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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03625882
Other study ID # SHP-GCB-401
Secondary ID
Status Completed
Phase
First received
Last updated
Start date September 12, 2014
Est. completion date May 14, 2024

Study information

Verified date May 2024
Source Takeda
Contact n/a
Is FDA regulated No
Health authority
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 May 14, 2024
Est. primary completion date May 14, 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: -

Study Design


Related Conditions & MeSH terms


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)
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