Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease
Primary Purpose
Gaucher Disease
Status
Completed
Phase
Phase 3
Locations
Japan
Study Type
Interventional
Intervention
velaglucerase alfa
Sponsored by
About this trial
This is an interventional treatment trial for Gaucher Disease
Eligibility Criteria
Inclusion Criteria:
- The patient has a documented diagnosis of Gaucher disease
- The patient is at least 2 years of age
- Female patients of child bearing potential must agree to use a medically acceptable method of contraception at all times during the study
- The patient, the patient's parent(s) or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC)
- The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator
Patients who are switched from imiglucerase ERT must meet the following additional criteria:
- Received treatment with imiglucerase for a minimum of 12 consecutive months
- Meet predefined limits for hemoglobin concentration and platelet counts
Patients naïve to treatment for Gaucher disease must meet the following additional criteria:
- Not received treatment for Gaucher disease (investigational or approved products) within 12 months prior to study entry
- Have Gaucher disease related anemia and at least one of the following: moderate splenomegaly or, Gaucher disease-related thrombocytopenia or Gaucher disease-related enlarged liver
Exclusion Criteria:
- Treatment with any investigational drug or device within the 30 days prior to study entry (time of informed consent); such use during the study is not permitted
- Positive for hepatitis B or hepatitis C.
- Non-Gaucher disease related anemia
- The patient, patient's parent(s), or patient's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study
- Significant comorbidity, as determined by the Investigator that might affect study data or confound the study results
- The patient is unable to comply with the protocol or is unlikely to complete the study, as determined by the Investigator
- The patient has experienced a severe (grade 3 or higher) infusion-related hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any ERT (approved or investigational)
- Currently receiving red blood cell growth factor, (eg, erythropoietin) or chronic systemic corticosteroids in the last 6 months
- Patient has had a splenectomy or the patient has an active, clinically significant spleen infarction within 12 months of screening
- Patient has worsening bone necrosis within 12 months of screening
- The patient is pregnant or lactating.
Sites / Locations
- Hamamatsu University School of Medicine
- The Jikei University School of Medicine
- Osaka City University Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Investigational
Arm Description
velaglucerase alfa
Outcomes
Primary Outcome Measures
Number of Severe Adverse Events (SAE)
Number of Treatment Emergent Adverse Events (TEAE)
Development of Anti-velaglucerase Alfa Antibody
Number of Infusion- Related Adverse Events
Number of Patients With Concomitant Medication
Secondary Outcome Measures
Change From Baseline in Hemoglobin Concentration
Change From Baseline in Platelet Count
Change From Baseline in Liver Volume, Normalized to Body Weight
Change From Baseline in Spleen Volume, Normalized to Body Weight
Change From Baseline in Plasma Chitotriosidase Levels
Change From Baseline in CCL18 Levels
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01614574
Brief Title
Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease
Official Title
A Multicenter, Open-Label Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease
Study Type
Interventional
2. Study Status
Record Verification Date
June 2021
Overall Recruitment Status
Completed
Study Start Date
March 2, 2012 (Actual)
Primary Completion Date
May 25, 2013 (Actual)
Study Completion Date
May 25, 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shire
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. The disease has been classified into 3 clinical subtypes based on the presence or absence of neurological symptoms and severity of neurological disease. Type 1 Gaucher disease affects an estimated 30,000 persons worldwide and is the most common. Type 1 Gaucher disease does not involve the central nervous system. Patients with type 2 Gaucher disease present with acute neurological deterioration, which leads to early death. Those with type 3 disease typically display a more sub-acute neurological course, with later onset and slower progression.
The primary objective of this study is to evaluate the safety of every other week dosing of velaglucerase alfa in Japanese patients with Gaucher disease.
Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type 1 Gaucher disease.
Detailed Description
Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain.
Gaucher disease has been designated in the list of Specified Rare and Intractable Diseases by Specified Disease Treatment Research Program of Ministry of Health, Labor and Welfare (MHLW) as one of "lysosomal storage diseases" since 2001. Gaucher disease is also designated in the Medical Aid Program for Specified Categories of Chronic Pediatric Diseases.
