithiasis	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Renal and urinary disorders Renal failure	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Renal and urinary disorders Vesicoureteric reflux	3.3% 2/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Renal and urinary disorders Renal impairment	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Renal and urinary disorders Acute kidney injury	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
General disorders Death	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
General disorders Pyrexia	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
General disorders General physical health deterioration	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
General disorders Multiple organ dysfunction syndrome	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Injury, poisoning and procedural complications Head injury	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Injury, poisoning and procedural complications Road traffic accident	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Injury, poisoning and procedural complications Foreign body in throat	1.7% 1/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.

Other adverse events

Other adverse events
Measure	Recombinant Velaglucerase Alfa Intravenous Infusion n=60 participants at risk Participants with Gaucher disease received Recombinant Velaglucerase Alfa intravenous infusion as part of a routine medical care.
Infections and infestations Bronchitis	5.0% 3/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Infections and infestations Pneumonia	8.3% 5/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Infections and infestations Urinary tract infection	6.7% 4/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Blood and lymphatic system disorders Anaemia	10.0% 6/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Metabolism and nutrition disorders Hypokalaemia	5.0% 3/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Metabolism and nutrition disorders Zinc deficiency	5.0% 3/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Psychiatric disorders Insomnia	5.0% 3/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Nervous system disorders Myoclonus	5.0% 3/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Gastrointestinal disorders Diarrhoea	5.0% 3/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Hepatobiliary disorders Hepatic function abnormal	10.0% 6/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
General disorders Pyrexia	13.3% 8/60 • Baseline up to end of the study (8 years) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.

Additional Information

Study Director

Takeda

Phone: +1-877-825-3327

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place