EUCTR2007-003359-35-DE进行中（未招募）不适用
A RANDOMIZED, OPEN-LABEL STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF AT2101 IN TREATMENT-NAIVE ADULT PATIENTS WITH TYPE 1 GAUCHER DISEASE
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 16
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
- •Subjects must meet all of the following criteria in order to be considered for enrollment into the study:
- •Confirmed diagnosis of type 1 Gaucher disease with a known genotype and a documented missense gene mutation in at least one of the two mutated GBA alleles
- •Clinically stable
- •Treatment naïve to ERT/SRT or has not received ERT/SRT in the 12 months before screening
- •Willing not to initiate ERT or SRT treatment during study participation
- •Male or female subjects, 18 to 74 years old inclusive
- •At the screening period visit (Day -21 to Day -1), subjects must meet at least two of the following criteria:
- •- Platelet count of =150,000 per microliter (=150 x 109 per liter)
- •- Hemoglobin =12 g/dL for females and =13 g/dL for males
- •- Liver volume =1.25 multiples of normal
- •- Spleen volume =2 multiples of normal
- •All subjects of reproductive potential are required to practice an acceptable method of contraception as defined in Section 5.5 of the protocol
- •Provide written informed consent to participate in 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
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Subjects presenting with any of the following will not be included in the study:
- •A clinically significant disease, severe complications from Gaucher disease, or serious intercurrent illness that may preclude participation in the study in the opinion of the Investigator
- •During the screening period, any clinically significant findings, based on physical and brief neurological examination, medical history review, laboratory assessment, vital sign assessment and/or other significant finding which would compromise the safety of the subject, or preclude the subject from completing the study as deemed by the Investigator
- •Partial or total splenectomy
- •Documentation of moderate or severe pulmonary hypertension, defined as pulmonary arterial pressure (PAP) >35 mmHg or significant Gaucher related lung disease
- •History of allergy or sensitivity to the study drug or any excipients, including any prior serious allergic reaction to iminosugars (e.g., miglustat)
- •Screening period visit (Day -21 to Day -1) or Day 1 12-lead ECG demonstrating a QTc >450 msec for males or >470 msec for females, prior to dosing
- •Pacemaker or other contraindication for MRI scanning
- •Pregnant or breast-feeding
- •Current/recent drug or alcohol abuse
- •Treatment with any investigational product in the last 90 days before study entry
- •Treatment in the previous 90 days with any drug known to have a well defined potential for toxicity to a major organ
- •Presence or sequelae of gastrointestinal, liver or kidney disease, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs
- •Subject is otherwise unsuitable for the study in the opinion of the Investigator
研究者
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