A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

A Multicenter, Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Velaglucerase Alfa in Chinese Subjects With Type 1 Gaucher Disease

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.

Study Overview

• Status: Completed
• Conditions: Gaucher Disease
• Intervention / Treatment: Drug: Velaglucerase Alfa

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Phase 3

Contacts and Locations

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100045
      • Beijing Children's Hospital, Capital Medical University
    • Beijing, Beijing, China, 100039
      • Chinese PLA General Hospital
    • Beijing, Beijing, China, 100730
      • Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
  • Gansu
    • Lanzhou, Gansu, China, 730030
      • Lanzhou University Second Hospital
  • Guangdong
    • Guangzhou, Guangdong, China, 510080
      • The First Affiliated Hospital, Sun Yat-sen University
    • Guangzhou, Guangdong, China, 510623
      • Guangzhou Women and Children's Medical Center
  • Hebei
    • Shijiazhuang, Hebei, China, 050000
      • The Second Hospital of Hebei Medical University
  • Hubei
    • Wuhan, Hubei, China, 430030
      • Tongji Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology
  • Jiangsu
    • Nanjing, Jiangsu, China, 210008
      • Nanjing Children's Hospital
  • Tianjin
    • Tianjin, Tianjin, China, 300020
      • Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion:

• Has a documented, confirmed diagnosis of type 1 Gaucher disease based on the following, as determined by the investigator:

  1. Decreased glucocerebrosidase (GCB) activity level that is ≤30% of normal or
  2. Decreased GCB activity level that is >30% of normal, but with confirmation of genetic mutation test
• 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

  1. Hemoglobin levels of ≥1 g/dL below the lower limit of normal for their age and gender AND/OR
  2. A platelet count of <90 × 10^9/L below the lower limit of normal for their age and gender

• Has Gaucher disease-related viscera abnormalities, defined as the following:

  • Participant has at least moderate splenomegaly, assessed by palpation (2 to 3 cm below the left costal margin), or by abdominal radiology scan (magnetic resonance imaging [MRI] or computed tomography [CT] scan, with spleen volume >5 times normal) AND/OR
  • Participant has hepatomegaly, assessed by palpation or by abdominal radiology scan (MRI or CT scan); Participants who have undergone splenectomy must have satisfied these criteria for this study.

Exclusion:

• Has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease as assessed by the investigator
• Has had a splenectomy or an active, clinically significant spleen infarction within the 12 months prior to screening
• Has received treatment with any investigational drug or device within 30 days prior to screening, or within 5 half-lives of that investigational product, whichever is greater; such treatment during the study will not be permitted
• Is currently receiving red blood cell growth factor (eg, erythropoietin), chronic systemic corticosteroids, or has been on such treatment within the 6 months prior to screening
• Presents with non-Gaucher disease related exacerbated anemia at screening
• Has experienced a severe (grade 3 or higher) infusion-related hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any ERT (approved or investigational)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Velaglucerase Alfa (VPRIV); 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.	Drug: Velaglucerase Alfa; VPRIV intravenous infusion every other week for 60 minutes.; Other Names: VPRIV

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Secondary Outcome Measures

Outcome Measure	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.	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

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Study Director, Takeda

Publications and helpful links

Helpful Links

• To obtain more information on the study, click here/on this link

Study record dates

Study Major Dates

• Study Start (Actual): January 3, 2023
• Primary Completion (Actual): August 5, 2024
• Study Completion (Actual): August 5, 2024

Study Registration Dates

• First Submitted: September 2, 2022
• First Submitted That Met QC Criteria: September 2, 2022
• First Posted (Actual): September 7, 2022

Study Record Updates

• Last Update Posted (Actual): June 26, 2025
• Last Update Submitted That Met QC Criteria: June 18, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Keywords: Gaucher Disease; VPRIV
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Lipid Metabolism Disorders; Lysosomal Storage Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases, Nervous System; Sphingolipidoses; Lipidoses; Gaucher Disease
• Other Study ID Numbers: TAK-669-3001; 2022-002323-35 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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.
• IPD Sharing Access Criteria: 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.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No