Gaucher Disease, Type 1 Clinical Trial
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
Verified date | June 2021 |
Source | Takeda |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
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 |
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 |
Lead Sponsor | Collaborator |
---|---|
Shire |
Argentina, Israel, Paraguay, Russian Federation, Tunisia,
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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