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| ID | Type | Description | Link |
|---|---|---|---|
| 2015-001578-17 | EudraCT Number |
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The primary purpose of this study is to evaluate the effect of VPRIV therapy (60 units per kilogram [U/kg] every other week [EOW]) in treatment-naive participants with type 1 Gaucher disease on change from baseline in lumbar spine (LS) bone mineral density (BMD) Z-score as measured by dual energy x-ray absorptiometry (DXA) after 24 months of treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Velaglucerase alfa 60 U/kg | Experimental | Participants will receive 60-minute intravenous infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other week (EOW) and an oral daily dose of 800 IU vitamin D for 24 months (101 weeks). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Velaglucerase alfa | Drug | Participants will receive 60-minute intravenous infusion of 60 U/kg velaglucerase alfa EOW. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-Score up to End of Study (EOS) (Week 103) | 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. | Baseline up to EOS (Week 103) |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Lumbar Spine (LS) BMD Z-score at Week 51 | 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. |
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Inclusion Criteria:
Exclusion criteria
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Shire | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cedars Sinai Medical Center | Beverly Hills | California | 90211 | United States | ||
| Kaiser Permanente |
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
A total of 21 participants were enrolled and received treatment in this study.
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).
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| ID | Title | Description |
|---|---|---|
| FG000 | 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). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 2, 2020 | Nov 7, 2021 |
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| Vitamin D | Dietary Supplement | Participants will receive 800 IU vitamin D orally daily. |
|
| Baseline, Week 51 |
| Change From Baseline in Lumbar Spine BMD at 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. | Baseline, Week 51 and EOS (Week 103) |
| Change From Baseline in Total Bone Marrow Burden (BMB) Score at 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. | Baseline, Week 51 and EOS (Week 103) |
| Change From Baseline in Hemoglobin Concentration at 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. | Baseline, Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) |
| Change From Baseline in Platelet Count at 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. | Baseline, Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) |
| Change From Baseline in Normalized Liver Volume at 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. | Baseline, Week 51 and EOS (Week 103) |
| Change From Baseline in Normalized Spleen Volume at 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. | Baseline, Week 51 and EOS (Week 103) |
| Change From Baseline in Severity of Bone Pain at 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. | Baseline, Week 51 and EOS (Week 103) |
| Change From Baseline in Bone Pain Interference Score at 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. | Baseline, Week 51 and EOS (Week 103) |
| Change From Baseline in Overall Fatigue Measured by Brief Fatigue Inventory (BFI) at 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". | Baseline, Week 51 and EOS (Week 103) |
| Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at 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. | Baseline, Week 51 and EOS (Week 103) |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | 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. | From start of study drug infusion up to follow-up (107 weeks) |
| Number of Participants Who Developed Positive Anti-velaglucerase Alfa Antibody Status | 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. | Baseline up to EOS (Week 103) |
| Los Angeles |
| California |
| 90027 |
| United States |
| Emory Genetics | Atlanta | Georgia | 30322 | United States |
| Ann and Robert H Lurie Childrens Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| NYU School of Medicine | New York | New York | 10016 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Lysosomal and Rare Disorders Research and Treatment Center | Fairfax | Virginia | 22030 | United States |
| Rambam Health Care Campus | Haifa | 3109601 | Israel |
| Shaare Zedek Medical Center | Jerusalem | 91031 | Israel |
| Rabin Medical Center | Petah Tikva | 49100 | Israel |
| Hospital Universitario Ramon y Cajal | Madrid | 28034 | Spain |
| Hospital Quironsalud Zaragoza | Zaragoza | 50006 | Spain |
| Addenbrooke's Hospital | Cambridge | CB20QQ | United Kingdom |
| Royal Free Hospital | London | NW3 2QG | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Safety population was defined as all enrolled participants who received at least one study drug infusion (full or partial).
