Survey Study for Velaglucerase Alfa (VPRIV) in Japan

Gaucher Disease Clinical Trial

Official title:

VPRIV Drug Use-Result Survey (Japan)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03625882
• Other study ID #: SHP-GCB-401
• Status: Completed
• Start date: September 12, 2014
• Est. completion date: January 17, 2024

Study information

• Verified date: March 2024
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 77
• Est. completion date: January 17, 2024
• Est. primary completion date: January 17, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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: -

Related Conditions & MeSH terms

• Gaucher Disease

Locations

Country	Name	City	State
Japan	Chiba	Chiba
Japan	Fukuyama	Fukuyama	Hiroshima
Japan	Gifu	Gifu
Japan	Hamamatsu	Hamamatsu	Shizuoka
Japan	Hamamatsu	Hamamatsu	Shizuoka
Japan	Higashihiroshima	Higashihiroshima	Hiroshima
Japan	Hiroshima	Hiroshima
Japan	Hiroshima	Hiroshima
Japan	Iwata	Iwata	Shizuoka
Japan	Kagoshima	Kagoshima
Japan	Kawagoe	Kawagoe	Saitama
Japan	Kitakyushu	Kitakyushu	Fukuoka
Japan	Konan	Konan	Aichi
Japan	Kumamoto	Kumamoto
Japan	Kurume	Kurume	Fukuoka
Japan	Kyoto	Kyoto
Japan	Matsue	Matsue	Shimane
Japan	Minato	Minato	Tokyo
Japan	Moriyama	Moriyama	Shiga
Japan	Nagoya	Nagoya	Aichi
Japan	Obihiro	Obihiro	Hokkaido
Japan	Okayama	Okayama
Japan	Osaka	Osaka
Japan	Osaka	Osaka
Japan	Osaka	Osaka
Japan	Otsu	Otsu	Shiga
Japan	Sagamihara	Sagamihara	Kanagawa
Japan	Saitama	Saitama
Japan	Sendai	Sendai	Miyagi
Japan	Shizuoka	Shizuoka
Japan	Suita	Suita	Osaka
Japan	Sumida-ku	Sumida-ku	Tokyo
Japan	Tokorozawa	Tokorozawa	Saitama
Japan	Tondabayashi	Tondabayashi	Osaka
Japan	Yonago	Yonago	Tottori

Sponsors (1)

Lead Sponsor	Collaborator
Takeda

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	Change From Baseline in Hemoglobin Concentration	Hemoglobin concentration will be assessed.	Baseline, Every 12 weeks up to 8 years
Primary	Change From Baseline in Platelet Count	Platelet count will be assessed.	Baseline, Every 12 weeks up to 8 years
Primary	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
Primary	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
Primary	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
Primary	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)
Primary	Number of Participants with anti-velaglucerase alfa antibodies	Anti-velaglucerase alfa antibodies will be assessed.	Baseline up to end of study (8 years)

External Links

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