Trial Outcomes & Findings for Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease (NCT NCT02574286)

Last Updated: 2022-02-01

Results Overview

Bone mineral density of the lumbar spine was measured by dual energy x-ray absorptiometry (DXA), and the results was converted to Z-scores appropriate for age, sex, and race. The Z-score indicated the number of standard deviations away from a reference population in the same age range, race and with the same sex. 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. Baseline was defined as last data collected prior to the first administration of study drug. Change from baseline in lumbar spine BMD Z-Score up to EOS (Week 103) was reported.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

21 participants

Primary outcome timeframe

Baseline up to EOS (Week 103)

Results posted on

2022-02-01

Participant Flow

This study was conducted in the United States, Israel, Spain and United Kingdom from 29 June 2016 (first participants first visit) to 30 November 2020 (last participants last visit).

A total of 21 participants were enrolled and received treatment in this study.

Participant milestones

Participant milestones
Measure	Velaglucerase Alfa 60 U/kg Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Overall Study STARTED	21
Overall Study COMPLETED	16
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Reasons for withdrawal
Measure	Velaglucerase Alfa 60 U/kg Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Overall Study Adverse Event	3
Overall Study Withdrawal by Subject	2

Baseline Characteristics

Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Velaglucerase Alfa 60 U/kg n=21 Participants Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa EOW for 24 months (up to 101 weeks).
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	0 Participants n=99 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants
Race (NIH/OMB) Black or African American	0 Participants n=99 Participants
Race (NIH/OMB) White	20 Participants n=99 Participants
Race (NIH/OMB) More than one race	1 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=99 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	19 Participants n=99 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=99 Participants
Age, Continuous	43.9 Years STANDARD_DEVIATION 14.2 • n=99 Participants
Sex: Female, Male Female	11 Participants n=99 Participants
Sex: Female, Male Male	10 Participants n=99 Participants

PRIMARY outcome

Timeframe: Baseline up to EOS (Week 103)

Population: Intent-to-Treat (ITT) Population was defined as all enrolled participants who received at least one study drug infusion (full or partial). Here, "Overall number of participants analyzed" signifies participants who were evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Velaglucerase Alfa 60 U/kg n=16 Participants Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-Score up to End of Study (EOS) (Week 103)	0.17 Z-score Standard Deviation 0.394

SECONDARY outcome

Timeframe: Baseline, Week 51

Population: ITT Population was defined as all enrolled participants who received at least one study drug infusion (full or partial). Here, "Overall number of participants analyzed" signifies participants who were evaluable for this outcome measure.

BMD of the LS was measured by DXA, and the results was converted to Z-scores appropriate for age, sex, and race. The Z-score indicated the number of standard deviations away from a reference population in the same age range, race and with the same sex. 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. Baseline was defined as last data collected prior to the first administration of study drug. Change From baseline in LS BMD Z-score at Week 51 was reported. ITT Population was defined as all enrolled subjects who received at least one study drug infusion (full or partial). Here, "number of subjects analysed" signifies subjects who were evaluable for this endpoint.

Outcome measures

Outcome measures
Measure	Velaglucerase Alfa 60 U/kg n=16 Participants Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Change From Baseline in Lumbar Spine (LS) BMD Z-score at Week 51	0.02 Z-Score Standard Deviation 0.431

SECONDARY outcome

Timeframe: Baseline, Week 51 and EOS (Week 103)

Bone mineral density of the LS was measured by DXA, and the results was measured in gram per square centimeter (g/cm\^2). Baseline was defined as last data collected prior to the first administration of study drug. Change From baseline in LS BMD at Week 51 and EOS (Week 103) was reported.

Outcome measures

Outcome measures
Measure	Velaglucerase Alfa 60 U/kg n=16 Participants Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Change From Baseline in Lumbar Spine BMD at Week 51 and EOS (Week 103) Change at Week 51	0.006 g/cm^2 Standard Deviation 0.0342
Change From Baseline in Lumbar Spine BMD at Week 51 and EOS (Week 103) Change at EOS (Week 103)	0.011 g/cm^2 Standard Deviation 0.0474

SECONDARY outcome

Timeframe: Baseline, Week 51 and EOS (Week 103)

BMB score was a semi-quantitative magnetic resonance imaging (MRI) scoring system for assessing the extent of bone marrow involvement in Gaucher disease. BMB Score was measured using magnetic resonance imaging (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 BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. Baseline was defined as last data collected prior to the first administration of study drug. Change from baseline in total BMB Score at Week 51 and EOS (Week 103) was reported.

