Back to resultsTreatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
Primary Purpose
Gaucher Disease, Type 1
Status
Approved for marketing
Phase
Locations
United States
Study Type
Expanded Access
Intervention
velaglucerase alfa
Sponsored by
About this trial
This is an expanded access trial for Gaucher Disease, Type 1 focused on measuring VPRIV, Enzyme Replacement Therapy, Gaucher disease, glucocerebrosidase, beta-glucocerebrosidase, Acid beta-glucocerebrosidase, glucosylceramidase, D-glucosyl-N-acylsphingosine glucohydrolase, gene activation, human
Eligibility Criteria
Inclusion Criteria:
- The patient has a documented diagnosis of type 1 Gaucher disease
- The patient is > 2 years of age
- The patient has NOT previously experienced an anaphylactic or anaphylactoid reaction to another ERT including imiglucerase
- Women of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study; and must have a negative result to a pregnancy test as required throughout their participation in the study. Male patients must use a medically acceptable method of birth control throughout their participation in the study and must report their partner's pregnancy.
- The patient is sufficiently cooperative to participate in this treatment plan as judged by the Investigator
If the patient is naïve or new to treatment, the patient has one or more of the following (in absence of the following criteria, please call the sponsor for treatment justification):
- Gaucher disease-related anemia
- Moderate splenomegaly (2 to 3 cm below the left costal margin), by palpation
- Gaucher disease-related thrombocytopenia
- Gaucher disease-related palpable enlarged liver
Exclusion Criteria: None
Sites / Locations
- St Joseph's Hospital & Medical Center
- Tower Hematology Oncology
- Rady's Children's Hospital of San Diego
- Southern California Permanente Medical Group
- The Permanente Medical Group
- Stanford University Medical Genetics
- Rocky Mountain Cancer Centers
- Yale University
- University Research Foundation for Lysosomal Storage Diseases
- Gainesville Hematology Oncology Associates
- Adventis Healthcare System dba Florida Hospital
- East Lake Oncology
- Emory Genetics
- Children's Memorial Hospital
- University of Iowa Hospitals and Clinics
- Annapolis Oncology Center
- Sinai Hospital of Baltimore
- University of Massachusetts
- Children's Hospitals and Clinics of Minnesota
- The University Research Foundation for Lysosomal Storage Diseases
- St. Joseph's
- Hemophilia Center of Western New York Incorporated
- North Shore Hematology/Oncology - Manhasset
- New York University School of Medicine
- Mount Sinai School of Medicine
- Fullerton Genetic
- Duke Medical Center
- Akron Children's Hospital
- Cincinnati Children's Hospital Medical Center
- Children's Hospital of Philadelphia
- University of Virginia Health Systems
- O & O Alpan, LLC
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00954460
Brief Title
Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
Official Title
Multicenter Open-Label Treatment Protocol to Observe the Safety of Gene-Activated™ Human Glucocerebrosidase (GA-GCB, Velaglucerase Alfa) ERT in Newly Diagnosed or Previously Treated (With Imiglucerase) Patients With Type 1 Gaucher Disease
Study Type
Expanded Access
2. Study Status
Record Verification Date
May 2021
Overall Recruitment Status
Approved for marketing
Study Start Date
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Primary Completion Date
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Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shire
4. Oversight
5. Study Description
Brief Summary
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this treatment protocol is to observe the safety of velaglucerase alfa in patients with type 1 Gaucher disease who are either treatment naive (newly diagnosed) or who are currently being treated with the Enzyme Replacement Therapy (ERT) imiglucerase.
Detailed Description
Type 1 Gaucher disease, the most common form, accounts for more than 90% of all cases of Gaucher disease and does not involve the CNS. Typical manifestations of type 1 Gaucher disease include hepatomegaly, splenomegaly, thrombocytopenia, bleeding tendencies, anemia, hypermetabolism, skeletal pathology, growth retardation, pulmonary disease, and decreased quality of life. Velaglucerase alfa (Gene-Activated™ human glucocerebrosidase;GA-GCB) is produced in a continuous human cell line using proprietary gene-activation technology and has an identical amino acid sequence to the naturally occurring human enzyme. Velaglucerase alfa contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease. This treatment protocol will observe the safety of velaglucerase alfa in patients with type 1 Gaucher disease who are either treatment naive (newly diagnosed) or who are currently being treated with the Enzyme Replacement Therapy (ERT) imiglucerase. Patients currently being treated with ERT for their Gaucher disease will receive the same number of units of velaglucerase alfa per month as their imiglucerase dose for doses between 30-120 U/kg/month. For patients who experienced dose reductions in their imiglucerase treatment due to supply constraints the pre-reduction monthly dose may be used to determine the monthly dose of velaglucerase alfa.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gaucher Disease, Type 1
Keywords
VPRIV, Enzyme Replacement Therapy, Gaucher disease, glucocerebrosidase, beta-glucocerebrosidase, Acid beta-glucocerebrosidase, glucosylceramidase, D-glucosyl-N-acylsphingosine glucohydrolase, gene activation, human
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
velaglucerase alfa
Other Intervention Name(s)
VPRIV, Gene activated human glucocerebrosidase, GA-GCB
Intervention Description
up to 60 U/kg, every other week via intravenous infusion
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Eligibility Criteria
Inclusion Criteria:
The patient has a documented diagnosis of type 1 Gaucher disease
The patient is > 2 years of age
The patient has NOT previously experienced an anaphylactic or anaphylactoid reaction to another ERT including imiglucerase
Women of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study; and must have a negative result to a pregnancy test as required throughout their participation in the study. Male patients must use a medically acceptable method of birth control throughout their participation in the study and must report their partner's pregnancy.
