Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2022-002323-35 | EudraCT Number |
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The main purpose of this study is to observe the side effects of VPRIV in participants with type 1 Gaucher disease who are either treatment-naïve (newly diagnosed) or who are currently being treated with enzyme replacement therapy (ERT).
Participants will receive VPRIV intravenously during the treatment period (up to 51 weeks), followed by the end-of-treatment (EOT) visit after 2 weeks.
The drug being tested in this study is called Velaglucerase Alfa (VPRIV). VPRIV will be tested to treat participants with type 1 Gaucher disease. This study will look at the safety, efficacy and pharmacokinetics of VPRIV in the treatment of type 1 Gaucher disease.
The study will enroll approximately 20 participants with Gaucher disease. The study comprises a screening period (Day -21 through Day -4), baseline period (Day -3 through Day 0), treatment period (Week 1 to Week 51), and safety follow-up period. Participants will be assigned to the following drug administration:
• Velaglucerase Alfa (VPRIV)
Participants will receive VPRIV as intravenous (IV) infusion every other week from Week 1 through Week 51 during the Treatment Period. Percentage of participants with at least one serious treatment-emergent adverse event (TEAE) will be evaluated throughout the study.
This multi-center trial will be conducted in China. The overall time to participate in this study is approximately 59 weeks. Participants will make a safety follow-up telephone call or visit to the site after 30 (±7) days of the last infusion of the study drug.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Velaglucerase Alfa (VPRIV) | Experimental | Participants received VPRIV IV infusion at 60 units per kilogram (U/kg) body weight once every other week (EOW) for 60 (+10) minutes for up to 51 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Velaglucerase Alfa | Drug | VPRIV intravenous infusion every other week for 60 minutes. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With at Least One Serious Treatment-Emergent Adverse Event (TEAE) | Adverse event(AE)=any untoward medical occurrence in clinical investigation participant administered a drug;it does not necessarily have to have causal relationship with this treatment. AE can therefore be any unfavorable&unintended sign (example,clinically significant abnormal laboratory value),symptom/disease temporally associated with use of drug whether or not it is considered related to drug. TEAE=any event emerging or manifesting at or after initiation of investigational product or any existing event that worsens in either intensity or frequency following exposure to investigational product. SAE=any untoward clinical manifestation of signs, symptoms, or outcomes(whether considered related to investigational product or not)&at any dose: results in death,is life-threatening,requires in-patient hospitalization/prolongation of hospitalization,results in persistent/significant disability/incapacity,results in congenital abnormality/birth defect,or is an important medical event. | Up to 56.2 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With TEAEs | An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. A TEAE is defined as any event emerging or manifesting at or after the initiation of the investigational product or any existing event that worsens in either intensity or frequency following exposure to the investigational product. |
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Inclusion:
Has a documented, confirmed diagnosis of type 1 Gaucher disease based on the following, as determined by the investigator:
Is at least 2 years of age, inclusive, at screening
Is naive to treatment for Gaucher disease (Has not received treatment for Gaucher disease [investigational or approved products] within the 12 months prior to screening) OR Is receiving or has recently received Imiglucerase ERT (Has received Imiglucerase treatment within the 12 months prior to screening and not within the 14 days prior to screening)
Has Gaucher disease-related hematological abnormalities, defined as
Has Gaucher disease-related viscera abnormalities, defined as the following:
Exclusion:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Chinese PLA General Hospital | Beijing | Beijing Municipality | 100039 | China | ||
| Beijing Children's Hospital, Capital Medical University |
Not provided
| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link | View source |
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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.
Participants with a diagnosis of type 1 Gaucher disease were enrolled in this study to receive velaglucerase alfa (VPRIV) as intravenous (IV) infusion.
Participants took part in the study at various investigative sites in China from 3 January 2023 to 5 August 2024.
