Clinical trial NCT03625882: Survey Study for Velaglucerase Alfa (VPRIV) in Japan
Official title: VPRIV Drug Use-Result Survey (Japan)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Gaucher Disease
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2018-08-10
Enrollment: 63
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.


Eligibility criteria: 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:

-

Primary outcomes:
measure: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa
timeFrame: Baseline up to end of the study (8 years)
Secondary outcomes:
measure: Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration
timeFrame: Baseline, Every 12 weeks up to 8 years
measure: Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts
timeFrame: Baseline, Every 12 weeks up to 8 years
measure: Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes
timeFrame: Baseline, Every 24 weeks up to 8 years
measure: Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes
timeFrame: Baseline, Every 24 weeks up to 8 years
measure: Lumbar Spine Bone Mineral Density (BMD) T-scores and Femur Neck BMD T-scores at Baseline and the Last Data Collection
timeFrame: Baseline, at the time of last data collection (up to 8 years)
measure: Lumbar Spine BMD Z-scores and Femur Neck BMD Z-scores at Baseline and the Last Data Collection
timeFrame: Baseline, at the time of last data collection (up to 8 years)
measure: Lumbar Spine BMD and Femur Neck BMD at Baseline and the Last Data Collection
timeFrame: Baseline, at the time of last data collection (up to 8 years)
measure: Number of Participants With Positive Anti-velaglucerase Alfa Antibody Test Results
timeFrame: Baseline up to end of the study (8 years)
measure: Number of Participants With Adverse Drug Reaction and Serious Adverse Drug Reaction Following Initiation of Treatment With Velaglucerase Alfa
timeFrame: Baseline up to end of the study (8 years)
measure: Number of Participants With Adverse Reactions Categorized as Infusion Reactions Following Initiation of Treatment With Velaglucerase Alfa
timeFrame: Baseline up to end of the study (8 years)
Other outcomes:
Locations:
city: Konan
country: Japan
facility: Konan
state: Aichi
city: Nagoya
country: Japan
facility: Nagoya
state: Aichi
city: Kitakyushu
country: Japan
facility: Kitakyushu
state: Fukuoka
city: Kurume
country: Japan
facility: Kurume
state: Fukuoka
city: Fukuyama
country: Japan
facility: Fukuyama
state: Hiroshima
city: Higashihiroshima
country: Japan
facility: Higashihiroshima
state: Hiroshima
city: Obihiro
country: Japan
facility: Obihiro
state: Hokkaido
city: Sagamihara
country: Japan
facility: Sagamihara
state: Kanagawa
city: Sendai
country: Japan
facility: Sendai
state: Miyagi
city: Suita
country: Japan
facility: Suita
state: Osaka
city: Tondabayashi
country: Japan
facility: Tondabayashi
state: Osaka
city: Kawagoe
country: Japan
facility: Kawagoe
state: Saitama
city: Tokorozawa
country: Japan
facility: Tokorozawa
state: Saitama
city: Moriyama
country: Japan
facility: Moriyama
state: Shiga
city: Otsu
country: Japan
facility: Otsu
state: Shiga
city: Matsue
country: Japan
facility: Matsue
state: Shimane
city: Hamamatsu
country: Japan
facility: Hamamatsu
state: Shizuoka
city: Hamamatsu
country: Japan
facility: Hamamatsu
state: Shizuoka
city: Iwata
country: Japan
facility: Iwata
state: Shizuoka
city: Minato
country: Japan
facility: Minato
state: Tokyo
city: Sumida-ku
country: Japan
facility: Sumida-ku
state: Tokyo
city: Yonago
country: Japan
facility: Yonago
state: Tottori
city: Chiba
country: Japan
facility: Chiba
city: Gifu
country: Japan
facility: Gifu
city: Hiroshima
country: Japan
facility: Hiroshima
city: Hiroshima
country: Japan
facility: Hiroshima
city: Kagoshima
country: Japan
facility: Kagoshima
city: Kumamoto
country: Japan
facility: Kumamoto
city: Kyoto
country: Japan
facility: Kyoto
city: Okayama
country: Japan
facility: Okayama
city: Osaka
country: Japan
facility: Osaka
city: Osaka
country: Japan
facility: Osaka
city: Osaka
country: Japan
facility: Osaka
city: Saitama
country: Japan
facility: Saitama
city: Shizuoka
country: Japan
facility: Shizuoka
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References:
citation: 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.