EUCTR2013-005324-41-GB进行中（未招募）1 期
A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed with Fabry Disease
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 8
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
- Male
入选标准
- •The patient is =18 years of age and <50 years of age.
- •The patient is male.
- •The patient has provided a signed informed consent.
- •The patient had a confirmed diagnosis of Fabry disease as documented by leukocyte a-Galactosidase A (aGAL) activity of <4 nmol/hr/mg leukocyte (preferred assay; results from a central laboratory) or plasma aGAL <1.5 nmol/hr/mL (results from a central laboratory).
- •The patient has a plasma globotriaosylsphingosine (lyso-GL3) >80 ng/mL.
- •The patient has never been treated with a Fabry disease-specific treatment.
- •If the patient is on renin-angiotensin-aldosterone system (RAAS) blockers and antidepressants, the dose should be stable (ie, prescribed dose and frequency) for at least the immediate 3 months prior to screening.
- •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 8
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •The patient has an estimated glomerular filtration rate (eGFR) <60 mL/min/1.73 m^2 using (Chronic Kidney Disease Epidemiology Collaboration) CKD-EPI.
- •The patient has a median urine protein/creatinine ratio (PCR) =0.5 g/g (median of 3 overnight urine collections. Collection of each of the 3 samples must occur between 4 and 7 days of each other, and all samples must be collected within a 21 day period). All 3 samples must be collected regardless of the results and results available prior to Day 1.
- •The patient had undergone a kidney transplant.
- •The patient has either active or a history of clinically significant organic disease (with the exception of the symptoms related to Fabry disease), including clinically significant cardiovascular, hepatic, pulmonary, hematologic, neurological or renal disease, or other medical condition, serious inter-current illness, or extenuating circumstances that, in the opinion of the Investigator, would preclude participation in the trial.
- •The patient has abnormal liver function (serum total bilirubin >the upper limit of normal, or serum alanine aminotransferase [ALT] and aspartate aminotransferase [AST] >2.0 times the upper limit of normal).
- •The patient has, according to World Health Organization (WHO) Grading a cortical cataract (COR) >one-quarter of the lens circumference (Grade COR-2) or a posterior subcapsular cataract (PSC) >2 mm (Grade PSC-2). Patients with nuclear cataracts are not excluded.
- •The patient is being administered a chronic regimen (more frequently than every 2 weeks) of any dose or route of corticosteroids or any medication that may cause cataract, according to the prescribing information.
- •The patient has received strong or moderate inducers or inhibitors of Cytochrome P450 3A4 (CYP3A4) per Food and Drug Administration (FDA) classification within 14 days prior to enrolment or within 5 times the elimination half-life or PD half-life of the medication, whichever is longer.
- •The patient is scheduled for in-patient hospitalization, including elective surgery, during the study.
- •The patient has a positive result on any of the following tests: hepatitis B surface antigen (HBsAg), anti-hepatitis C virus (anti-HCV) antibodies, anti-human immunodeficiency virus 1 and 2 antibodies (anti-HIV1 and anti-HIV2 Ab). Patients with a positive hepatitis B surface antibody (HBsAb) test with a history of prior hepatitis B immunization are eligible if other criteria are met (ie, negative tests for:
- •HBsAg, hepatitis B core antibody [HBcAb], and hepatitis C virus antibody [HCVAb]).
- •The patient has participated in a study involving an investigational drug within the past 30 days of the start of the trial.
- •The patient is unwilling to comply with the requirements of the protocol.
- •The patient is a sexually active man who is not willing to use 2 forms of birth control including a barrier method during the study.
- •The patient has history or ongoing clinically significant cardiac arrhythmia, defined as either atrial fibrillation, sustained or non-sustained ventricular tachycardia
- •The patient has any contraindication to magnetic resonance imaging (MRI).
- •The patient has one of the following central nervous system exclusion criteria:
- •Acute stroke, within 3 months of the screening visit.
- •History of seizures.
研究者
相似试验
进行中（未招募）
1 期
Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ402671 in Treatment-naïve Adult Male Patients with Fabry DiseaseFabry diseaseMedDRA version: 17.1Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2013-005324-41-CZGenzyme Corporation8
进行中（未招募）
1 期
Safety, Pharmacokinetics and Clinical Activity of AZD0171 in Combination with Durvalumab and Chemotherapy in Locally Advanced or Metastatic Solid TumoursPancreatic ductal adenocarcinomaMedDRA version: 21.0Level: LLTClassification code 10033599Term: Pancreatic adenocarcinoma metastaticSystem Organ Class: 100000004864EUCTR2021-002040-78-ESAstraZeneca AB115
招募中
1 期
(VELA) Study of BLU-222 in Advanced Solid TumorsHR+ Breast CancerCCNE1 AmplificationHER2-negative Breast CancerAdvanced Solid TumorOvarian CancerEndometrial CancerGastric CancerMedDRA version: 21.1Level: LLTClassification code: 10065147Term: Malignant solid tumor Class: 10029104CTIS2022-502528-29-00Blueprint Medicines Corp.366
进行中（未招募）
1 期
A Phase 2A Study Investigating the Safety, Pharmacokinetics, Immunogenicity, and Exploratory Efficacy of Dupilumab in Patients Aged =6 to <18 Years With Atopic DermatitisEUCTR2014-003263-37-HURegeneron Pharmaceuticals, Inc.78
进行中（未招募）
不适用
A study to determine the safety and tolerability of REGN668 in patients aged =6 to <18 years with atopic dermatitis (eczema)EUCTR2014-003263-37-PLRegeneron Pharmaceuticals, Inc.80