Trial record 6 of 54 for:
GBA
Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase
This study has been completed.
Sponsor:
Shire Human Genetic Therapies, Inc.
Information provided by:
Shire Human Genetic Therapies, Inc.
ClinicalTrials.gov Identifier:
NCT00478647
First received: May 23, 2007
Last updated: September 20, 2010
Last verified: September 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 the 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 safety and efficacy of every other week dosing of GA-GCB (velaglucerase alfa) in patients with type 1 Gaucher disease who were previously treated with imiglucerase.
| Condition | Intervention | Phase |
|---|---|---|
|
Gaucher Disease, Type 1 |
Biological: GA-GCB (velaglucerase alfa) |
Phase 2 Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Multicenter Open-Label Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase |
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:
- Patients Which Experienced at Least One Adverse Event [ Time Frame: Week 53 ] [ Designated as safety issue: Yes ]
Safety was assessed throughout the study by assessments including adverse events, concomitant medication use, and vital signs. Additional safety assessments, including 12-lead ECGs, physical examinations, clinical laboratory tests and determination of the presence of anti-velaglucerase alfa antibodies.
Refer to Adverse event section for further details.
Secondary Outcome Measures:
- Change From Baseline to Week 53 in Hemoglobin Concentration [ Time Frame: Week 53 ] [ Designated as safety issue: No ]ITT patient population.
- Percent Change From Baseline to Week 53 in Platelet Count [ Time Frame: Week 53 ] [ Designated as safety issue: No ]ITT patient population.
- Percent Change From Baseline to Week 51 in Normalized Liver Volume [ Time Frame: Week 51 ] [ Designated as safety issue: No ]Liver volume has been normalized for percentage (%) of body weight. Liver size relative to body weight= (Liver volume [cc]/Body weight [kg])*100
- Percent Change From Baseline to Week 51 in Normalized Spleen Volume [ Time Frame: Week 51 ] [ Designated as safety issue: No ]Spleen volume has been normalized for percentage (%) of body weight. Spleen size relative to body weight= (Spleen volume [cc]/Body weight [kg])*100
| Enrollment: | 40 |
| Study Start Date: | July 2007 |
| Study Completion Date: | August 2009 |
| Primary Completion Date: | July 2009 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: GA-GCB (velaglucerase alfa) |
Biological: GA-GCB (velaglucerase alfa)
15-60 U/kg, every other week via intravenous infusion
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 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 safety of GA-GCB in men, women, and children with Type 1 Gaucher disease who were previously treated with imiglucerase. Each patient's duration of treatment will be 12 months.
Eligibility| Ages Eligible for Study: | 2 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
Includes:
- The 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 the patient/legal guardian is willing and able to provide written informed consent prior to initiating any study-related procedures
- The patient has received consistent treatment with imiglucerase at a dose ≤ 60 U/kg and ≥ 15 U/kg every other week for a minimum of 30 consecutive months. Patients who are anti-imiglucerase antibody positive will be allowed to enter this study
- The patient is at least 2 years of age
- 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 co-operative to participate in the study as judged by the Investigator.
