Trial Outcomes & Findings for A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease (NCT NCT05529992)
NCT ID: NCT05529992
Last Updated: 2025-06-26
Results Overview
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.
COMPLETED
PHASE3
20 participants
Up to 56.2 weeks
2025-06-26
Participant Flow
Participants took part in the study at various investigative sites in China from 3 January 2023 to 5 August 2024.
Participants with a diagnosis of type 1 Gaucher disease were enrolled in this study to receive velaglucerase alfa (VPRIV) as intravenous (IV) infusion.
Participant milestones
| Measure |
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.
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
COMPLETED
|
19
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
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.
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease
Baseline characteristics by cohort
| Measure |
VPRIV
n=20 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Age, Continuous
|
14.3 years
STANDARD_DEVIATION 9.68 • n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
20 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
20 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 56.2 weeksPopulation: The Safety Set (SAF) included all participants in the ITT Set who received at least 1 dose of VPRIV.
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.
Outcome measures
| Measure |
VPRIV
n=20 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Percentage of Participants With at Least One Serious Treatment-Emergent Adverse Event (TEAE)
|
20 percentage of participants
|
SECONDARY outcome
Timeframe: Up to 56.2 weeksPopulation: The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
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.
Outcome measures
| Measure |
VPRIV
n=20 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Percentage of Participants With TEAEs
|
95 percentage of participants
|
SECONDARY outcome
Timeframe: Up to 56.2 weeksPopulation: The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
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.
Outcome measures
| Measure |
VPRIV
n=20 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Percentage of Participants With Infusion-related Reactions Reported as an Adverse Event
|
5 percentage of participants
|
SECONDARY outcome
Timeframe: Week 53Population: 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.
Percentages were rounded off to the nearest single decimal place.
Outcome measures
| Measure |
VPRIV
n=19 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Percentage of Participants With Development of Anti-VPRIV Antibodies and Neutralizing Antibodies at Week 53
Anti-VPRIV Antibodies
|
15.8 percentage of participants
|
|
Percentage of Participants With Development of Anti-VPRIV Antibodies and Neutralizing Antibodies at Week 53
Neutralizing Antibodies
|
10.5 percentage of participants
|
SECONDARY outcome
Timeframe: Week 53Population: 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.
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.
Outcome measures
| Measure |
VPRIV
n=19 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53
Hematology: Erythrocytes Mean Corpuscular Volume
|
2 Participants
|
|
Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53
Hematology: Erythrocytes
|
2 Participants
|
|
Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53
Hematology: Hematocrit
|
5 Participants
|
|
Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53
Hematology: Hemoglobin
|
4 Participants
|
|
Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53
Hematology: Leukocytes
|
2 Participants
|
|
Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53
Hematology: Lymphocytes
|
1 Participants
|
|
Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53
Hematology: Erythrocytes Mean Corpuscular Hemoglobin Concentration
|
2 Participants
|
|
Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53
Hematology: Erythrocytes Mean Corpuscular Hemoglobin
|
3 Participants
|
|
Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53
Hematology: Monocytes
|
1 Participants
|
|
Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53
Hematology: Neutrophils
|
2 Participants
|
|
Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53
Hematology: Platelets
|
14 Participants
|
|
Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53
Serum Chemistry: Bilirubin
|
3 Participants
|
|
Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53
Serum Chemistry: Ferritin
|
5 Participants
|
|
Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53
Serum Chemistry: Iron
|
2 Participants
|
SECONDARY outcome
Timeframe: Week 53Population: 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.
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.
Outcome measures
| Measure |
VPRIV
n=19 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Number of Participants With Abnormal Changes in Laboratory Assessments at Week 53: Urinalysis
Urinalysis: Bilirubin
|
1 Participants
|
|
Number of Participants With Abnormal Changes in Laboratory Assessments at Week 53: Urinalysis
Urinalysis: Ketones
|
0 Participants
|
|
Number of Participants With Abnormal Changes in Laboratory Assessments at Week 53: Urinalysis
Urinalysis: Glucose
|
1 Participants
|
|
Number of Participants With Abnormal Changes in Laboratory Assessments at Week 53: Urinalysis
Urinalysis: Nitrite
|
1 Participants
|
|
Number of Participants With Abnormal Changes in Laboratory Assessments at Week 53: Urinalysis
Urinalysis: Occult Blood
|
7 Participants
|
|
Number of Participants With Abnormal Changes in Laboratory Assessments at Week 53: Urinalysis
Urinalysis: Protein
|
3 Participants
|
|
Number of Participants With Abnormal Changes in Laboratory Assessments at Week 53: Urinalysis
Urinalysis: Urobilinogen
|
3 Participants
|
SECONDARY outcome
Timeframe: Week 53Population: 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.