Outcome measures

Outcome measures
Measure	Velaglucerase Alfa 60 U/kg n=15 Participants Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Change From Baseline in Total Bone Marrow Burden (BMB) Score at Week 51 and EOS (Week 103) Change at Week 51	-3.0 Score on a Scale Standard Deviation 1.85
Change From Baseline in Total Bone Marrow Burden (BMB) Score at Week 51 and EOS (Week 103) Change at EOS (Week 103)	-3.0 Score on a Scale Standard Deviation 2.27

SECONDARY outcome

Timeframe: Baseline, Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103)

Blood samples were collected for measurement of hemoglobin concentration. Baseline was defined as last data collected prior to the first administration of study drug. Change from baseline in hemoglobin concentration at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) was reported.

Outcome measures

Outcome measures
Measure	Velaglucerase Alfa 60 U/kg n=18 Participants Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Change From Baseline in Hemoglobin Concentration at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at Week 13	0.532 Grams per deciliter (g/dL) Standard Deviation 0.7624
Change From Baseline in Hemoglobin Concentration at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at Week 25	0.764 Grams per deciliter (g/dL) Standard Deviation 0.6688
Change From Baseline in Hemoglobin Concentration at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at Week 37	0.765 Grams per deciliter (g/dL) Standard Deviation 0.7836
Change From Baseline in Hemoglobin Concentration at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at Week 51	0.935 Grams per deciliter (g/dL) Standard Deviation 0.6588
Change From Baseline in Hemoglobin Concentration at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at Week 65	1.029 Grams per deciliter (g/dL) Standard Deviation 0.8239
Change From Baseline in Hemoglobin Concentration at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at Week 77	1.015 Grams per deciliter (g/dL) Standard Deviation 1.1577
Change From Baseline in Hemoglobin Concentration at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at Week 89	1.138 Grams per deciliter (g/dL) Standard Deviation 0.8910
Change From Baseline in Hemoglobin Concentration at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at Week EOS (Week 103)	0.897 Grams per deciliter (g/dL) Standard Deviation 1.2309

SECONDARY outcome

Timeframe: Baseline, Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103)

Blood samples were collected for measurement of platelet count. Baseline was defined as last data collected prior to the first administration of study drug. Change from baseline over time in platelet count at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) was reported.

Outcome measures

Outcome measures
Measure	Velaglucerase Alfa 60 U/kg n=17 Participants Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Change From Baseline in Platelet Count at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at Week 13	38.06 10^9 platelets per liter Standard Deviation 35.571
Change From Baseline in Platelet Count at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at Week 25	53.24 10^9 platelets per liter Standard Deviation 46.955
Change From Baseline in Platelet Count at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at Week 37	62.23 10^9 platelets per liter Standard Deviation 46.834
Change From Baseline in Platelet Count at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at Week 51	79.66 10^9 platelets per liter Standard Deviation 89.701
Change From Baseline in Platelet Count at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at Week 65	75.03 10^9 platelets per liter Standard Deviation 52.163
Change From Baseline in Platelet Count at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at Week 77	87.19 10^9 platelets per liter Standard Deviation 70.528
Change From Baseline in Platelet Count at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at Week 89	71.96 10^9 platelets per liter Standard Deviation 60.772
Change From Baseline in Platelet Count at Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) Change at EOS (Week 103)	69.16 10^9 platelets per liter Standard Deviation 53.451

SECONDARY outcome

Timeframe: Baseline, Week 51 and EOS (Week 103)

Normalized liver volume was measured by abdominal MRI. Baseline was defined as last data collected prior to the first administration of study drug. Liver volume has been normalized for percent (%) body weight. Liver size relative to body weight = (Liver volume \[cubic centimeter (cc)\]/Body weight \[kg\])\*100. Change from baseline in normalized liver volume at Week 51 and EOS (Week 103) was reported.

