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Gaucher DiseaseNCT03625882

Survey Study for Velaglucerase Alfa (VPRIV) in Japan

An observational study of Gaucher Disease, run by Takeda at 35 sites in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Any age
Conditions the study is for
Gaucher Disease
How many people
63 participants
Where
35 sitesJapan
Study dates
2 Sep 2014to 14 May 2024
Phase
Observational
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

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.

Who may be able to join

  • 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

Who cannot join

  • -

Trial locations and contacts

35 sites in 1 country.

35 sites in 1 country

Japan35 sites

  • Chiba

    Chiba, 266-0007

  • Fukuyama

    Fukuyama, Hiroshima, 721-0927

  • Gifu

    Gifu, 500-8212

  • Hamamatsu

    Hamamatsu, Shizuoka, 431-3125

  • Hamamatsu

    Hamamatsu, Shizuoka, 434-8511

  • Higashihiroshima

    Higashihiroshima, Hiroshima, 739-0041

  • Hiroshima

    Hiroshima, 730-0046

  • Hiroshima

    Hiroshima, 734-0037

  • Iwata

    Iwata, Shizuoka, 438-0002

  • Kagoshima

    Kagoshima, 892-0853

  • Kawagoe

    Kawagoe, Saitama, 350-8550

  • Kitakyushu

    Kitakyushu, Fukuoka, 802-0803

  • Konan

    Kōnan, Aichi-ken, 483-8704

  • Kumamoto

    Kumamoto, 860-8556

  • Kurume

    Kurume, Fukuoka, 830-0011

  • Kyoto

    Kyoto, 606-8507

  • Matsue

    Matsue, Shimane, 690-0864

  • Minato

    Minato, Tokyo, 105-8471

  • Moriyama

    Moriyama, Shiga, 524-0022

  • Nagoya

    Nagoya, Aichi-ken, 453-0801

  • Obihiro

    Obihiro, Hokkaido, 080-0016

  • Okayama

    Okayama, 701-1192

  • Osaka

    Osaka, 534-0021

  • Osaka

    Osaka, 545-8586

  • Osaka

    Osaka, 553-0003

  • Otsu

    Ōtsu, Shiga, 520-2192

  • Sagamihara

    Sagamihara, Kanagawa, 252-0375

  • Saitama

    Saitama, 339-8551

  • Sendai

    Sendai, Miyagi, 980-8574

  • Shizuoka

    Shizuoka, 420-8688

  • Suita

    Suita, Osaka, 565-0871

  • Sumida-ku

    Sumida-ku, Tokyo, 130-0005

  • Tokorozawa

    Tokorozawa, Saitama, 359-8513

  • Tondabayashi

    Tondabayashi, Osaka, 584-0000

  • Yonago

    Yonago, Tottori, 683-8504

Other trials in Japan

About this study

Official title: 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.

Conditions
Gaucher Disease
Medical subject headings
Gaucher Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT03625882
Study groupRoleWhat they receive
Gaucher Disease Participants Treated With VPRIVParticipants 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 being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Baseline up to end of the study (8 years)

Secondary outcomes (10)

  1. Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration

    Number of participants of efficacy categories in assessment of change from baseline in hemoglobin concentration during the study was reported. The efficacy categories were classified as Good: increased by at least 1.5g/dL, Moderate response: increase of at least 0.5 g/dL and less than 1.5 g/dL, and Ineffective: increase less than 0.5 g/dL.

    Measured: Baseline, Every 12 weeks up to 8 years

  2. Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts

    Number of participants of efficacy categories in assessment of change from baseline in platelet counts during the study was reported. The efficacy categories were classified as Good: increase of at least 30 × 10^9/L, Moderate response: increase of at least 15 × 10^9/L and less than 30 × 10^9/L, and Ineffective: less than 15 × 10^9/L.

    Measured: Baseline, Every 12 weeks up to 8 years

  3. Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes

    Number of participants of efficacy categories in assessment of change from baseline in liver volumes during the study was reported. The efficacy categories were classified as Good: reduction of at least 30%, Moderate response: reduction of at least 10% and less than 30%, and Ineffective: less than 10% reduction.

    Measured: Baseline, Every 24 weeks up to 8 years

  4. Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes

    Number of participants of efficacy categories in assessment of change from baseline in spleen volumes during the study was reported. The efficacy categories were classified as Good: reduction of at least 30%, Moderate response: reduction of at least 10% and less than 30%, and Ineffective: less than 10% reduction.

    Measured: Baseline, Every 24 weeks up to 8 years

  5. Lumbar Spine Bone Mineral Density (BMD) T-scores and Femur Neck BMD T-scores at Baseline and the Last Data Collection

    T-scores were the number of standard deviations (SDs) above or below the average for a young adult at peak BMD. A T-score of 0 was equal to the mean. Negative numbers indicated values lower than the mean and positive numbers indicated values higher than the mean. The reported data in this outcome measure was lumbar spine BMD T-scores and femur neck BMD T-scores related to bone density at baseline and the last data collection.

    Measured: Baseline, at the time of last data collection (up to 8 years)

  6. Lumbar Spine BMD Z-scores and Femur Neck BMD Z-scores at Baseline and the Last Data Collection

    Z-scores express the BMD as the number of SDs above or below the average BMD of a healthy participant of the same age and gender. A Z-score of 0 was equal to the mean. Negative numbers indicated values lower than the mean and positive numbers indicated values higher than the mean. The reported data in this outcome measure was lumbar spine BMD Z-scores and femur neck BMD Z-scores related to bone density at baseline and the last data collection.

    Measured: Baseline, at the time of last data collection (up to 8 years)

  7. Lumbar Spine BMD and Femur Neck BMD at Baseline and the Last Data Collection

    The reported data in this outcome measure was lumbar spine BMD and femur neck BMD related to bone density at baseline and the last data collection.

    Measured: Baseline, at the time of last data collection (up to 8 years)

  8. Number of Participants With Positive Anti-velaglucerase Alfa Antibody Test Results

    Number of participants with positive anti-velaglucerase alfa antibody test results was reported.

    Measured: Baseline up to end of the study (8 years)

  9. Number of Participants With Adverse Drug Reaction and Serious Adverse Drug Reaction 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. Adverse drug reaction refers to AE related to administered drug.

    Measured: Baseline up to end of the study (8 years)

  10. Number of Participants With Adverse Reactions Categorized as Infusion Reactions 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. Adverse drug reaction refers to AE related to administered drug. Infusion reactions were defined as reactions occurring up to 24 hours after the start of the infusion.

    Measured: Baseline up to end of the study (8 years)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Aug 2018
  2. Study start2 Sep 2014
  3. Primary completion14 May 2024
  4. Study completion14 May 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
63 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03625882
Sponsor's protocol ID
SHP-GCB-401
Status last verified
Feb 2026
First submitted
8 Aug 2018
Last updated
4 Mar 2026
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: 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.

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.

    PubMed 34863216

Questions about this trial

What is this clinical trial studying?

An observational study of Gaucher Disease, run by Takeda at 35 sites in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 4 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 35 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 10 Jan 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa (time frame: Baseline up to end of the study (8 years)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03625882, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Mar 2026.

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