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.
Outcome measures
| Measure |
VPRIV
n=20 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Number of Participants With at Least One Abnormal Change in an Infusion Vital Sign Parameter at Week 53
Body Temperature: Above Normal
|
1 Participants
|
|
Number of Participants With at Least One Abnormal Change in an Infusion Vital Sign Parameter at Week 53
Pulse: Above Normal
|
15 Participants
|
|
Number of Participants With at Least One Abnormal Change in an Infusion Vital Sign Parameter at Week 53
Body Temperature: Below Normal
|
18 Participants
|
|
Number of Participants With at Least One Abnormal Change in an Infusion Vital Sign Parameter at Week 53
Respiratory Rate: Above Normal
|
9 Participants
|
|
Number of Participants With at Least One Abnormal Change in an Infusion Vital Sign Parameter at Week 53
Systolic BP: Above Normal
|
11 Participants
|
|
Number of Participants With at Least One Abnormal Change in an Infusion Vital Sign Parameter at Week 53
Systolic BP: Below Normal
|
5 Participants
|
|
Number of Participants With at Least One Abnormal Change in an Infusion Vital Sign Parameter at Week 53
Diastolic BP: Above Normal
|
8 Participants
|
|
Number of Participants With at Least One Abnormal Change in an Infusion Vital Sign Parameter at Week 53
Diastolic BP: Below Normal
|
3 Participants
|
SECONDARY outcome
Timeframe: Week 53Population: 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.
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.
Outcome measures
| Measure |
VPRIV
n=19 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Measurements at Week 53
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline, Week 53Population: 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.
Outcome measures
| Measure |
VPRIV
n=18 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Change From Baseline to Week 53 in Hemoglobin Concentration
|
2.34 grams per deciliter (g/dL)
Standard Deviation 1.305
|
SECONDARY outcome
Timeframe: Baseline, Week 53Population: 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.
Outcome measures
| Measure |
VPRIV
n=18 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Change From Baseline to Week 53 in Platelet Count
|
42.1 platelets*10^9/liter
Standard Deviation 27.68
|
SECONDARY outcome
Timeframe: Baseline, Week 53Population: 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.
Normalized liver volume was measured by abdominal magnetic resonance imaging (MRI) or computed tomography (CT) scan. Liver volume was normalized for percent body weight.
Outcome measures
| Measure |
VPRIV
n=19 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Change From Baseline to Week 53 in Normalized Liver Volume
|
-1.11 percentage of body weight (%BW)
Standard Deviation 0.917
|
SECONDARY outcome
Timeframe: Baseline, Week 53Population: 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.
Spleen volume was normalized for percent body weight.
Outcome measures
| Measure |
VPRIV
n=19 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Change From Baseline to Week 53 in Normalized Spleen Volume
|
-3.09 % BW
Standard Deviation 1.627
|
SECONDARY outcome
Timeframe: Baseline, Week 53Population: 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.
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.
Outcome measures
| Measure |
VPRIV
n=4 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Change From Baseline to Week 53 in 36-item Short Form General Health Survey (SF-36) Scores
Mental Component Summary
|
2.858 score on a scale
Standard Deviation 12.5103
|
|
Change From Baseline to Week 53 in 36-item Short Form General Health Survey (SF-36) Scores
Physical Component Summary
|
7.015 score on a scale
Standard Deviation 5.8464
|
SECONDARY outcome
Timeframe: Baseline, Week 53Population: 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.
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.
Outcome measures
| Measure |
VPRIV
n=15 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Change From Baseline to Week 53 in Childhood Health Questionnaire-Parent Form 50 (CHQ-PF50) Scores
Psychosocial Summary Score
|
0.38 score on a scale
Standard Deviation 11.462
|
|
Change From Baseline to Week 53 in Childhood Health Questionnaire-Parent Form 50 (CHQ-PF50) Scores
Physical Summary Score
|
-0.02 score on a scale
Standard Deviation 12.745
|
SECONDARY outcome
Timeframe: Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1Population: 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.