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| ID | Title | Description |
|---|---|---|
| BG000 | Velaglucerase Alfa 60 U/kg | Participants received 60-minute intravenous (IV) infusion of 60 units per kilogram (U/kg) velaglucerase alfa EOW for 24 months (up to 101 weeks). |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-Score up to End of Study (EOS) (Week 103) | 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. | 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. | Posted | Mean | Standard Deviation | Z-score | Baseline up to EOS (Week 103) |
|
|
| |||||||||||||||||||||||||
| Secondary | Change From Baseline in Lumbar Spine (LS) BMD Z-score at Week 51 | 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. | 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. | Posted | Mean | Standard Deviation | Z-Score | Baseline, Week 51 |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Lumbar Spine BMD at 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. | 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. | Posted | Mean | Standard Deviation | g/cm^2 | Baseline, Week 51 and EOS (Week 103) |
|
| ||||||||||||||||||||||||||
| Secondary | Change From Baseline in Total Bone Marrow Burden (BMB) Score at 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. | 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 and "number analyzed" signifies participants evaluable at specific time point. | Posted | Mean | Standard Deviation | Score on a Scale | Baseline, Week 51 and EOS (Week 103) |
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| Secondary | Change From Baseline in Hemoglobin Concentration at 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. | 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 and "number analyzed" signifies participants evaluable at specific time point. | Posted | Mean | Standard Deviation | Grams per deciliter (g/dL) | Baseline, Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) |
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| Secondary | Change From Baseline in Platelet Count at 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. | 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 and "number analyzed" signifies participants evaluable at specific time point. | Posted | Mean | Standard Deviation | 10^9 platelets per liter | Baseline, Week 13, 25, 37, 51, 65, 77, 89, and EOS (Week 103) |
|
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| Secondary | Change From Baseline in Normalized Liver Volume at 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. | 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 and "number analyzed" signifies participants evaluable at specific time point. | Posted | Mean | Standard Deviation | Percent body weight | Baseline, Week 51 and EOS (Week 103) |
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| Secondary | Change From Baseline in Normalized Spleen Volume at 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. | 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 and "number analyzed" signifies participants evaluable at specific time point. | Posted | Mean | Standard Deviation | Percent body weight | Baseline, Week 51 and EOS (Week 103) |
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| Secondary | Change From Baseline in Severity of Bone Pain at 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. | 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. | Posted | Mean | Standard Deviation | Score on a Scale | Baseline, Week 51 and EOS (Week 103) |
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| Secondary | Change From Baseline in Bone Pain Interference Score at 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. | 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. | Posted | Mean | Standard Deviation | Score on a Scale | Baseline, Week 51 and EOS (Week 103) |
| |||||||||||||||||||||||||||
| Secondary | Change From Baseline in Overall Fatigue Measured by Brief Fatigue Inventory (BFI) at 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". | 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. | Posted | Mean | Standard Deviation | Score on a Scale | Baseline, Week 51 and EOS (Week 103) |
| |||||||||||||||||||||||||||
| Secondary | Number of Participants With Shift in World Health Organization (WHO) BMD Classifications Based on LS T-Scores at 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. | 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 and "number analyzed" signifies participants evaluable at specific time point. | Posted | Count of Participants | Participants | Baseline, Week 51 and EOS (Week 103) |
|
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| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | 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. | Safety population was defined as all enrolled participants who received at least one study drug infusion (full or partial). | Posted | Count of Participants | Participants | From start of study drug infusion up to follow-up (107 weeks) |
|
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| Secondary | Number of Participants Who Developed Positive Anti-velaglucerase Alfa Antibody Status | 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. | Safety population was defined as all enrolled participants who received at least one study drug infusion (full or partial). | Posted | Count of Participants | Participants | Baseline up to EOS (Week 103) |
|
|
From start of study drug infusion up to follow-up (107 weeks)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Velaglucerase Alfa 60 U/kg | 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). | 0 | 21 | 2 | 21 | 20 | 21 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyelonephritis | Infections and infestations | MedDRA18.1 | Non-systematic Assessment |
| |
| Foot fracture | Injury, poisoning and procedural complications | MedDRA18.1 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Asthenia | General disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Injection site bruising | General disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Pain | General disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA18.1 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA18.1 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA18.1 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA18.1 | Non-systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA18.1 | Non-systematic Assessment |
| |
| Blood iron decreased | Investigations | MedDRA18.1 | Non-systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA18.1 | Non-systematic Assessment |
| |
| Eye pain | Eye disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Bundle branch block right | Cardiac disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA18.1 | Non-systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA18.1 | Non-systematic Assessment |
|
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 30, 2021 | Jan 3, 2022 | SAP_001.pdf |
| ID | Term |
|---|---|
| D005776 | Gaucher Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |
Not provided
Not provided
| ID | Term |
|---|---|
| D005962 | Glucosylceramidase |
| D014807 | Vitamin D |
| ID | Term |
|---|---|
| D005959 | Glucosidases |
| D006026 | Glycoside Hydrolases |
| D006867 | Hydrolases |
| D004798 | Enzymes |
| D045762 | Enzymes and Coenzymes |
| D012632 | Secosteroids |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
Not provided
Not provided
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Unknown or Not Reported |
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