The prevalence of mutations and the phenotype of patients with Gaucher disease in Japan differs from that in non-Japanese populations. Some patients with type 1 Gaucher disease in Japan have more severe and progressive disease compared to non-Japanese patients and the disease is characterized by an earlier onset of symptoms.
Velaglucerase alfa, a highly-purified form of the naturally occurring enzyme glucocerebrosidase, has been developed as an enzyme replacement therapy for Gaucher disease for the symptoms (anemia, thrombocytopenia, hepatomegaly, splenomegaly, and bone manifestation).
The primary objective of this study is to evaluate the safety of every other week dosing of velaglucerase alfa in Japanese patients (naive or previously treated with imiglucerase) 2 years of age and older with Gaucher disease.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gaucher Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Investigational
Arm Type
Experimental
Arm Description
velaglucerase alfa
Intervention Type
Biological
Intervention Name(s)
velaglucerase alfa
Other Intervention Name(s)
VPRIV, Gene activated human glucocerebrosidase
Intervention Description
60 U/kg every other week intravenous infusion
Primary Outcome Measure Information:
Title
Number of Severe Adverse Events (SAE)
Time Frame
Baseline to week 51
Title
Number of Treatment Emergent Adverse Events (TEAE)
Time Frame
Baseline to week 51
Title
Development of Anti-velaglucerase Alfa Antibody
Time Frame
Baseline to week51
Title
Number of Infusion- Related Adverse Events
Time Frame
Baseline to week 51
Title
Number of Patients With Concomitant Medication
Time Frame
Baseline to week 51
Secondary Outcome Measure Information:
Title
Change From Baseline in Hemoglobin Concentration
Time Frame
Baseline to week 51
Title
Change From Baseline in Platelet Count
Time Frame
Baseline to week 51
Title
Change From Baseline in Liver Volume, Normalized to Body Weight
Time Frame
Baseline to week 51
Title
Change From Baseline in Spleen Volume, Normalized to Body Weight
Time Frame
Baseline to week 51
Title
Change From Baseline in Plasma Chitotriosidase Levels
Time Frame
Baseline to week 51
Title
Change From Baseline in CCL18 Levels
Time Frame
Baseline to week 51
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
The patient has a documented diagnosis of Gaucher disease
The patient is at least 2 years of age
Female patients of child bearing potential must agree to use a medically acceptable method of contraception at all times during the study
The patient, the patient's parent(s) or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC)
The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator
Patients who are switched from imiglucerase ERT must meet the following additional criteria:
Received treatment with imiglucerase for a minimum of 12 consecutive months
Meet predefined limits for hemoglobin concentration and platelet counts
Patients naïve to treatment for Gaucher disease must meet the following additional criteria:
Not received treatment for Gaucher disease (investigational or approved products) within 12 months prior to study entry
Have Gaucher disease related anemia and at least one of the following: moderate splenomegaly or, Gaucher disease-related thrombocytopenia or Gaucher disease-related enlarged liver
Exclusion Criteria:
Treatment with any investigational drug or device within the 30 days prior to study entry (time of informed consent); such use during the study is not permitted
Positive for hepatitis B or hepatitis C.
Non-Gaucher disease related anemia
The patient, patient's parent(s), or patient's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study
Significant comorbidity, as determined by the Investigator that might affect study data or confound the study results
The patient is unable to comply with the protocol or is unlikely to complete the study, as determined by the Investigator
The patient has experienced a severe (grade 3 or higher) infusion-related hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any ERT (approved or investigational)
Currently receiving red blood cell growth factor, (eg, erythropoietin) or chronic systemic corticosteroids in the last 6 months
Patient has had a splenectomy or the patient has an active, clinically significant spleen infarction within 12 months of screening
Patient has worsening bone necrosis within 12 months of screening
The patient is pregnant or lactating.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
Hamamatsu University School of Medicine
City
Hamamatsu
State/Province
Shizuoka
ZIP/Postal Code
431-3192
Country
Japan
Facility Name
The Jikei University School of Medicine
City
Minato-ku
State/Province
Toyko
ZIP/Postal Code
105-8461
Country
Japan
Facility Name
Osaka City University Hospital
City
Osaka
ZIP/Postal Code
545-0051
Country
Japan
12. IPD Sharing Statement
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Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease
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