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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00430625
Other study ID # TKT032
Secondary ID
Status Completed
Phase Phase 3
First received
Last updated
Start date February 15, 2007
Est. completion date April 1, 2009

Study information

Verified date June 2021
Source Takeda
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to this deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this study is to evaluate the efficacy of every other week dosing of Gene-Activated® Human Glucocerebrosidase (GA-GCB, velaglucerase alfa) at doses of 45 and 60 U/kg in treatment-naïve patients with type 1 Gaucher disease.


Description:

Type 1 Gaucher disease, the most common form, accounts for more than 90% of all cases and does not involve the Central Nervous System (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. Velaglucerase alfa 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 efficacy, safety and pharmacokinetics of GA-GCB in men, women, and children with Type 1 Gaucher disease. Each patients duration of treatment was 12 months.


Recruitment information / eligibility

Status Completed
Enrollment 25
Est. completion date April 1, 2009
Est. primary completion date April 1, 2009
Accepts healthy volunteers No
Gender All
Age group 2 Years and older
Eligibility Inclusion Criteria: - Patient has a documented diagnosis of type 1 Gaucher disease, as determined by deficient glucocerebrosidase (GCB) activity relative to normal as measured in leukocytes or by genotype analysis and is willing and able to provide written informed consent prior to initiating any study-related procedures - Patient is at least 2 years of age - Patient has Gaucher disease-related anemia and - Patient has at least moderate splenomegaly or - Patient has Gaucher disease-related thrombocytopenia or - Patient has a readily palpable enlarged liver - Patient has not received treatment for Gaucher disease within 30 months prior to study entry - Female patients of child-bearing potential agree to use a medically acceptable method of contraception. Male patients must agree to use a medically acceptable method of birth control. - Patient must be sufficiently cooperative to participate in the study as judged by the Investigator. Exclusion Criteria: Includes: - Patient has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease - Patient is antibody-positive to imiglucerase during screening or has experienced an anaphylactic reaction to imiglucerase - Patient has received treatment with any investigational drug or device within the 30 days prior to study entry - Patient is Human immunodeficiency virus (HIV) positive - Patient is hepatitis positive - Patient presents with iron, folic acid and/or vitamin B12 deficiency sustained anemia during screening - 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 - Patient has a significant comorbidity(ies)that might affect study data or confound the study results - Patient is a pregnant and/or lactating female - Patient is unable to comply with the protocol or is unlikely to complete the study, as determined by the Investigator

Study Design


Related Conditions & MeSH terms


Intervention

Biological:
VPRIV ®,
Intravenous (IV) infusion, every other week via intravenous infusion for 12 months

Locations

Country Name City State
Argentina Hipolito Yrigoyen Buenos Aires
Israel Shaare Zedek Medical Center Jerusalem
Paraguay Sociedad Espanola de Socorros Mutuos Asuncion
Russian Federation National Research Center for Haematology Moscow
Tunisia La Rabta Hospital Tunis

Sponsors (1)

Lead Sponsor Collaborator
Shire

Countries where clinical trial is conducted

Argentina,  Israel,  Paraguay,  Russian Federation,  Tunisia, 

Outcome

Type Measure Description Time frame Safety issue
Primary Change From Baseline to 12 Months in Hemoglobin Concentration for the 60 U/kg Treatment Group. Efficacy endpoint Week 53
Secondary Change From Baseline to 12 Months in Hemoglobin Concentration in 45 U/kg Treatment Group Week 53
Secondary Change From Baseline to 12 Months in Platelet Counts for Each Treatment Group. intent to treat (ITT) Population Week 53
Secondary Change From Baseline to 12 Months in Normalized Liver Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI) Liver Volume has been normalized for percentage of body weight for each treatment arm. Liver size relative to body weight = (Liver volume [cc]/Body weight [kg])*100 Week 51
Secondary Change From Baseline to 12 Months in Normalized Spleen Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI)) 12 patients in the 60 U/kg group and 13 patients in the 45 U/kg group were analyzed for efficacy in the intent to treat (ITT) population. Spleen Volume has been normalized for percent of body weight for each treatment arm. Spleen size relative to body weight = (Spleen volume [cc]/Body weight [kg])*100 Week 51
Secondary Percent Change From Baseline to 12 Months in Plasma Chitotriosidase for Each Treatment Group Percent Change from Baseline to Weeks 53 by Randomized velaglucerase alfa Treatment Group - Subset of intent to treat (ITT) Population who were wild type homozygous for chitotriosidase. Week 53
Secondary Percent Change From Baseline to 12 Months in Chemokine (C-C Motif) Ligand 18 (CCL18) Week 53
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