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An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00635427
Recruitment Status : Completed
First Posted : March 13, 2008
Results First Posted : January 28, 2014
Last Update Posted : June 10, 2021
Sponsor:
Information provided by (Responsible Party):
Takeda ( Shire )

Study Description
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Brief Summary:
The purpose of this study is to evaluate the long-term safety of every other week dosing of Gene-Activated® human glucocerebrosidase (GA-GCB, velaglucerase alfa) intravenously in patients with type 1 Gaucher disease.

Condition or disease Intervention/treatment Phase
Gaucher Disease, Type 1 Biological: VPRIV® Phase 3

Detailed Description:
Type 1 Gaucher disease, the most common form,accounts for more than 90% of all cases 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. Gene-Activated® human glucocerebrosidase (GA-GCB,velaglucerase alfa) 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. GA-GCB contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease. This study was designed to determine the long-term safety of GA-GCB in men, women, and children with Type 1 Gaucher disease.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 95 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease
Actual Study Start Date : March 13, 2008
Actual Primary Completion Date : December 28, 2012
Actual Study Completion Date : December 28, 2012

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039)

This arm is the Overall velaglucerase alfa (VPRIV) 60 U/kg and includes patients from the following groups:

VPRIV 45 U/kg or 60 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044 to maintain blindness or 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)

 Biological: VPRIV®
Intravenous infusion, every other week (EOW)
Other Names:
  • velaglucerase alfa
  • Gene-Activated® Human Glucocerebrosidase(GA-GCB)
Experimental: VPRIV 60 U/kg (Parent study-imiglucerase(60 U/kg) HGT-GCB-039)
imiglucerase 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)and switched 60 U/kg VPRIV in HGT-GCB-044
 Biological: VPRIV®
Intravenous infusion, every other week (EOW)
Other Names:
  • velaglucerase alfa
  • Gene-Activated® Human Glucocerebrosidase(GA-GCB)
Experimental: VPRIV 15-60 U/kg (Parent study VPRIV (15-60 U/kg) TKT034)
VPRIV 15- 60 U/kg, IV, EOW for 51 weeks in parent study TKT034 (NCT00478647) and continued in HGT-GCB-044 at the same dose as prescribed in TKT034
 Biological: VPRIV®
Intravenous infusion, every other week (EOW)
Other Names:
  • velaglucerase alfa
  • Gene-Activated® Human Glucocerebrosidase(GA-GCB)


Outcome Measures
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Primary Outcome Measures :
  1. Overall Summary of Treatment Emergent Adverse Events [ Time Frame: Baseline to termination of study ]
    Safety was evaluated by an analysis of adverse events (AEs), concomitant medication use, clinical laboratory tests, vital signs during the infusion of study drug, physical examination, and the development of anti-velaglucerase alfa. No formal comparisons or statistical tests were applied for the safety analyses, including for differences between the groups.


Secondary Outcome Measures :
  1. Change From Baseline to 24 Months in Hemoglobin Concentration for Each Treatment Group [ Time Frame: Baseline to 24 months ]
  2. Change From Baseline to 24 Months in Platelet Counts for Each Treatment Group [ Time Frame: Baseline to 24 months ]
  3. Change From Baseline to 24 Months in Normalized Liver Volume for Each Treatment Group [ Time Frame: Baseline to 24 months ]
  4. Percentage Change From Baseline to 24 Months in Normalized Spleen Volume for Each Treatment Group [ Time Frame: Baseline to 24 months ]

Eligibility Criteria
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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. The patient has completed study TKT032 or TKT034, or study HGT-GCB-039.
  2. Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have negative results to a pregnancy test performed at the time of enrollment and as required throughout their participation in the study.
  3. Male patients must agree to use a medically acceptable method of contraception at all times during the study and report a partner's pregnancy to the investigator.
  4. 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).
  5. The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator

Exclusion Criteria:

  1. The patient has received treatment with any non-Gaucher disease-related investigational drug or device within the 30 days prior to study entry; such use during the study is not permitted.
  2. The patient is pregnant or lactating.
  3. 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.
  4. The patient has a significant comorbidity(ies) that might affect study data or confound the study results (e.g., malignancies, primary biliary cirrhosis, autoimmune liver disease, etc.).
  5. The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult, has an uncooperative attitude, is unable to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator
Contacts and Locations
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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): NCT00635427


Locations
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Sponsors and Collaborators
Shire
Investigators
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Study Director: Study Director Takeda
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00635427    
Other Study ID Numbers: HGT-GCB-044
2008-001965-27 ( EudraCT Number )
First Posted: March 13, 2008    Key Record Dates
Results First Posted: January 28, 2014
Last Update Posted: June 10, 2021
Last Verified: May 2021
Keywords provided by Takeda ( Shire ):
VPRIV
Enzyme Replacement Therapy
Gaucher disease
glucocerebrosidase
beta-glucocerebrosidase
 Acid beta-glucocerebrosidase
glucosylceramidase
D-glucosyl-N-acylsphingosine glucohydrolase
gene activation
human
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