NCT00790127已完成1 期
A Multi-National, Blinded, Placebo-Controlled, Dose Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HQK-1001 in Subjects With Beta Thalassemia Intermedia, Including Hemoglobin E Beta Thalassemia
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 21
- 试验地点
- 2
- 主要终点
- Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam
研究概览
简要总结
The purpose of this study is to evaluate the safety and tolerability of HQK-1001 administered daily for 8 weeks in subjects with beta thalassemia intermedia
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 12 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of beta thalassemia intermedia or hemoglobin E beta thalassemia
- •Average of the initial two hemoglobin levels of less than 10 g/dL within ≥ 30 days prior to randomization.
- •Age ≥ 12 and ≤ 60 years
- •Able and willing to give informed consent or assent (if less than 18 years of age) and comply with all study procedures
- •If female and of childbearing potential, must have a documented negative pregnancy test at Day -1 and must agree to use one or more locally medically accepted methods of contraception in the month prior to randomization, through the entire study, and for 4 weeks after the last dosing of study medication
排除标准
- •Spleen palpable ≥ 2 cm below the left costal margin
- •Pulmonary hypertension requiring oxygen therapy
- •QTc > 450 msec on screening ECG
- •Infection with hepatitis C, hepatitis B requiring therapy
- •Known infection with HIV
- •Red blood cell (RBC) transfusions within 2 months prior to administration of study medication with stable hemoglobin levels
- •Fever greater than 38.5°C in the week prior to administration of study medication
- •ALT > 4x upper limit of normal (ULN)
- •Baseline elevation of CPK value prior to randomization
- •Treatment with hydroxyurea within 2 months prior to administration of study medication
- •Cancer diagnosis within 5 years of randomization (except for non-melanoma cutaneous malignancies)
- •Serum creatinine > 1.5 mg/dl
- •Received investigational systemic therapy within 30 days prior to randomization
- •Currently pregnant or breast feeding a child
- •Subject history of clinically significant arrhythmias or syncope
- •Known current drug or alcohol abuse
研究组 & 干预措施
Placebo
Placebo Comparator
Placebo
干预措施: Placebo (Drug)
HQK-1001
Experimental
HQK-1001
干预措施: HQK-1001 (Drug)
结局指标
主要结局
Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam
时间窗: 168 days
次要结局
- Pharmacokinetics assessed by plasma drug concentration levels(140 days)
- Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin(168 days)
研究者
研究点 (2)
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