Survey Study for Velaglucerase Alfa (VPRIV) in Japan
VPRIV Drug Use-Result Survey (Japan)
1 other identifier
observational
63
1 country
35
Brief Summary
The objective of this post-marketing survey study is to collect data to determine the safety and efficacy of velaglucerase alfa (VPRIV) in participants with Gaucher disease who are new to therapy or have been switched from another therapeutic agent for Gaucher disease.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started Sep 2014
Longer than P75 for all trials
35 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
September 2, 2014
CompletedFirst Submitted
Initial submission to the registry
August 8, 2018
CompletedFirst Posted
Study publicly available on registry
August 10, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 14, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
May 14, 2024
CompletedResults Posted
Study results publicly available
January 10, 2025
CompletedMarch 4, 2026
February 1, 2026
9.7 years
August 8, 2018
November 11, 2024
February 11, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa
An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalisation or results in prolongation of existing hospitalisation; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event.
Baseline up to end of the study (8 years)
Secondary Outcomes (10)
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration
Baseline, Every 12 weeks up to 8 years
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts
Baseline, Every 12 weeks up to 8 years
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes
Baseline, Every 24 weeks up to 8 years
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes
Baseline, Every 24 weeks up to 8 years
Lumbar Spine Bone Mineral Density (BMD) T-scores and Femur Neck BMD T-scores at Baseline and the Last Data Collection
Baseline, at the time of last data collection (up to 8 years)
- +5 more secondary outcomes
Study Arms (1)
Gaucher Disease Participants Treated With VPRIV
Participants with Gaucher disease will be enrolled in this survey, who are in VPRIV treatment-naïve therapy or have been switched from another therapeutic agent for Gaucher disease.
Eligibility Criteria
Participants with confirmed Gaucher disease (types 1, 2, or 3) irrespective of any age or gender who are either naïve to treatment or participants that have been treated with another therapeutic agent for Gaucher disease.
You may qualify if:
- Male or female participants with a confirmed diagnosis of Gaucher disease
- Participants who are either naïve to treatment or participants that have been treated with another therapeutic agent for Gaucher disease
- Participants who start VPRIV treatment or transition from VPRIV clinical studies during the enrollment period
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Takedalead
Study Sites (35)
Konan
Kōnan, Aichi-ken, 483-8704, Japan
Nagoya
Nagoya, Aichi-ken, 453-0801, Japan
Kitakyushu
Kitakyushu, Fukuoka, 802-0803, Japan
Kurume
Kurume, Fukuoka, 830-0011, Japan
Fukuyama
Fukuyama, Hiroshima, 721-0927, Japan
Higashihiroshima
Higashihiroshima, Hiroshima, 739-0041, Japan
Obihiro
Obihiro, Hokkaido, 080-0016, Japan
Sagamihara
Sagamihara, Kanagawa, 252-0375, Japan
Sendai
Sendai, Miyagi, 980-8574, Japan
Suita
Suita, Osaka, 565-0871, Japan
Tondabayashi
Tondabayashi, Osaka, 584-0000, Japan
Kawagoe
Kawagoe, Saitama, 350-8550, Japan
Tokorozawa
Tokorozawa, Saitama, 359-8513, Japan
Moriyama
Moriyama, Shiga, 524-0022, Japan
Otsu
Ōtsu, Shiga, 520-2192, Japan
Matsue
Matsue, Shimane, 690-0864, Japan
Hamamatsu
Hamamatsu, Shizuoka, 431-3125, Japan
Hamamatsu
Hamamatsu, Shizuoka, 434-8511, Japan
Iwata
Iwata, Shizuoka, 438-0002, Japan
Minato
Minato, Tokyo, 105-8471, Japan
Sumida-ku
Sumida-ku, Tokyo, 130-0005, Japan
Yonago
Yonago, Tottori, 683-8504, Japan
Chiba
Chiba, 266-0007, Japan
Gifu
Gifu, 500-8212, Japan
Hiroshima
Hiroshima, 730-0046, Japan
Hiroshima
Hiroshima, 734-0037, Japan
Kagoshima
Kagoshima, 892-0853, Japan
Kumamoto
Kumamoto, 860-8556, Japan
Kyoto
Kyoto, 606-8507, Japan
Okayama
Okayama, 701-1192, Japan
Osaka
Osaka, 534-0021, Japan
Osaka
Osaka, 545-8586, Japan
Osaka
Osaka, 553-0003, Japan
Saitama
Saitama, 339-8551, Japan
Shizuoka
Shizuoka, 420-8688, Japan
Related Publications (1)
Sagara R, Ishigaki M, Otsuka M, Murayama K, Ida H, Fernandez J. Long-term safety and effectiveness of velaglucerase alfa in Gaucher disease: 6-year interim analysis of a post-marketing surveillance in Japan. Orphanet J Rare Dis. 2021 Dec 4;16(1):502. doi: 10.1186/s13023-021-02119-2.
PMID: 34863216DERIVED
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Takeda
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 8, 2018
First Posted
August 10, 2018
Study Start
September 2, 2014
Primary Completion
May 14, 2024
Study Completion
May 14, 2024
Last Updated
March 4, 2026
Results First Posted
January 10, 2025
Record last verified: 2026-02
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.
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.