Outcome measures

Outcome measures
Measure	Velaglucerase Alfa 60 U/kg n=17 Participants Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Change From Baseline in Normalized Liver Volume at Week 51 and EOS (Week 103) Change at Week 51	-0.353 Percent body weight Standard Deviation 0.3485
Change From Baseline in Normalized Liver Volume at Week 51 and EOS (Week 103) Change at EOS (Week 103)	-0.447 Percent body weight Standard Deviation 0.4048

SECONDARY outcome

Timeframe: Baseline, Week 51 and EOS (Week 103)

Normalized spleen volume was measured by MRI. Spleen volume was normalized for % of body weight. Spleen size relative to body weight= (Spleen volume \[cc\]/Body weight \[kg\])\*100. Baseline was defined as last data collected prior to the first administration of study drug. Change from baseline in normalized spleen volume at Week 51 and EOS (Week 103) was reported.

Outcome measures

Outcome measures
Measure	Velaglucerase Alfa 60 U/kg n=17 Participants Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Change From Baseline in Normalized Spleen Volume at Week 51 and EOS (Week 103) Change at Week 51	-0.443 Percent body weight Standard Deviation 0.5987
Change From Baseline in Normalized Spleen Volume at Week 51 and EOS (Week 103) Change at EOS (Week 103)	-0.556 Percent body weight Standard Deviation 0.7398

SECONDARY outcome

Timeframe: Baseline, Week 51 and EOS (Week 103)

Bone pain was measured by questions taken from the Brief Pain Inventory-short form (BPI-SF). Pain severity was evaluated based on the average of 4 questions from BPI-SF (Questions 3 through 6) assessing worst pain, least pain, average pain, and pain right now, each rated on a scale from 0 (no pain) to 10 (pain as bad as you can imagine) with mild pain- score (1 to 4), moderate pain- score (5 to 6), and severe pain score (7 to 10). Overall severity score was calculated as average of 4 questions ranging from 0 (no pain) to 10 (pain as bad as you can imagine). A negative change from baseline score indicates improvement. Baseline was defined as last data collected prior to the first administration of study drug. Change from baseline in severity of bone pain at Week 51 and EOS (Week 103) was reported.

Outcome measures

Outcome measures
Measure	Velaglucerase Alfa 60 U/kg n=1 Participants Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Change From Baseline in Severity of Bone Pain at Week 51 and EOS (Week 103) Change at Week 51	-2.750 Score on a Scale Standard Deviation NA Here "NA" signifies standard deviation was not estimated due to single participant.
Change From Baseline in Severity of Bone Pain at Week 51 and EOS (Week 103) Change at EOS (Week 103)	-3.250 Score on a Scale Standard Deviation NA Here "NA" signifies standard deviation was not estimated due to single participant.

SECONDARY outcome

Timeframe: Baseline, Week 51 and EOS (Week 103)

Bone pain interference was measured by questions taken from the BPI-SF. Pain interference was evaluated based upon average of 7 questions from BPI-SF (9A through 9G) regarding the extent to which pain interfered with daily activities, including general activity, mood, walking ability, normal work, relations with other people, sleep, and enjoyment of life in the last 24 hours, each rated on a scale from 0 (does not interfere) to 10 (completely interferes). Overall pain interference score was calculated as average of 7 questions ranging from 0 (does not interfere) to 10 (completely interferes). A negative change from baseline score indicates improvement. Baseline was defined as last data collected prior to the first administration of study drug. Change from baseline in bone pain interference score at Week 51 and EOS (Week 103) was reported.

Outcome measures

Outcome measures
Measure	Velaglucerase Alfa 60 U/kg n=1 Participants Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Change From Baseline in Bone Pain Interference Score at Week 51 and EOS (Week 103) Change at Week 51	-1.286 Score on a Scale Standard Deviation NA Here "NA" signifies standard deviation was not estimated due to single participant.
Change From Baseline in Bone Pain Interference Score at Week 51 and EOS (Week 103) Change at EOS (Week 103)	-4.429 Score on a Scale Standard Deviation NA Here "NA" signifies standard deviation was not estimated due to single participant.

SECONDARY outcome

Timeframe: Baseline, Week 51 and EOS (Week 103)

Overall fatigue was measured by the BFI. BFI was a 9-item questionnaire developed to assess subjective fatigue. Each question asked the respondent to rate the level of their experienced fatigue over the past 24 hours on an 11-point (0-10) scale. First 3 questions measure fatigue severity at current, usual, and worst levels, respectively, with 0 indicating "no fatigue" and 10 indicating fatigue "as bad as you can imagine". Next 6 questions assessed the level fatigue interference with daily activities included general activity, mood, walking ability, normal work (both inside and outside the home), relations with other people, and enjoyment of life. A score of 0="no interference" while a score of 10="complete interference. Overall fatigue score was calculated as average score of all 9 items on the BFI ranging from 0="no fatigue" to 10="as bad as you can imagine".

