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

NCT05529992 | Completed Phase 3 Results

• 2 other identifiers: TAK-669-30012022-002323-35
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 10

Brief Summary

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.

Conditions

Gaucher Disease

Keywords

VPRIV; Gaucher Disease

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (22)

• Percentage of Participants With TEAEs

  Up to 56.2 weeks

• Percentage of Participants With Infusion-related Reactions Reported as an Adverse Event

  Up to 56.2 weeks

• Percentage of Participants With Development of Anti-VPRIV Antibodies and Neutralizing Antibodies at Week 53

  Week 53

• Number of Participants With Clinically Significant Changes in Laboratory Assessments at Week 53

  Week 53

• Number of Participants With Abnormal Changes in Laboratory Assessments at Week 53: Urinalysis

  Week 53

Study Arms (1)

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.

Drug: Velaglucerase Alfa

Interventions

Velaglucerase Alfa DRUG

VPRIV intravenous infusion every other week for 60 minutes.

Also known as: VPRIV

Velaglucerase Alfa (VPRIV)

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Has a documented, confirmed diagnosis of type 1 Gaucher disease based on the following, as determined by the investigator:
• Decreased glucocerebrosidase (GCB) activity level that is ≤30% of normal or
• 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
• Hemoglobin levels of ≥1 g/dL below the lower limit of normal for their age and gender AND/OR
• 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.

You may not qualify if:

• 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)

Sponsors & Collaborators

• Takeda: lead

Study Sites (10)

Chinese PLA General Hospital

Beijing, Beijing Municipality, 100039, China

Location

Beijing Children's Hospital, Capital Medical University

Beijing, Beijing Municipality, 100045, China

Location

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences

Beijing, Beijing Municipality, 100730, China

Location

Lanzhou University Second Hospital

Lanzhou, Gansu, 730030, China

Location

The First Affiliated Hospital, Sun Yat-sen University

Guangzhou, Guangdong, 510080, China

Location

Guangzhou Women and Children's Medical Center

Guangzhou, Guangdong, 510623, China

Location

The Second Hospital of Hebei Medical University

Shijiazhuang, Hebei, 050000, China

Location

Tongji Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology

Wuhan, Hubei, 430030, China

Location

Nanjing Children's Hospital

Nanjing, Jiangsu, 210008, China

Location

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 300020, China

Location

Related Links

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

MeSH Terms

Conditions

Gaucher Disease

Interventions

Glucosylceramidase

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Glucosidases; Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes

Results Point of Contact

• Title: Study Director
• Organization: Takeda

TrialDisclosures@takeda.com

+1-877-825-3327

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 2, 2022
• First Posted: September 7, 2022
• Study Start: January 3, 2023
• Primary Completion: August 5, 2024
• Study Completion: August 5, 2024
• Last Updated: June 26, 2025
• Results First Posted: February 26, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• 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.

Locations

CN (10)