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Survey Study for Velaglucerase Alfa (VPRIV) in Japan

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ClinicalTrials.gov Identifier: NCT03625882
Recruitment Status : Recruiting
First Posted : August 10, 2018
Last Update Posted : May 8, 2019
Sponsor:
Information provided by (Responsible Party):
Shire

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.

Condition or disease
Gaucher Disease

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Study Type : Observational
Estimated Enrollment : 30 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: VPRIV Drug Use-Result Survey (Japan)
Actual Study Start Date : September 12, 2014
Estimated Primary Completion Date : September 22, 2022
Estimated Study Completion Date : September 22, 2022


Group/Cohort
Gaucher Disease Participants Treated With VPRIV
Participants 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.



Primary Outcome Measures :
  1. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa (VPRIV) [ Time Frame: 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.

  2. Number of Participants With Absence/presence of Anti-velaglucerase Alfa Antibodies [ Time Frame: Baseline up to end of the study (8 years) ]
    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).

  3. Number of Participants With Clinically Significant Change in Laboratory Assessment [ Time Frame: Baseline up to end of the study (8 years) ]
    Clinically significant changes in laboratory assessments will be reported.

  4. Number of Participants With Hypersensitivity Reactions [ Time Frame: Baseline up to end of the study (8 years) ]
    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.

  5. Occurrences of Pregnancies/breastfeeding for Women of Child-bearing Potential [ Time Frame: Baseline up to end of the study (8 years) ]
    Pregnancy testing for women of child-bearing potential will be collected throughout the survey.

  6. Change From Baseline in Hemoglobin Concentration [ Time Frame: Baseline, Every 12 weeks up to 8 years ]
    Hemoglobin concentration will be assessed.

  7. Change From Baseline in Platelet Count [ Time Frame: Baseline, Every 12 weeks up to 8 years ]
    Platelet count will be assessed.

  8. Change From Baseline in Liver Volume [ Time Frame: Baseline, Every 24 weeks up to 8 years ]
    Liver volume will be measured at the physician's discretion per standard clinical practice.

  9. Change From Baseline in Spleen Volume [ Time Frame: Baseline, Every 24 weeks up to 8 years ]
    Spleen volume will be measured at the physician's discretion per standard clinical practice.

  10. Change From Baseline in Bone Density [ Time Frame: Baseline, Every 52 weeks up to 8 years ]
    Bone density will be assessed at the physician's discretion per standard clinical practice.

  11. Number of Participants With Infusion-related Reactions (IRRs) [ Time Frame: Baseline up to end of the study (8 years) ]
    Infusion-related reactions are defined as reactions occurring up to 24 hours after the start of the infusion.

  12. Number of Participants with anti-velaglucerase alfa antibodies [ Time Frame: Baseline up to end of study (8 years) ]
    Anti-velaglucerase alfa antibodies will be assessed.



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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:

-


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03625882


Contacts
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Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com

Locations
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Japan
Konan Recruiting
Konan, Aichi, Japan, 483-8704
Nagoya Recruiting
Nagoya, Aichi, Japan, 453-0801
Kitakyushu Recruiting
Kitakyushu, Fukuoka, Japan, 802-0803
Kurume Recruiting
Kurume, Fukuoka, Japan, 830-0011
Fukuyama Recruiting
Fukuyama, Hiroshima, Japan, 721-0927
Higashihiroshima Recruiting
Higashihiroshima, Hiroshima, Japan, 739-0041
Obihiro Recruiting
Obihiro, Hokkaido, Japan, 080-0016
Sagamihara Recruiting
Sagamihara, Kanagawa, Japan, 252-0375
Sendai Recruiting
Sendai, Miyagi, Japan, 980-8574
Suita Recruiting
Suita, Osaka, Japan, 565-0871
Tondabayashi Recruiting
Tondabayashi, Osaka, Japan, 584-0000
Kawagoe Recruiting
Kawagoe, Saitama, Japan, 350-8550
Tokorozawa Recruiting
Tokorozawa, Saitama, Japan, 359-8513
Moriyama Recruiting
Moriyama, Shiga, Japan, 524-0022
Otsu Recruiting
Otsu, Shiga, Japan, 520-2192
Matsue Recruiting
Matsue, Shimane, Japan, 690-0864
Hamamatsu Recruiting
Hamamatsu, Shizuoka, Japan, 431-3125
Hamamatsu Recruiting
Hamamatsu, Shizuoka, Japan, 434-8511
Iwata Recruiting
Iwata, Shizuoka, Japan, 438-0002
Minato Recruiting
Minato, Tokyo, Japan, 105-8471
Sumida-ku Recruiting
Sumida-ku, Tokyo, Japan, 130-0005
Yonago Recruiting
Yonago, Tottori, Japan, 683-8504
Chiba Recruiting
Chiba, Japan, 266-0007
Gifu Recruiting
Gifu, Japan, 500-8212
Hiroshima Recruiting
Hiroshima, Japan, 730-0046
Hiroshima Recruiting
Hiroshima, Japan, 734-0037
Kagoshima Recruiting
Kagoshima, Japan, 892-0853
Kumamoto Recruiting
Kumamoto, Japan, 860-8556
Kyoto Recruiting
Kyoto, Japan, 606-8507
Okayama Recruiting
Okayama, Japan, 701-1192
Osaka Recruiting
Osaka, Japan, 534-0021
Osaka Recruiting
Osaka, Japan, 545-8586
Osaka Recruiting
Osaka, Japan, 553-0003
Saitama Recruiting
Saitama, Japan, 339-8551
Shizuoka Recruiting
Shizuoka, Japan, 420-8688
Sponsors and Collaborators
Shire
Investigators
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Study Director: Study Director Shire

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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT03625882     History of Changes
Other Study ID Numbers: SHP-GCB-401
First Posted: August 10, 2018    Key Record Dates
Last Update Posted: May 8, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Shire provides access to the de-identified individual participant data for eligible studies to aid qualified researchers in addressing legitimate scientific objectives. These IPDs will be provided following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.shiretrials.com website. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
URL: https://www.shiretrials.com/en/our-commitment-to-transparency/data-sharing-with-researchers

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Gaucher Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders