EUCTR2009-015811-42-SE进行中（未招募）1 期
A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once Daily versus Twice Daily Dosing of Genz-112638 in Patients with Gaucher Disease Type 1 who have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638 - EDGE
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 170
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Patients must meet all of the following inclusion criteria in order to
- •participate in this study:
- •1.The patient is willing and able to provide signed informed consent
- •prior to any study-related procedures.
- •2.The patient is =18 years of age.
- •3.The patient has a diagnosis of Gaucher disease type 1 confirmed by a
- •documented deficiency of acid ß glucosidase activity by enzyme assay.
- •The patient may be previously untreated, off prior treatment, or
- •receiving enzyme replacement therapy for Gaucher disease.
- •4.The patient meets all of the following criteria at the time of Screening:
- •Hemoglobin level =9 g/dL (mean of 2 measurements);
- •Platelet count =70,000/mm3 (mean of 2 measurements);
- •Spleen volume =25 multiples of normal (MN);
- •Liver volume =2.0 MN.
- •5.The patient consents to provide a blood sample for genotyping for Gaucher disease and for cytochrome P450 2D6 (CYP2D6) to categorize
- •the patient's predicted rate of metabolism, if these genotyping results
- •are not already available for the patient.
- •6.Female patients of childbearing potential must have a documented
- •negative pregnancy test prior to administration of the first dose of Genz-
- •112638 in this study. In addition, all female patients of childbearing
- •potential must use a medically accepted form of contraception
- •throughout the study, i.e., either a barrier method or hormonal
- •contraceptive with norethindrone and ethinyl estradiol or similar active
- •components.
- •7.The patient is willing to abstain from consumption of grapefruit,
- •grapefruit juice, or grapefruit products for 72 hours prior to
- •administration of the first dose of Genz 112638 and throughout the
- •duration of the study.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 170
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range 0
排除标准
- •Patients will be excluded from participation in this study if they meet
- •any of the following exclusion criteria:
- •1.The patient is participating in GZGD02607 study, A Phase 3,
- •Randomized, Multi-Center, Multi-National, Open-Label, Active
- •Comparator Study to Evaluate the Efficacy and Safety of Genz 112638 in
- •Patients with Gaucher Disease Type 1 who have been Stabilized with
- •Cerezyme, or is eligible for inclusion in GZGD02607 (while enrollment is
- •ongoing) and has access to a physician participating in GZGD02607.
- •The patient is participating in GZGD02507 study, A Phase 3,
- •Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study
- •Confirming the Efficacy and Safety of Genz 112638 in Patients with
- •Gaucher Disease Type 1, or is eligible for inclusion in GZGD02507 (while
- •enrollment is ongoing) and has access to a physician participating in
- •GZGD02507.
- •2.The patient received miglustat within 6 months prior to administration
- •of the first dose of Genz 112638 in this study.
- •3.The patient has had a partial or total splenectomy within 3 years prior
- •to administration of the first dose of Genz 112638 in this study.
- •4.The patient has any evidence of neurologic disorder (e.g., peripheral
- •neuropathy, tremor, seizures, Parkinsonism or cognitive impairment) or
- •pulmonary involvement (e.g., pulmonary hypertension) as related to
- •Gaucher disease.
- •5.The patient is transfusion-dependent.
- •6.The patient has a documented deficiency of iron, vitamin B-12, or
- •folate that requires treatment not yet initiated or, if initiated, the patient
- •has not been stable under treatment for at least 3 months prior to
- •administration of the first dose of Genz 112638 in this study.
- •7.The patient has documented prior esophageal varices or clinically
- •significant liver infarction or current liver enzymes (alanine
- •transaminase [ALT]/aspartate aminotransferase [AST]) or Total
- •Bilirubin > 2 times the upper limit of normal (ULN), unless the patient
- •has a diagnosis of Gilbert Syndrome.
- •8.The patient has any clinically significant disease, other than Gaucher
- •disease, including cardiovascular, renal, hepatic, gastrointestinal,
- •pulmonary, neurologic, endocrine, metabolic (including hypokalaemia or
- •hypomagnesemia), or psychiatric disease, other medical conditions, or
- •serious intercurrent illnesses that may preclude participation in the
- •9.The patient is known to have any of the following: Clinically significant
- •coronary artery disease including history of myocardial infarction [MI] or
- •ongoing signs or symptoms consistent with cardiac ischemia or heart
- •failure; or clinically significant arrhythmias or conduction defect such as 2nd or 3rd degree atrioventricular (AV) block, complete bundle branch
- •block, prolonged QTc interval, or sustained ventricular tachycardia (VT).
- •10.The patient is known to have tested positive for the human
- •immunodeficiency virus (HIV) antibody, Hepatitis C antibody, or
- •Hepatitis B surface antigen.
- •11.The patient has received an investigational product (other than Genz-
- •112638) within 30 days prior to administration of the first dose of Genz
- •112638 in this study.
- •12.The patient is scheduled for in-patient hospitalization, including
- •elective surgery, during the study.
- 另有 5 项未显示
研究者
相似试验
进行中（未招募）
不适用
A Phase 3, Randomized, Multi-Center, Multi-National, Double-Blind Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once Daily versus Twice Daily Dosing of Genz-112638 in Patients with Gaucher Disease Type 1 who have Demonstrated Clinical Stability on a Twice Daily Dose of Genz-112638 - EDGEGaucher Disease Type IMedDRA version: 12.1Level: PTClassification code 10018048Term: Gaucher's diseaseEUCTR2009-015811-42-GRGenzyme Europe B.V.234
进行中（未招募）
不适用
A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic goals with Enzyme Replacement Therapy - ENCOREGaucher Disease type IMedDRA version: 9.1Level: PTClassification code 10018048Term: Gaucher's diseaseEUCTR2008-005223-28-CZGenzyme Europe B.V.186
进行中（未招募）
1 期
A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic Goals with Enzyme Replacement Therapy - ENCOREGaucher Disease type IMedDRA version: 9.1Level: PTClassification code 10018048Term: Gaucher's diseaseEUCTR2008-005223-28-NLGenzyme Europe B.V.159
进行中（未招募）
不适用
A Study of Eliglustat Tartrate (Genz-112638) in Patients with Gaucher Disease Who Have Reached Thereapeutic Goals with Enzyme Replacement TherapyGaucher Disease type IMedDRA version: 18.0Level: PTClassification code 10018048Term: Gaucher's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2008-005223-28-DEGenzyme Europe B.V.150
进行中（未招募）
1 期
A study with Eliglustat Tartrate (Genz-112638) in Patients with Gaucher disease to Evaluate Once Daily Dosing versus Twice DailyGaucher Disease Type IMedDRA version: 14.1Level: PTClassification code 10018048Term: Gaucher's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2009-015811-42-PTGenzyme Europe B.V.170