Survey Study for Velaglucerase Alfa (VPRIV) in Japan

NCT ID: NCT03625882

Last Updated: 2025-01-10

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

63 participants

Study Classification

OBSERVATIONAL

Study Start Date

2014-09-02

Study Completion Date

2024-05-14

Brief Summary

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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.

Detailed Description

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Conditions

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Gaucher Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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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.

No interventions assigned to this group

Eligibility Criteria

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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

\-
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Takeda

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

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Konan

Kōnan, Aichi-ken, Japan

Site Status

Nagoya

Nagoya, Aichi-ken, Japan

Site Status

Kitakyushu

Kitakyushu, Fukuoka, Japan

Site Status

Kurume

Kurume, Fukuoka, Japan

Site Status

Fukuyama

Fukuyama, Hiroshima, Japan

Site Status

Higashihiroshima

Higashihiroshima, Hiroshima, Japan

Site Status

Obihiro

Obihiro, Hokkaido, Japan

Site Status

Sagamihara

Sagamihara, Kanagawa, Japan

Site Status

Sendai

Sendai, Miyagi, Japan

Site Status

Suita

Suita, Osaka, Japan

Site Status

Tondabayashi

Tondabayashi, Osaka, Japan

Site Status

Kawagoe

Kawagoe, Saitama, Japan

Site Status

Tokorozawa

Tokorozawa, Saitama, Japan

Site Status

Moriyama

Moriyama, Shiga, Japan

Site Status

Otsu

Ōtsu, Shiga, Japan

Site Status

Matsue

Matsue, Shimane, Japan

Site Status

Hamamatsu

Hamamatsu, Shizuoka, Japan

Site Status

Hamamatsu

Hamamatsu, Shizuoka, Japan

Site Status

Iwata

Iwata, Shizuoka, Japan

Site Status

Minato

Minato, Tokyo, Japan

Site Status

Sumida-ku

Sumida-ku, Tokyo, Japan

Site Status

Yonago

Yonago, Tottori, Japan

Site Status

Chiba

Chiba, , Japan

Site Status

Gifu

Gifu, , Japan

Site Status

Hiroshima

Hiroshima, , Japan

Site Status

Hiroshima

Hiroshima, , Japan

Site Status

Kagoshima

Kagoshima, , Japan

Site Status

Kumamoto

Kumamoto, , Japan

Site Status

Kyoto

Kyoto, , Japan

Site Status

Okayama

Okayama, , Japan

Site Status

Osaka

Osaka, , Japan

Site Status

Osaka

Osaka, , Japan

Site Status

Osaka

Osaka, , Japan

Site Status

Saitama

Saitama, , Japan

Site Status

Shizuoka

Shizuoka, , Japan

Site Status

Countries

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Japan

References

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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.

Reference Type DERIVED
PMID: 34863216 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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https://clinicaltrials.takeda.com/study-detail/5f6b5fe94db2bf003ab47b4e

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

Other Identifiers

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SHP-GCB-401

Identifier Type: -

Identifier Source: org_study_id

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