Trial record 17 of 19 for:
"Gaucher disease type 1"
A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease
This study has been completed.
Sponsor:
Shire Human Genetic Therapies, Inc.
Information provided by:
Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier:
NCT00430625
First received: February 1, 2007
Last updated: August 19, 2010
Last verified: August 2010
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Gaucher Disease, Type 1 |
Biological: VPRIV ®, |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Investigator) Primary Purpose: Treatment |
| Official Title: | A Multicenter, Randomized, Double-Blind, Parallel Group, Two-Dose Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease |
Resource links provided by NLM:
Genetics Home Reference related topics:
Chanarin-Dorfman syndrome
cholesteryl ester storage disease
Farber lipogranulomatosis
Gaucher disease
Schindler disease
succinic semialdehyde dehydrogenase deficiency
MedlinePlus related topics:
Gaucher's Disease
U.S. FDA Resources
Further study details as provided by Shire Human Genetic Therapies, Inc.:
Primary Outcome Measures:
- Change From Baseline to 12 Months in Hemoglobin Concentration for the 60 U/kg Treatment Group. [ Time Frame: Week 53 ] [ Designated as safety issue: No ]Efficacy endpoint
Secondary Outcome Measures:
- Change From Baseline to 12 Months in Hemoglobin Concentration in 45 U/kg Treatment Group [ Time Frame: Week 53 ] [ Designated as safety issue: No ]
- Change From Baseline to 12 Months in Platelet Counts for Each Treatment Group. [ Time Frame: Week 53 ] [ Designated as safety issue: No ]intent to treat (ITT) Population
- Change From Baseline to 12 Months in Normalized Liver Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI) [ Time Frame: Week 51 ] [ Designated as safety issue: No ]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
- Change From Baseline to 12 Months in Normalized Spleen Volume (Percent Body Weight) for Each Treatment Group (Measured by Magnetic Resonance Imaging (MRI)) [ Time Frame: Week 51 ] [ Designated as safety issue: No ]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
- Percent Change From Baseline to 12 Months in Plasma Chitotriosidase for Each Treatment Group [ Time Frame: Week 53 ] [ Designated as safety issue: No ]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.
- Percent Change From Baseline to 12 Months in Chemokine (C-C Motif) Ligand 18 (CCL18) [ Time Frame: Week 53 ] [ Designated as safety issue: No ]
| Enrollment: | 25 |
| Study Start Date: | January 2007 |
| Study Completion Date: | May 2009 |
| Primary Completion Date: | April 2009 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: VPRIV®-45 U/kg, IV, every other week
VPRIV® (velaglucerase alfa, Gene Activated® human glucocerebrosidase, GA-GCB)
|
Biological: VPRIV ®,
Intravenous (IV) infusion, every other week via intravenous infusion for 12 months
Other Names:
|
|
Experimental: VPRIV®-60 U/kg, IV, every other week
VPRIV® (velaglucerase alfa, Gene Activated® human glucocerebrosidase,GA-GCB)
|
Biological: VPRIV ®,
Intravenous (IV) infusion, every other week via intravenous infusion for 12 months
Other Names:
|
Detailed 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.
Eligibility| Ages Eligible for Study: | 2 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00430625
Locations
| Argentina | |
| Hipolito Yrigoyen | |
| Buenos Aires, Argentina | |
| Israel | |
| Shaare Zedek Medical Center | |
| Jerusalem, Israel | |
| Paraguay | |
| Sociedad Espanola de Socorros Mutuos | |
| Asuncion, Paraguay | |
| Russian Federation | |
| National Research Center for Haematology | |
| Moscow, Russian Federation | |
| Tunisia | |
| La Rabta Hospital | |
| Tunis, Tunisia | |
Sponsors and Collaborators
Shire Human Genetic Therapies, Inc.
Investigators
| Study Director: | Kiran Bhirangi, M.D., FRCS | Shire Human Genetic Therapies, Inc. |
| Principal Investigator: | Ari Zimran, M.D. | Shaare Zedek Medical Center |
| Principal Investigator: | Derlis Emilio Gonzalez Rodriguez, M.D. | Sociedad Espanola de Socorros Mutuos |
| Principal Investigator: | Marie-Francoise Ben Dridi, Professor | La Rabta Hospital |
| Principal Investigator: | Isaac Kisinovsky, M.D. | Hipolito Yrigoyen |
| Principal Investigator: | Elena A. Lukina, Professor | National Research Center for Haematology |
More Information
Additional Information:
No publications provided
| Responsible Party: | Tiffany Crump, Medical Communications Manager, Shire Human Genetic Therapies, Inc. |
| ClinicalTrials.gov Identifier: | NCT00430625 History of Changes |
| Other Study ID Numbers: | TKT032 |
| Study First Received: | February 1, 2007 |
| Results First Received: | June 3, 2010 |
| Last Updated: | August 19, 2010 |
| Health Authority: | United States: Food and Drug Administration Paraguay: Ministerio de Salud Pública y Bienestar Social Israel: Ministry of Health Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica Russia: Ministry of Health of the Russian Federation Tunisia: Office of Pharmacies and Medicines |
Keywords provided by Shire Human Genetic Therapies, Inc.:
|
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:
|
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 |
ClinicalTrials.gov processed this record on May 19, 2013