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| ID | Title | Description |
|---|---|---|
| FG000 | VPRIV | Participants received VPRIV IV infusion at 60 units per kilogram (U/kg) body weight once every other week (EOW) for 60 (+10) minutes up to Week 51. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
The Intent-to-Treat (ITT) Set included all participants who signed the informed consent form (ICF) [and assent form, if applicable] and were eligible for the study based on the defined inclusion/exclusion criteria.
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| ID | Title | Description |
|---|---|---|
| BG000 | VPRIV | Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | Percentage of Participants With at Least One Serious Treatment-Emergent Adverse Event (TEAE) | Adverse event(AE)=any untoward medical occurrence in clinical investigation participant administered a drug;it does not necessarily have to have causal relationship with this treatment. AE can therefore be any unfavorable&unintended sign (example,clinically significant abnormal laboratory value),symptom/disease temporally associated with use of drug whether or not it is considered related to drug. TEAE=any event emerging or manifesting at or after initiation of investigational product or any existing event that worsens in either intensity or frequency following exposure to investigational product. SAE=any untoward clinical manifestation of signs, symptoms, or outcomes(whether considered related to investigational product or not)&at any dose: results in death,is life-threatening,requires in-patient hospitalization/prolongation of hospitalization,results in persistent/significant disability/incapacity,results in congenital abnormality/birth defect,or is an important medical event. | The Safety Set (SAF) included all participants in the ITT Set who received at least 1 dose of VPRIV. | Posted | Number | percentage of participants | Up to 56.2 weeks |
Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
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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 | Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diabetes mellitus inadequate control | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Conjunctivitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Takeda | +1-877-825-3327 | TrialDisclosures@takeda.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Aug 2, 2022 | Feb 4, 2025 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 13, 2024 | Feb 4, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D005776 | Gaucher Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
Not provided
Not provided
| ID | Term |
|---|---|
| D005962 | Glucosylceramidase |
| ID | Term |
|---|---|
| D005959 | Glucosidases |
| D006026 | Glycoside Hydrolases |
| D006867 | Hydrolases |
| D004798 | Enzymes |
Not provided
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| Up to 56.2 weeks |
| Percentage of Participants With Infusion-related Reactions Reported as an Adverse Event | An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. An infusion-related AE was defined as an AE that 1) began either during or within 12 hours after the start of the infusion and 2) was judged as possibly or probably related to the study treatment. | Up to 56.2 weeks |
| Percentage of Participants With Development of Anti-VPRIV Antibodies and Neutralizing Antibodies at Week 53 | Percentages were rounded off to the nearest single decimal place. | Week 53 |
| Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53 | Clinical laboratory assessments included hematology and serum chemistry. Any clinically significant changes in the clinical laboratory assessment values based on the investigator's interpretation were reported. Only categories with at least one participant with event are presented. | Week 53 |
| Number of Participants With Abnormal Changes in Laboratory Assessments at Week 53: Urinalysis | Any abnormal changes in the urinalysis assessment values based on the investigator's interpretation were reported. Urinalysis comprised of the following parameters: bilirubin, ketones, glucose, nitrite, occult blood, protein, and urobilinogen. | Week 53 |
| Number of Participants With at Least One Abnormal Change in an Infusion Vital Sign Parameter at Week 53 | Participants with atleast 1 abnormal change (above or below normal) in an infusion vital sign parameter of pulse, temperature, respiratory rate,& blood pressure were reported. Normal ranges for each vital sign parameter were, pulse (beats per minutes [bpm]): 40-100(≥12 years old), 55-95(≥6 but <12 years old), 65-110(≥2 but <6 years old); body temperature(degree Celsius[˚C]): 36.5 to 37.2(all age groups),respiration rate(breaths/minutes):12-24(≥12 years old),12-22 (≥6 but <12 years old),20-30(≥2 but <6 years old);systolic blood pressure(BP) [millimeters of mercury{mm Hg}]: high: ≥140 (≥18 years old), ≥20+ 80+ 2*age(<18 years old), low: <90 (≥18 years old), < -20+ 80+ 2*age(<18 years old); diastolic BP(mm Hg):high:≥90 (≥18 years old), ≥20+ (80+2*age)*(2/3) (<18 years old) low: <50 (≥18 years old), < -20+ (80+2*age)*(2/3) (<18 years old). As per planned analysis,data was collected in a combined manner for all participants irrespective of age. Only non-zero categories are presented. | Week 53 |
| Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Measurements at Week 53 | Participants with clinically significant changes in any ECG measurement, such as PR, QRS, QT, corrected QT intervals, and heart rate based on the investigator's interpretation were reported. | Week 53 |
| Change From Baseline to Week 53 in Hemoglobin Concentration | Baseline, Week 53 |
| Change From Baseline to Week 53 in Platelet Count | Baseline, Week 53 |
| Change From Baseline to Week 53 in Normalized Liver Volume | Normalized liver volume was measured by abdominal magnetic resonance imaging (MRI) or computed tomography (CT) scan. Liver volume was normalized for percent body weight. | Baseline, Week 53 |
| Change From Baseline to Week 53 in Normalized Spleen Volume | Spleen volume was normalized for percent body weight. | Baseline, Week 53 |
| Change From Baseline to Week 53 in 36-item Short Form General Health Survey (SF-36) Scores | SF-36 Version 2 is a multipurpose, participant completed, short-form health survey with 36 questions that consists of an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures. Physical component summary (PCS) is mostly contributed by physical function (PF), role physical (RP), bodily pain (BP), and general health (GH). Mental component summary (MCS) is mostly contributed by mental health (MH), role emotional (RE), social function (SF), and vitality (VT). Each component on the SF-36 item health survey is scored from 0 (best) to 100 (worst). Total score ranges from 0-100 for each component summary (i.e., PCS and MCS), where higher scores are associated with less disability and better quality of life. | Baseline, Week 53 |
| Change From Baseline to Week 53 in Childhood Health Questionnaire-Parent Form 50 (CHQ-PF50) Scores | The CHQ-PF-50 is a 50-item questionnaire to be completed by the parents or guardians of children between 5 and 18 years of age. The 50 questions measure 14 domains (global health, physical functioning, role/social limitations (emotional/behavioral and physical), bodily pain/discomfort, behavior, global behavior, mental health, self-esteem, general health perceptions, change in health, parental impact (emotional and time), family activities, and family cohesion) which were summarized as the physical and psychological summary scores. Each summary score was transformed and could range from 0 to 100, with higher score indicating better physical and psychosocial health. A positive change from Baseline indicates improved well-being. | Baseline, Week 53 |
| Cmax: Maximum Observed Serum Concentration for VPRIV at Week 1 | Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1 |
| Serum Concentration for VPRIV at Week 37 | Within 3 minutes prior to the end of the 60-minute infusion at Week 37 |
| Tmax: Time to Reach the Maximum Serum Concentration (Cmax) for VPRIV | Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1 |
| AUCinf: Area Under the Plasma Concentration-time Curve From Time 0 to Infinity for VPRIV | ng*min/mL denotes nanograms*minutes per milliliter. | Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1 |
| Terminal Phase Elimination Half-life (T1/2) for VPRIV | Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1 |
| Oral Clearance (CL) for VPRIV | mL/min/kg denotes milliliters per minutes per kilogram. | Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1 |
| Apparent Steady-state Volume of Distribution (Vss) for VPRIV | Pre-dose and at multiple timepoints(up to 120 minutes post-dose on Day 1 of Week 1 |
| Percent Change From Baseline to Week 53 in Biomarker: Plasma Chemokine [C-C Motif] Ligand 18 | Baseline, Week 53 |
| Percent Change From Baseline to Week 53 in Biomarker: Glucopsychosine | Baseline, Week 53 |
| Beijing |
| Beijing Municipality |
| 100045 |
| China |
| Peking Union Medical College Hospital, Chinese Academy of Medical Sciences | Beijing | Beijing Municipality | 100730 | China |
| Lanzhou University Second Hospital | Lanzhou | Gansu | 730030 | China |
| The First Affiliated Hospital, Sun Yat-sen University | Guangzhou | Guangdong | 510080 | China |
| Guangzhou Women and Children's Medical Center | Guangzhou | Guangdong | 510623 | China |
| The Second Hospital of Hebei Medical University | Shijiazhuang | Hebei | 050000 | China |
| Tongji Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology | Wuhan | Hubei | 430030 | China |