The patient is sufficiently cooperative to participate in this treatment plan as judged by the Investigator
If the patient is naïve or new to treatment, the patient has one or more of the following (in absence of the following criteria, please call the sponsor for treatment justification):
Gaucher disease-related anemia
Moderate splenomegaly (2 to 3 cm below the left costal margin), by palpation
Gaucher disease-related thrombocytopenia
Gaucher disease-related palpable enlarged liver
Exclusion Criteria: None
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
St Joseph's Hospital & Medical Center
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85013
Country
United States
Facility Name
Tower Hematology Oncology
City
Beverly Hills
State/Province
California
ZIP/Postal Code
90211-1850
Country
United States
Facility Name
Rady's Children's Hospital of San Diego
City
La Jolla
State/Province
California
ZIP/Postal Code
92093
Country
United States
Facility Name
Southern California Permanente Medical Group
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
The Permanente Medical Group
City
Sacramento
State/Province
California
ZIP/Postal Code
95815
Country
United States
Facility Name
Stanford University Medical Genetics
City
Stanford
State/Province
California
ZIP/Postal Code
94305-5208
Country
United States
Facility Name
Rocky Mountain Cancer Centers
City
Denver
State/Province
Colorado
ZIP/Postal Code
80218
Country
United States
Facility Name
Yale University
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06510
Country
United States
Facility Name
University Research Foundation for Lysosomal Storage Diseases
City
Coral Springs
State/Province
Florida
ZIP/Postal Code
33065
Country
United States
Facility Name
Gainesville Hematology Oncology Associates
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32605-4218
Country
United States
Facility Name
Adventis Healthcare System dba Florida Hospital
City
Orlando
State/Province
Florida
ZIP/Postal Code
32804-4603
Country
United States
Facility Name
East Lake Oncology
City
Palm Harbor
State/Province
Florida
ZIP/Postal Code
34685
Country
United States
Facility Name
Emory Genetics
City
Decatur
State/Province
Georgia
ZIP/Postal Code
30033
Country
United States
Facility Name
Children's Memorial Hospital
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60614
Country
United States
Facility Name
University of Iowa Hospitals and Clinics
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
Annapolis Oncology Center
City
Annapolis
State/Province
Maryland
ZIP/Postal Code
21401
Country
United States
Facility Name
Sinai Hospital of Baltimore
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21215
Country
United States
Facility Name
University of Massachusetts
City
Shrewsbury
State/Province
Massachusetts
ZIP/Postal Code
01545
Country
United States
Facility Name
Children's Hospitals and Clinics of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55404
Country
United States
Facility Name
The University Research Foundation for Lysosomal Storage Diseases
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108-4619
Country
United States
Facility Name
St. Joseph's
City
Paterson
State/Province
New Jersey
ZIP/Postal Code
07503
Country
United States
Facility Name
Hemophilia Center of Western New York Incorporated
City
Buffalo
State/Province
New York
ZIP/Postal Code
14215
Country
United States
Facility Name
North Shore Hematology/Oncology - Manhasset
City
Manhasset
State/Province
New York
ZIP/Postal Code
11030
Country
United States
Facility Name
New York University School of Medicine
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Mount Sinai School of Medicine
City
New York
State/Province
New York
ZIP/Postal Code
10029-6500
Country
United States
Facility Name
Fullerton Genetic
City
Asheville
State/Province
North Carolina
ZIP/Postal Code
28801-4420
Country
United States
Facility Name
Duke Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Akron Children's Hospital
City
Akron
State/Province
Ohio
ZIP/Postal Code
44308
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
University of Virginia Health Systems
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908-0386
Country
United States
Facility Name
O & O Alpan, LLC
City
Springfield
State/Province
Virginia
ZIP/Postal Code
22152
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
24263462
Citation
Pastores GM, Rosenbloom B, Weinreb N, Goker-Alpan O, Grabowski G, Cohn GM, Zahrieh D. A multicenter open-label treatment protocol (HGT-GCB-058) of velaglucerase alfa enzyme replacement therapy in patients with Gaucher disease type 1: safety and tolerability. Genet Med. 2014 May;16(5):359-66. doi: 10.1038/gim.2013.154. Epub 2013 Nov 21.
Results Reference
derived
Learn more about this trial
Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
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