Outcome measures
| Measure |
VPRIV
n=16 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Cmax: Maximum Observed Serum Concentration for VPRIV at Week 1
|
5220 nanograms per milliliter (ng/mL)
Standard Deviation 1610
|
SECONDARY outcome
Timeframe: Within 3 minutes prior to the end of the 60-minute infusion at Week 37Population: 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.
Outcome measures
| Measure |
VPRIV
n=15 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Serum Concentration for VPRIV at Week 37
|
3450 ng/mL
Standard Deviation 1390
|
SECONDARY outcome
Timeframe: Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1Population: 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.
Outcome measures
| Measure |
VPRIV
n=16 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Tmax: Time to Reach the Maximum Serum Concentration (Cmax) for VPRIV
|
49.00 minutes (min)
Interval 19.0 to 60.0
|
SECONDARY outcome
Timeframe: Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1Population: 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.
ng\*min/mL denotes nanograms\*minutes per milliliter.
Outcome measures
| Measure |
VPRIV
n=16 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
AUCinf: Area Under the Plasma Concentration-time Curve From Time 0 to Infinity for VPRIV
|
235000 ng*min/mL
Standard Deviation 85000
|
SECONDARY outcome
Timeframe: Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1Population: 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.
Outcome measures
| Measure |
VPRIV
n=16 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Terminal Phase Elimination Half-life (T1/2) for VPRIV
|
10.12 min
Standard Deviation 2.81
|
SECONDARY outcome
Timeframe: Pre-dose and at multiple timepoints up to 120 minutes post-dose on Day 1 of Week 1Population: 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.
mL/min/kg denotes milliliters per minutes per kilogram.
Outcome measures
| Measure |
VPRIV
n=16 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Oral Clearance (CL) for VPRIV
|
7.27 mL/min/kg
Standard Deviation 2.87
|
SECONDARY outcome
Timeframe: Pre-dose and at multiple timepoints(up to 120 minutes post-dose on Day 1 of Week 1Population: 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.
Outcome measures
| Measure |
VPRIV
n=16 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Apparent Steady-state Volume of Distribution (Vss) for VPRIV
|
96.3 milliliters per kilogram (mL/kg)
Standard Deviation 52.0
|
SECONDARY outcome
Timeframe: Baseline, Week 53Population: 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.
Outcome measures
| Measure |
VPRIV
n=19 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Percent Change From Baseline to Week 53 in Biomarker: Plasma Chemokine [C-C Motif] Ligand 18
|
-58.94 percent change
Standard Deviation 14.339
|
SECONDARY outcome
Timeframe: Baseline, Week 53Population: 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.
Outcome measures
| Measure |
VPRIV
n=19 Participants
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Percent Change From Baseline to Week 53 in Biomarker: Glucopsychosine
|
-63.88 percent change
Standard Deviation 14.359
|
Adverse Events
Velaglucerase Alfa
Serious adverse events
| Measure |
Velaglucerase Alfa
n=20 participants at risk
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Metabolism and nutrition disorders
Diabetes mellitus inadequate control
|
5.0%
1/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
|
Infections and infestations
Pneumonia
|
5.0%
1/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
|
Infections and infestations
Pneumonia mycoplasmal
|
5.0%
1/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
|
Injury, poisoning and procedural complications
Splenic injury
|
5.0%
1/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
Other adverse events
| Measure |
Velaglucerase Alfa
n=20 participants at risk
Participants received VPRIV IV infusion at 60 U/kg body weight once EOW for 60 (+10) minutes up to Week 51.
|
|---|---|
|
Infections and infestations
Conjunctivitis
|
10.0%
2/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
25.0%
5/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
|
Injury, poisoning and procedural complications
Fall
|
10.0%
2/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
|
Metabolism and nutrition disorders
Hypertriglyceridaemia
|
10.0%
2/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
|
Infections and infestations
Nasopharyngitis
|
10.0%
2/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
|
Musculoskeletal and connective tissue disorders
Osteoporosis
|
10.0%
2/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
|
Infections and infestations
Pneumonia
|
10.0%
2/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
|
General disorders
Pyrexia
|
15.0%
3/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
|
Cardiac disorders
Supraventricular extrasystoles
|
10.0%
2/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
|
Infections and infestations
Upper respiratory tract infection
|
70.0%
14/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
|
Investigations
Urinary occult blood positive
|
10.0%
2/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
|
Infections and infestations
Urinary tract infection
|
10.0%
2/20 • Up to 56.2 weeks
The SAF included all participants in the ITT Set who received at least 1 dose of VPRIV.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place