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Treatment Protocol of Velaglucerase Alfa for 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: NCT00954460
Expanded Access Status : Approved for marketing
First Posted : August 7, 2009
Last Update Posted : May 21, 2021
Sponsor:
Information provided by (Responsible Party):
Takeda ( Shire )

Study Description
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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.

Condition or disease Intervention/treatment
Gaucher Disease, Type 1 Drug: velaglucerase alfa

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.
Study Design
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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Gaucher Disease

Interventions
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Intervention Details:
  • Drug: velaglucerase alfa
    up to 60 U/kg, every other week via intravenous infusion
    Other Names:
    • VPRIV
    • Gene activated human glucocerebrosidase
    • GA-GCB
Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Criteria

Inclusion Criteria:

  1. The patient has a documented diagnosis of type 1 Gaucher disease
  2. The patient is > 2 years of age
  3. The patient has NOT previously experienced an anaphylactic or anaphylactoid reaction to another ERT including imiglucerase
  4. 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.
  5. The patient is sufficiently cooperative to participate in this treatment plan as judged by the Investigator

  6. 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

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): NCT00954460


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: NCT00954460    
Other Study ID Numbers: HGT-GCB-058
First Posted: August 7, 2009    Key Record Dates
Last Update Posted: May 21, 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