Survey Study for Velaglucerase Alfa (VPRIV) in Japan

VPRIV Drug Use-Result Survey (Japan)

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.

Study Overview

• Status: Completed
• Conditions: Gaucher Disease

Detailed Description

Survey Study for Velaglucerase Alfa (VPRIV) in Japan

• Study Type: Observational
• Enrollment (Actual): 77

Contacts and Locations

Study Locations

• Japan
  • Chiba, Japan, 266-0007
    • Chiba
  • Gifu, Japan, 500-8212
    • Gifu
  • Hiroshima, Japan, 730-0046
    • Hiroshima
  • Hiroshima, Japan, 734-0037
    • Hiroshima
  • Kagoshima, Japan, 892-0853
    • Kagoshima
  • Kumamoto, Japan, 860-8556
    • Kumamoto
  • Kyoto, Japan, 606-8507
    • Kyoto
  • Okayama, Japan, 701-1192
    • Okayama
  • Osaka, Japan, 534-0021
    • Osaka
  • Osaka, Japan, 545-8586
    • Osaka
  • Osaka, Japan, 553-0003
    • Osaka
  • Saitama, Japan, 339-8551
    • Saitama
  • Shizuoka, Japan, 420-8688
    • Shizuoka
  • Aichi
    • Konan, Aichi, Japan, 483-8704
      • Konan
    • Nagoya, Aichi, Japan, 453-0801
      • Nagoya
  • Fukuoka
    • Kitakyushu, Fukuoka, Japan, 802-0803
      • Kitakyushu
    • Kurume, Fukuoka, Japan, 830-0011
      • Kurume
  • Hiroshima
    • Fukuyama, Hiroshima, Japan, 721-0927
      • Fukuyama
    • Higashihiroshima, Hiroshima, Japan, 739-0041
      • Higashihiroshima
  • Hokkaido
    • Obihiro, Hokkaido, Japan, 080-0016
      • Obihiro
  • Kanagawa
    • Sagamihara, Kanagawa, Japan, 252-0375
      • Sagamihara
  • Miyagi
    • Sendai, Miyagi, Japan, 980-8574
      • Sendai
  • Osaka
    • Suita, Osaka, Japan, 565-0871
      • Suita
    • Tondabayashi, Osaka, Japan, 584-0000
      • Tondabayashi
  • Saitama
    • Kawagoe, Saitama, Japan, 350-8550
      • Kawagoe
    • Tokorozawa, Saitama, Japan, 359-8513
      • Tokorozawa
  • Shiga
    • Moriyama, Shiga, Japan, 524-0022
      • Moriyama
    • Otsu, Shiga, Japan, 520-2192
      • Otsu
  • Shimane
    • Matsue, Shimane, Japan, 690-0864
      • Matsue
  • Shizuoka
    • Hamamatsu, Shizuoka, Japan, 431-3125
      • Hamamatsu
    • Hamamatsu, Shizuoka, Japan, 434-8511
      • Hamamatsu
    • Iwata, Shizuoka, Japan, 438-0002
      • Iwata
  • Tokyo
    • Minato, Tokyo, Japan, 105-8471
      • Minato
    • Sumida-ku, Tokyo, Japan, 130-0005
      • Sumida-ku
  • Tottori
    • Yonago, Tottori, Japan, 683-8504
      • Yonago

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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.

Description

Inclusion Criteria:

• 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

Exclusion Criteria:

-

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa (VPRIV)	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)
Number of Participants With Absence/presence of Anti-velaglucerase Alfa Antibodies	Effect of anti-velaglucerase alfa antibodies (including IgGs) will be performed at the physician's discretion per standard clinical practice. Immunoglobulin E (IgE) isotype-specific antibodies will also be measured when clinically indicated (for example, adverse event, serious adverse event or possible of lack of efficacy).	Baseline up to end of the study (8 years)
Number of Participants With Clinically Significant Change in Laboratory Assessment	Clinically significant changes in laboratory assessments will be reported.	Baseline up to end of the study (8 years)
Number of Participants With Hypersensitivity Reactions	Hypersensitivity reactions are defined as events of drug allergy, angioedema, anaphylactic reaction, anaphylactic shock, anaphylactoid reaction, anaphylaxis prophylaxis, anaphylaxis treatment, drug reaction with eosinophilia and systemic symptoms.	Baseline up to end of the study (8 years)
Occurrences of Pregnancies/breastfeeding for Women of Child-bearing Potential	Pregnancy testing for women of child-bearing potential will be collected throughout the survey.	Baseline up to end of the study (8 years)
Change From Baseline in Hemoglobin Concentration	Hemoglobin concentration will be assessed.	Baseline, Every 12 weeks up to 8 years
Change From Baseline in Platelet Count	Platelet count will be assessed.	Baseline, Every 12 weeks up to 8 years
Change From Baseline in Liver Volume	Liver volume will be measured at the physician's discretion per standard clinical practice.	Baseline, Every 24 weeks up to 8 years
Change From Baseline in Spleen Volume	Spleen volume will be measured at the physician's discretion per standard clinical practice.	Baseline, Every 24 weeks up to 8 years
Change From Baseline in Bone Density	Bone density will be assessed at the physician's discretion per standard clinical practice.	Baseline, Every 52 weeks up to 8 years
Number of Participants With Infusion-related Reactions (IRRs)	Infusion-related reactions are defined as reactions occurring up to 24 hours after the start of the infusion.	Baseline up to end of the study (8 years)
Number of Participants with anti-velaglucerase alfa antibodies	Anti-velaglucerase alfa antibodies will be assessed.	Baseline up to end of study (8 years)

Collaborators and Investigators

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

Publications and helpful links

General Publications

• 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.

Helpful Links

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

Study record dates

Study Major Dates

• Study Start (Actual): September 12, 2014
• Primary Completion (Actual): January 17, 2024
• Study Completion (Actual): January 17, 2024

Study Registration Dates

• First Submitted: August 8, 2018
• First Submitted That Met QC Criteria: August 8, 2018
• First Posted (Actual): August 10, 2018

Study Record Updates

• Last Update Posted (Actual): March 28, 2024
• Last Update Submitted That Met QC Criteria: March 27, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Lipidoses; Lipid Metabolism, Inborn Errors; Gaucher Disease
• Other Study ID Numbers: SHP-GCB-401

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