| Nanjing Children's Hospital | Nanjing | Jiangsu | 210008 | China |
| Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences | Tianjin | Tianjin Municipality | 300020 | China |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| ID | Title | Description |
|---|---|---|
| OG000 | VPRIV | Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51. |
|
|
| Secondary | Percentage of Participants With TEAEs | An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. A TEAE is defined as any event emerging or manifesting at or after the initiation of the investigational product or any existing event that worsens in either intensity or frequency following exposure to the investigational product. | The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV. | Posted | Number | percentage of participants | Up to 56.2 weeks |
|
|
|
| Secondary | Percentage of Participants With Infusion-related Reactions Reported as an Adverse Event | An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. An infusion-related AE was defined as an AE that 1) began either during or within 12 hours after the start of the infusion and 2) was judged as possibly or probably related to the study treatment. | The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV. | Posted | Number | percentage of participants | Up to 56.2 weeks |
|
|
|
| Secondary | Percentage of Participants With Development of Anti-VPRIV Antibodies and Neutralizing Antibodies at Week 53 | Percentages were rounded off to the nearest single decimal place. | The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV. Overall number analyzed is the number of participants with data available for analyses. | Posted | Number | percentage of participants | Week 53 |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53 | Clinical laboratory assessments included hematology and serum chemistry. Any clinically significant changes in the clinical laboratory assessment values based on the investigator's interpretation were reported. Only categories with at least one participant with event are presented. | The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV. Overall number of participants analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis for the specified category. | Posted | Count of Participants | Participants | Week 53 |
|
|
|
| Secondary | Number of Participants With Abnormal Changes in Laboratory Assessments at Week 53: Urinalysis | Any abnormal changes in the urinalysis assessment values based on the investigator's interpretation were reported. Urinalysis comprised of the following parameters: bilirubin, ketones, glucose, nitrite, occult blood, protein, and urobilinogen. | The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV. Overall number of participants analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis for the specified category. | Posted | Count of Participants | Participants | Week 53 |
|
|
|
| Secondary | Number of Participants With at Least One Abnormal Change in an Infusion Vital Sign Parameter at Week 53 | Participants with atleast 1 abnormal change (above or below normal) in an infusion vital sign parameter of pulse, temperature, respiratory rate,& blood pressure were reported. Normal ranges for each vital sign parameter were, pulse (beats per minutes [bpm]): 40-100(≥12 years old), 55-95(≥6 but <12 years old), 65-110(≥2 but <6 years old); body temperature(degree Celsius[˚C]): 36.5 to 37.2(all age groups),respiration rate(breaths/minutes):12-24(≥12 years old),12-22 (≥6 but <12 years old),20-30(≥2 but <6 years old);systolic blood pressure(BP) [millimeters of mercury{mm Hg}]: high: ≥140 (≥18 years old), ≥20+ 80+ 2*age(<18 years old), low: <90 (≥18 years old), < -20+ 80+ 2*age(<18 years old); diastolic BP(mm Hg):high:≥90 (≥18 years old), ≥20+ (80+2*age)*(2/3) (<18 years old) low: <50 (≥18 years old), < -20+ (80+2*age)*(2/3) (<18 years old). As per planned analysis,data was collected in a combined manner for all participants irrespective of age. Only non-zero categories are presented. | The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV. Number analyzed is the number of participants with data available for analysis for the specified category. | Posted | Count of Participants | Participants | Week 53 |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Measurements at Week 53 | Participants with clinically significant changes in any ECG measurement, such as PR, QRS, QT, corrected QT intervals, and heart rate based on the investigator's interpretation were reported. | The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV. Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Count of Participants | Participants | Week 53 |
|
|
|
| Secondary | Change From Baseline to Week 53 in Hemoglobin Concentration | The ITT Set included all participants who signed the ICF (and assent form, if applicable) and were eligible for the study based on the defined inclusion/exclusion criteria. Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | grams per deciliter (g/dL) | Baseline, Week 53 |