Outcome measures

Outcome measures
Measure	Velaglucerase Alfa 60 U/kg n=5 Participants Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Change From Baseline in Overall Fatigue Measured by Brief Fatigue Inventory (BFI) at Week 51 and EOS (Week 103) Change at Week 51	-0.111 Score on a Scale Standard Deviation 1.4551
Change From Baseline in Overall Fatigue Measured by Brief Fatigue Inventory (BFI) at Week 51 and EOS (Week 103) Change at EOS (Week 103)	0.044 Score on a Scale Standard Deviation 3.7132

SECONDARY outcome

Timeframe: Baseline, Week 51 and EOS (Week 103)

WHO BMD Classifications (Normal Bone Density, Osteopenia, Osteoporosis), bone mineral density was classified based on LS BMD T-scores. BMD T-score was a comparison of an individual's BMD compared to "normal". Also, BMD T-score is the standard deviation of the difference between measured BMD and that of the healthy young adult "normal". The T-score scale was as follows: -1 and above=normal, -1 to -2.5=osteopenia (below normal and may lead to osteoporosis), and -2.5 and below=osteoporosis. Number of participants with shift in WHO BMD classifications based on LS T-Scores at Week 51 and EOS (Week 103) were reported.

Outcome measures

Outcome measures
Measure	Velaglucerase Alfa 60 U/kg n=16 Participants Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Normal at Baseline- Normal at Week 51	0 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Normal at Baseline- Osteopenia at Week 51	0 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Normal at Baseline- Osteoporosis at Week 51	0 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Osteopenia at Baseline- Normal at Week 51	1 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Osteopenia at Baseline- Osteopenia at Week 51	6 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Osteopenia at Baseline- Osteoporosis at Week 51	3 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Osteoporosis at Baseline- Normal at Week 51	0 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Osteoporosis at Baseline- Osteopenia at Week 51	0 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Osteoporosis at Baseline- Osteoporosis at Week 51	6 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Normal at Baseline- Normal at Week 103	1 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Normal at Baseline- Osteopenia at Week 103	0 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Normal at Baseline- Osteoporosis at Week 103	0 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Osteopenia at Baseline- Normal at Week 103	2 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Osteopenia at Baseline- Osteopenia at Week 103	6 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Osteopenia at Baseline- Osteoporosis at Week 103	2 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Osteoporosis at Baseline- Normal at Week 103	0 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Osteoporosis at Baseline- Osteopenia at Week 103	0 Participants
Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at Week 51 and EOS (Week 103) Osteoporosis at Baseline- Osteoporosis at Week 103	5 Participants

SECONDARY outcome

Timeframe: From start of study drug infusion up to follow-up (107 weeks)

Population: Safety population was defined as all enrolled participants who received at least one study drug infusion (full or partial).

An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. A TEAE was defined as any AE that occurred on or after the time of the first infusion of study drug until 30 days after the last infusion of study drug. Number of participants with TEAEs were reported.

Outcome measures

Outcome measures
Measure	Velaglucerase Alfa 60 U/kg n=21 Participants Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	21 Participants

SECONDARY outcome

Timeframe: Baseline up to EOS (Week 103)

Population: Safety population was defined as all enrolled participants who received at least one study drug infusion (full or partial).

Anti-velaglucerase alfa antibody included anti-velaglucerase antibodies (ADA) and neutralizing anti-velaglucerase antibodies (NAb). The Anti-velaglucerase antibody status was summarized as categorical variable by positive and negative. Number of participants who developed positive anti-velaglucerase alfa antibody were reported.

