Survey Study for Velaglucerase Alfa (VPRIV) in Japan

Completed

• Conditions: Gaucher Disease

• Registration Number: NCT03625882
• Lead Sponsor: Takeda

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-09-02
• Study First Submitted: 2018-08-08
• Study First Submitted QC: 2018-08-08
• Study First Posted: 2018-08-10
• Primary Completion: 2024-05-14
• Study Completion: 2024-05-14
• Status Verified: 2024-11
• Results First Submitted: 2024-11-11
• Results First Submitted QC: 2024-11-11
• Last Update Submitted: 2024-11-11
• Results First Posted: 2025-01-10
• Last Update Posted: 2025-01-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 63

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

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Exclusion Criteria

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa	Baseline up to end of the study (8 years)	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.

Secondary Outcome Measures

Name	Time	Method
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 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.
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 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.
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 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.
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes	Baseline, Every 24 weeks up to 8 years	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.
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)	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.
Lumbar Spine BMD Z-scores and Femur Neck BMD Z-scores at Baseline and the Last Data Collection	Baseline, at the time of last data collection (up to 8 years)	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.
Lumbar Spine BMD and Femur Neck BMD at Baseline and the Last Data Collection	Baseline, at the time of last data collection (up to 8 years)	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.
Number of Participants With Positive Anti-velaglucerase Alfa Antibody Test Results	Baseline up to end of the study (8 years)	Number of participants with positive anti-velaglucerase alfa antibody test results was reported.
Number of Participants With Adverse Drug Reaction and Serious Adverse Drug Reaction Following Initiation of Treatment With Velaglucerase Alfa	Baseline up to end of the study (8 years)	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.
Number of Participants With Adverse Reactions Categorized as Infusion Reactions Following Initiation of Treatment With Velaglucerase Alfa	Baseline up to end of the study (8 years)	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.

Trial Locations

Locations (31)

Nagoya; 🇯🇵 Nagoya, Aichi, Japan

Higashihiroshima; 🇯🇵 Higashihiroshima, Hiroshima, Japan

Fukuyama; 🇯🇵 Fukuyama, Hiroshima, Japan

Minato; 🇯🇵 Minato, Tokyo, Japan

Saitama; 🇯🇵 Saitama, Japan

Konan; 🇯🇵 Konan, Aichi, Japan

Suita; 🇯🇵 Suita, Osaka, Japan

Tokorozawa; 🇯🇵 Tokorozawa, Saitama, Japan

Iwata; 🇯🇵 Iwata, Shizuoka, Japan

Kumamoto; 🇯🇵 Kumamoto, Japan

Kitakyushu; 🇯🇵 Kitakyushu, Fukuoka, Japan

Kurume; 🇯🇵 Kurume, Fukuoka, Japan

Sagamihara; 🇯🇵 Sagamihara, Kanagawa, Japan

Yonago; 🇯🇵 Yonago, Tottori, Japan

Chiba; 🇯🇵 Chiba, Japan

Osaka; 🇯🇵 Osaka, Japan

Sendai; 🇯🇵 Sendai, Miyagi, Japan

Tondabayashi; 🇯🇵 Tondabayashi, Osaka, Japan

Shizuoka; 🇯🇵 Shizuoka, Japan

Obihiro; 🇯🇵 Obihiro, Hokkaido, Japan

Kawagoe; 🇯🇵 Kawagoe, Saitama, Japan

Moriyama; 🇯🇵 Moriyama, Shiga, Japan

Hamamatsu; 🇯🇵 Hamamatsu, Shizuoka, Japan

Gifu; 🇯🇵 Gifu, Japan

Kagoshima; 🇯🇵 Kagoshima, Japan

Okayama; 🇯🇵 Okayama, Japan

Otsu; 🇯🇵 Otsu, Shiga, Japan

Matsue; 🇯🇵 Matsue, Shimane, Japan

Sumida-ku; 🇯🇵 Sumida-ku, Tokyo, Japan

Hiroshima; 🇯🇵 Hiroshima, Japan

Kyoto; 🇯🇵 Kyoto, Japan