|
|
|
| Secondary | Change From Baseline to Week 53 in Platelet Count | The ITT Set included all participants who signed the ICF (and assent form, if applicable) and were eligible for the study based on the defined inclusion/exclusion criteria. Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | platelets*10^9/liter | Baseline, Week 53 |
|
|
|
| Secondary | Change From Baseline to Week 53 in Normalized Liver Volume | Normalized liver volume was measured by abdominal magnetic resonance imaging (MRI) or computed tomography (CT) scan. Liver volume was normalized for percent body weight. | The ITT Set included all participants who signed the ICF (and assent form, if applicable) and were eligible for the study based on the defined inclusion/exclusion criteria. Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | percentage of body weight (%BW) | Baseline, Week 53 |
|
|
|
| Secondary | Change From Baseline to Week 53 in Normalized Spleen Volume | Spleen volume was normalized for percent body weight. | The ITT Set included all participants who signed the ICF (and assent form, if applicable) and were eligible for the study based on the defined inclusion/exclusion criteria. Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | % BW | Baseline, Week 53 |
|
|
|
| Secondary | Change From Baseline to Week 53 in 36-item Short Form General Health Survey (SF-36) Scores | SF-36 Version 2 is a multipurpose, participant completed, short-form health survey with 36 questions that consists of an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures. Physical component summary (PCS) is mostly contributed by physical function (PF), role physical (RP), bodily pain (BP), and general health (GH). Mental component summary (MCS) is mostly contributed by mental health (MH), role emotional (RE), social function (SF), and vitality (VT). Each component on the SF-36 item health survey is scored from 0 (best) to 100 (worst). Total score ranges from 0-100 for each component summary (i.e., PCS and MCS), where higher scores are associated with less disability and better quality of life. | The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV. Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Week 53 |
|
|
|
| Secondary | Change From Baseline to Week 53 in Childhood Health Questionnaire-Parent Form 50 (CHQ-PF50) Scores | The CHQ-PF-50 is a 50-item questionnaire to be completed by the parents or guardians of children between 5 and 18 years of age. The 50 questions measure 14 domains (global health, physical functioning, role/social limitations (emotional/behavioral and physical), bodily pain/discomfort, behavior, global behavior, mental health, self-esteem, general health perceptions, change in health, parental impact (emotional and time), family activities, and family cohesion) which were summarized as the physical and psychological summary scores. Each summary score was transformed and could range from 0 to 100, with higher score indicating better physical and psychosocial health. A positive change from Baseline indicates improved well-being. | The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV. Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Week 53 |
|
|
|
| Secondary | Cmax: Maximum Observed Serum Concentration for VPRIV at Week 1 | The Pharmacokinetic (PK) Set included all naïve participants in the ITT set who received at least 1 dose of VPRIV and provided evaluable PK concentration data. | Posted | Mean | Standard Deviation | nanograms per milliliter (ng/mL) | Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1 |
|
|
|
| Secondary | Serum Concentration for VPRIV at Week 37 | The PK Set included all naïve participants in the ITT set who received at least 1 dose of VPRIV and provided evaluable PK concentration data. Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | ng/mL | Within 3 minutes prior to the end of the 60-minute infusion at Week 37 |
|
|
|
| Secondary | Tmax: Time to Reach the Maximum Serum Concentration (Cmax) for VPRIV | The PK Set included all naïve participants in the ITT set who received at least 1 dose of VPRIV and provided evaluable PK concentration data. | Posted | Median | Full Range | minutes (min) | Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1 |
|
|
|
| Secondary | AUCinf: Area Under the Plasma Concentration-time Curve From Time 0 to Infinity for VPRIV | ng*min/mL denotes nanograms*minutes per milliliter. | The PK Set included all naïve participants in the ITT set who received at least 1 dose of VPRIV and provided evaluable PK concentration data. | Posted | Mean | Standard Deviation | ng*min/mL | Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1 |
|
|
|