Outcome measures

Outcome measures
Measure	Velaglucerase Alfa 60 U/kg n=21 Participants Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Number of Participants Who Developed Positive Anti-velaglucerase Alfa Antibody Status	1 Participants

Adverse Events

Velaglucerase Alfa 60 U/kg

Serious events: 2 serious events

Other events: 20 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Velaglucerase Alfa 60 U/kg n=21 participants at risk Subjects received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Infections and infestations Pyelonephritis	4.8% 1/21 • Number of events 1 • From start of study drug infusion up to follow-up (107 weeks)
Injury, poisoning and procedural complications Foot fracture	4.8% 1/21 • Number of events 1 • From start of study drug infusion up to follow-up (107 weeks)

Other adverse events

Other adverse events
Measure	Velaglucerase Alfa 60 U/kg n=21 participants at risk Subjects received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other Week (EOW) for 24 months (up to 101 weeks).
Musculoskeletal and connective tissue disorders Back pain	52.4% 11/21 • Number of events 20 • From start of study drug infusion up to follow-up (107 weeks)
Musculoskeletal and connective tissue disorders Pain in extremity	38.1% 8/21 • Number of events 15 • From start of study drug infusion up to follow-up (107 weeks)
Musculoskeletal and connective tissue disorders Arthralgia	23.8% 5/21 • Number of events 6 • From start of study drug infusion up to follow-up (107 weeks)
Musculoskeletal and connective tissue disorders Myalgia	19.0% 4/21 • Number of events 5 • From start of study drug infusion up to follow-up (107 weeks)
Musculoskeletal and connective tissue disorders Musculoskeletal pain	14.3% 3/21 • Number of events 3 • From start of study drug infusion up to follow-up (107 weeks)
Musculoskeletal and connective tissue disorders Bone pain	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)
Musculoskeletal and connective tissue disorders Joint swelling	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)
Musculoskeletal and connective tissue disorders Muscle spasms	9.5% 2/21 • Number of events 4 • From start of study drug infusion up to follow-up (107 weeks)
Nervous system disorders Headache	28.6% 6/21 • Number of events 15 • From start of study drug infusion up to follow-up (107 weeks)
Nervous system disorders Dizziness	23.8% 5/21 • Number of events 11 • From start of study drug infusion up to follow-up (107 weeks)
General disorders Fatigue	23.8% 5/21 • Number of events 6 • From start of study drug infusion up to follow-up (107 weeks)
General disorders Asthenia	14.3% 3/21 • Number of events 3 • From start of study drug infusion up to follow-up (107 weeks)
General disorders Influenza like illness	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)
General disorders Injection site bruising	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)
General disorders Non-cardiac chest pain	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)
General disorders Pain	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	28.6% 6/21 • Number of events 11 • From start of study drug infusion up to follow-up (107 weeks)
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	14.3% 3/21 • Number of events 3 • From start of study drug infusion up to follow-up (107 weeks)
Respiratory, thoracic and mediastinal disorders Cough	9.5% 2/21 • Number of events 3 • From start of study drug infusion up to follow-up (107 weeks)
Respiratory, thoracic and mediastinal disorders Nasal congestion	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)
Infections and infestations Nasopharyngitis	23.8% 5/21 • Number of events 6 • From start of study drug infusion up to follow-up (107 weeks)
Infections and infestations Pneumonia	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)
Infections and infestations Upper respiratory tract infection	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)
Infections and infestations Urinary tract infection	9.5% 2/21 • Number of events 3 • From start of study drug infusion up to follow-up (107 weeks)
Gastrointestinal disorders Gastrooesophageal reflux disease	14.3% 3/21 • Number of events 4 • From start of study drug infusion up to follow-up (107 weeks)
Gastrointestinal disorders Nausea	14.3% 3/21 • Number of events 4 • From start of study drug infusion up to follow-up (107 weeks)
Gastrointestinal disorders Constipation	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)
Injury, poisoning and procedural complications Infusion related reaction	9.5% 2/21 • Number of events 4 • From start of study drug infusion up to follow-up (107 weeks)
Investigations Blood iron decreased	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)
Investigations C-reactive protein increased	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)
Eye disorders Eye pain	14.3% 3/21 • Number of events 3 • From start of study drug infusion up to follow-up (107 weeks)
Metabolism and nutrition disorders Vitamin D deficiency	19.0% 4/21 • Number of events 4 • From start of study drug infusion up to follow-up (107 weeks)
Psychiatric disorders Depression	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)
Cardiac disorders Bundle branch block right	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)
Gastrointestinal disorders Dyspepsia	9.5% 2/21 • Number of events 4 • From start of study drug infusion up to follow-up (107 weeks)
Skin and subcutaneous tissue disorders Erythema	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)
Skin and subcutaneous tissue disorders Urticaria	9.5% 2/21 • Number of events 2 • From start of study drug infusion up to follow-up (107 weeks)

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee 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.
Publication restrictions are in place

Restriction type: OTHER