Exclusion Criteria:
Includes:
- The patient has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease
- The patient has received treatment with any investigational drug or device within the 30 days prior to study entry; such use during the study is not permitted
- Patient is HIV positive
- Patient is hepatitis B/C positive
- The patient presents with sustained iron, folic acid and/or vitamin B12 deficiency-related anemia during Screening
- 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
- The patient has a significant comorbidity that might affect study data or confound the study results
- The patient is unable to comply with the protocol or is otherwise unlikely to complete the study, as determined by the Investigator
- The patient has experienced an anaphylactic/anaphylactoid reaction during treatment with imiglucerase
- The patient has received miglustat during the 6 months prior to study enrollment
- The patient has an active, clinically significant spleen infarction
- The patient has active, progressive bone necrosis
- The patient is a pregnant and/or lactating female
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00478647
Locations
| United States, California | |
| Regional Metabolic Center | |
| Los Angeles, California, United States, 90027 | |
| Children's Hospital Oakland | |
| Oakland, California, United States, 94609 | |
| United States, Georgia | |
| Emory University | |
| Decatur, Georgia, United States, 30033 | |
| United States, Illinois | |
| Feinberg School of Medicine | |
| Chicago, Illinois, United States, 60614 | |
| United States, Minnesota | |
| Children's of Minnesota | |
| Minneapolis, Minnesota, United States, 55404 | |
| United States, Missouri | |
| Children's Mercy Hospital and Clinic | |
| Kansas City, Missouri, United States, 64108 | |
| United States, New York | |
| NYU School of Medicine | |
| New York, New York, United States, 10016 | |
| United States, Ohio | |
| Cincinatti Children's Hospital | |
| Cincinnati, Ohio, United States, 45229 | |
| United States, Texas | |
| Texas Children's Hospital | |
| Houston, Texas, United States, 77030 | |
| United States, Utah | |
| Medical Genetics/Pediatrics | |
| Salt Lake City, Utah, United States, 84132 | |
| United States, Wisconsin | |
| Children's Hospital of Wisconsin | |
| Milwaukee, Wisconsin, United States, 53226 | |
| Israel | |
| Shaare Zedek Medical Center | |
| Jerusalem, Israel | |
| Poland | |
| Children's Memorial Health Institute | |
| Warszawa, Poland | |
| Spain | |
| Hospital Universitario Miguel Servet | |
| Zaragoza, Spain, 500009 | |
| United Kingdom | |
| The Royal Free Hospital | |
| London, United Kingdom | |
Sponsors and Collaborators
Shire Human Genetic Therapies, Inc.
Investigators
| Study Director: | Eric Crombez, M.D. | Shire Human Genetic Therapies, Inc. |
| Principal Investigator: | Anna Tylki-Szmanska, M.D. | Children's Memorial Health Institute, Warszawa, Poland |
| Principal Investigator: | Joel Charrow, M.D. | Feinberg School of Medicine, Chicago, Illinois |
| Principal Investigator: | Laurie Smith, M.D. | Children's Mercy Hospital and Clinic, Kansas City, Missouri |
| Principal Investigator: | Ari Zimran, M.D. | Shaare Zedek Medical Center, Jerusalem, Israel |
| Principal Investigator: | Christine Eng, M.D. | Texas Children's Hospital, Houston, Texas |
| Principal Investigator: | Paul Fernhoff, M.D. | Emory University, Decatur, Georgia |
| Principal Investigator: | Gregory Grabowski, M.D. | Cincinatti Children's Hospital, Cincinnati, Ohio |
| Principal Investigator: | Paul Harmatz, M.D. | Children's Hospital Oakland, Oakland, California |
| Principal Investigator: | Margaret Heisel Kurth, M.D. | Chidlren's of Minnesota, Minneapolis, Minnesota |
| Principal Investigator: | Nicola Longo, M.D. | Medical Genetics/Pediatrics, Salt Lake City, Utah |
| Principal Investigator: | Rebecca Mardach, M.D. | Regional Metabolic Center, Los Angeles, California |
| Principal Investigator: | Gregory Pastores, M.D. | NYU School of Medicine, New York, New York |
| Principal Investigator: | William J. Rhead, M.D. | Children's Hospital of Wisconsin, Milwaukee, Wisconsin |
| Principal Investigator: | Athul Mehta, M.D. | The Royal Free Hosptial, London, United Kingdom |
| Principal Investigator: | Pilar Giraldo, M.D. | Hospital Universitario Miguel Servet, Zaragoza, Spain |
More Information
No publications provided
| Responsible Party: | Tiffany Crump, Senior Medical Affairs Associate, Shire Human Genetic Therapies, Inc. |
| ClinicalTrials.gov Identifier: | NCT00478647 History of Changes |
| Other Study ID Numbers: | TKT034 |
| Study First Received: | May 23, 2007 |
| Results First Received: | August 4, 2010 |
| Last Updated: | September 20, 2010 |
| Health Authority: | United States: Food and Drug Administration Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products Israel: Ministry of Health United Kingdom: Medicines and Healthcare Products Regulatory Agency Spain: Spanish Agency of Medicines |
Keywords provided by Shire Human Genetic Therapies, Inc.:
|
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