| Secondary | Terminal Phase Elimination Half-life (T1/2) for VPRIV | The PK Set included all naïve participants in the ITT set who received at least 1 dose of VPRIV and provided evaluable PK concentration data. | Posted | Mean | Standard Deviation | min | Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1 |
|
|
|
| Secondary | Oral Clearance (CL) for VPRIV | mL/min/kg denotes milliliters per minutes per kilogram. | The PK Set included all naïve participants in the ITT set who received at least 1 dose of VPRIV and provided evaluable PK concentration data. | Posted | Mean | Standard Deviation | mL/min/kg | Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1 |
|
|
|
| Secondary | Apparent Steady-state Volume of Distribution (Vss) for VPRIV | The PK Set included all naïve participants in the ITT set who received at least 1 dose of VPRIV and provided evaluable PK concentration data. | Posted | Mean | Standard Deviation | milliliters per kilogram (mL/kg) | Pre-dose and at multiple timepoints(up to 120 minutes post-dose on Day 1 of Week 1 |
|
|
|
| Secondary | Percent Change From Baseline to Week 53 in Biomarker: Plasma Chemokine [C-C Motif] Ligand 18 | The ITT Set included all participants who signed the ICF (and assent form, if applicable) and were eligible for the study based on the defined inclusion/exclusion criteria. Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | percent change | Baseline, Week 53 |
|
|
|
| Secondary | Percent Change From Baseline to Week 53 in Biomarker: Glucopsychosine | The ITT Set included all participants who signed the ICF (and assent form, if applicable) and were eligible for the study based on the defined inclusion/exclusion criteria. Overall number of participants analyzed is the number of participants with data available for analyses. | Posted | Mean | Standard Deviation | percent change | Baseline, Week 53 |
|
|
|
| 0 |
| 20 |
| 4 |
| 20 |
| 19 |
| 20 |
| Pneumonia | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
|
| Pneumonia mycoplasmal | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
|
| Splenic injury | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
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| Hypertriglyceridaemia | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
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| Osteoporosis | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 27.0 | Systematic Assessment |
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| Supraventricular extrasystoles | Cardiac disorders | MedDRA 27.0 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
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| Urinary occult blood positive | Investigations | MedDRA 27.0 | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
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Not provided
Not provided
| 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 |
| D045762 |
| Enzymes and Coenzymes |
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| Hematology: Erythrocytes Mean Corpuscular Volume |
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| Hematology: Erythrocytes |
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| Hematology: Hematocrit |
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| Hematology: Hemoglobin |
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| Hematology: Leukocytes |
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| Hematology: Lymphocytes |
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| Hematology: Monocytes |
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| Hematology: Neutrophils |
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| Hematology: Platelets |
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| Serum Chemistry: Bilirubin |
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| Serum Chemistry: Ferritin |
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| Serum Chemistry: Iron |
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| Urinalysis: Glucose |
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| Urinalysis: Nitrite |
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| Urinalysis: Occult Blood |
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| Urinalysis: Protein |
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| Urinalysis: Urobilinogen |
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| Body Temperature: Below Normal |
|
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| Respiratory Rate: Above Normal |
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| Systolic BP: Above Normal |
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| Systolic BP: Below Normal |
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| Diastolic BP: Above Normal |
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| Diastolic BP: Below Normal |
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