NCT01322269已完成2 期
A Randomized, Open-Label, Multi-Dose Study of HQK-1001 in Subjects With Sickle Cell Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 52
- 试验地点
- 16
- 主要终点
- Safety
研究概览
简要总结
The purpose of this study is to evaluate the safety and tolerability of three dose levels of HQK-1001 administered once daily for 26 weeks in subjects with sickle cell disease.
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
- None
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
- •Established diagnosis of SCD
- •Males and females between 12 and 60 years of age, inclusive
- •At least 3 episodes of a SCD-related crisis or complication in the 3 years prior to screening OR 1 episode of acute chest syndrome in the 5 years prior to screening
- •If receiving hydroxyurea, must be receiving a stable dose for at least 6 months prior to screening
- •If hydroxyurea treatment has been discontinued, at least 3 months have elapsed since last dose
- •If transfusion in the 4 months prior to screening, then HbA level < 20% at screening
- •Average of the initial two HbF levels ≥ 2.0 % within ≤ 7 days prior to the initial dose of HQK-
- •The two must be obtained ≥ 24 hours apart
- •Ability to swallow tablets
- •Able and willing to give informed consent and assent (if applicable)
- •If subject is a woman of child-bearing potential (WCBP), she must have a negative serum pregnancy test within 7 days of first dose of HQK-1001
- •If a subject is a WCBP, she must agree to use an effective form of contraception within 7 days of the initial dose of HQK-1001 and for one month after HQK-1001 discontinuation
- •Sexually active male subjects (with WCBP partners) must agree to use latex condoms or ensure that their partner(s) use an effective form of contraception
- •In the view of the Investigator, subject is able and willing to comply with necessary study procedures
排除标准
- •More than 4 hospitalizations for acute sickle cell related events in the previous 12 months prior to screening
- •Pulmonary hypertension requiring oxygen therapy
- •QTc > 450 msec (male) or 470 msec (female) on screening ECG (QT corrected by Fridericia's formula)
- •Assigned to a regular transfusion program
- •Use of erythropoiesis stimulating agents within 90 days of screening
- •ALT > 3x upper limit of normal (ULN)
- •Serum creatinine > 1.2 mg/dL
- •A serious, concurrent illness that would limit ability to complete or comply with the study requirements
- •An acute vaso-occlusive event within 3 weeks prior to screening
- •Creatine phosphokinase (CK) > 20% above the ULN
- •An acute illness (e.g., febrile, GI, respiratory) within 72 hours prior to screening
- •History of syncope, clinically significant dysrhythmias or resuscitation from sudden death
- •Chronic opiate use, which, in the view of the Investigator, could confound evaluation of an investigational drug
- •Current abuse of alcohol or drugs
- •Received another investigational agent within 4 weeks or 5 half-lives, whichever is longer, prior to screening
- •Currently pregnant or breast feeding a child
- •Known infection with HIV-1
- •Infection with hepatitis B or hepatitis C, such that patients are currently on therapy or will be placed on therapy during the trial
研究组 & 干预措施
HQK-1001 (50 mg/kg)
Experimental
干预措施: HQK-1001 (Drug)
HQK-1001 (30 mg/kg)
Experimental
干预措施: HQK-1001 (Drug)
HQK-1001 (40 mg/kg)
Experimental
干预措施: HQK-1001 (Drug)
结局指标
主要结局
Safety
时间窗: Day 1 through Week 30
Physical exams, vital signs, clinical laboratory safety assessments, ECG and adverse event monitoring.
次要结局
- Fetal hemoglobin levels(Day 1 and Weeks 4, 8, 12, 16, 20, 25, 26 and 30)
- Incidence of sickle cell crisis events(Day 1 through Week 30)
研究者
研究点 (16)
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