A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease
NCT ID: NCT05529992
Last Updated: 2025-06-26
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
20 participants
INTERVENTIONAL
2023-01-03
2024-08-05
Brief Summary
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Participants will receive VPRIV intravenously during the treatment period (up to 51 weeks), followed by the end-of-treatment (EOT) visit after 2 weeks.
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Detailed Description
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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.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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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.
Velaglucerase Alfa
VPRIV intravenous infusion every other week for 60 minutes.
Interventions
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Velaglucerase Alfa
VPRIV intravenous infusion every other week for 60 minutes.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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)
2 Years
ALL
No
Sponsors
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Takeda
INDUSTRY
Responsible Party
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Principal Investigators
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Study Director
Role: STUDY_DIRECTOR
Takeda
Locations
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Chinese PLA General Hospital
Beijing, Beijing Municipality, China
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, China
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Beijing, Beijing Municipality, China
Lanzhou University Second Hospital
Lanzhou, Gansu, China
The First Affiliated Hospital, Sun Yat-sen University
Guangzhou, Guangdong, China
Guangzhou Women and Children's Medical Center
Guangzhou, Guangdong, China
The Second Hospital of Hebei Medical University
Shijiazhuang, Hebei, China
Tongji Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology
Wuhan, Hubei, China
Nanjing Children's Hospital
Nanjing, Jiangsu, China
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, China
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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To obtain more information on the study, click here/on this link
Other Identifiers
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2022-002323-35
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
TAK-669-3001
Identifier Type: -
Identifier Source: org_study_id
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