NCT01601340终止2 期
A Randomized, Placebo-controlled, Phase 2 Study of HQK-1001 in Sickle Cell Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 77
- 试验地点
- 18
- 主要终点
- Change from baseline in % fetal hemoglobin
研究概览
简要总结
The purpose of this study is to evaluate the effects of HQK-1001 on Hb F 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
- 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
- •Males and females between 12 and 60 years of age
- •Diagnosis of SCD, type Hb SS or Hb S-B0 Thalassemia
- •At least 1 episode of SCD pain crisis, acute chest syndrome, other acute SCD complications, or leg ulcers in the 12 months prior to screening
- •Not being treated with Hydroxyurea (HU); if HU treatment has been previously administered and then discontinued, at least 3 months must have elapsed since last dose of HU
- •If subject has been transfused in the 3 months prior to screening, then Hb A level < 20% at screening
- •Baseline Hb F level obtained within 14 days prior to randomization
- •Able to swallow tablets
- •Able and willing to give informed consent and/or assent
- •If subject is a woman of child-bearing potential (WCBP), she must have a negative serum pregnancy test within 14 days of first dose of HQK-1001 and a negative urine pregnancy test prior to dosing on Day 1
- •If a subject is a WCBP, she must agree to use an effective form of contraception starting at screening and for one month after HQK-1001 discontinuation
- •Sexually active male subjects who have not had a vasectomy must agree to use latex condoms with WCBP partners or ensure that their partner(s) use an effective form of contraception starting at screening and for one month after HQK-1001 discontinuation.
排除标准
- •Assigned to a regular transfusion program
- •Use of erythropoiesis stimulating agents within 90 days prior to screening
- •An SCD pain crisis or SCD-related acute complication within 3 weeks prior to randomization
- •More than 5 SCD pain crisis or SCD-related acute complications within 12 months prior to screening
- •Pulmonary hypertension requiring therapy
- •ALT or AST > 3x ULN
- •Serum creatinine > 1.5x ULN
- •Serum amylase levels > 1.5x ULN
- •Serum lipase level > 1.5x ULN
- •A serious, concurrent illness that would limit ability to complete or comply with the study requirements
- •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 due to SCD-related complication
- •Symptomatic peptic ulcer, hiatus hernia, or gastroesophageal reflux disease (GERD)
- •History of pancreatitis
- •Chronic opiate use, which, in the view of the investigator, could confound evaluation of an investigational drug
- •Current abuse of alcohol or drugs
- •Use of 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 subjects are currently on anti-viral therapy or will be placed on therapy
研究组 & 干预措施
HQK-1001
Active Comparator
干预措施: HQK-1001 (Drug)
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
结局指标
主要结局
Change from baseline in % fetal hemoglobin
时间窗: Day 1 through Week 48
次要结局
- Safety measured by the frequency and severity of adverse events, and changes from baseline in vital signs, electrocardiogram (ECG) monitoring, and laboratory assessments(Day 1 through Week 52)
- HQK-1001 pharmacokinetic parameters(1 hour prior to, and 2 hours following morning dose on Weeks 12, 24 and 48)
- Incidence and number of SCD pain crises and SCD-related complications(Day 1 through Week 52)
- Subject reported daily pain scale scores and analgesic use(7 consecutive days following clinic visits at Day 1, and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48)
- Change in FACIT Fatigue Scale results(Day 1 and Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, and 48)
研究者
研究点 (18)
Loading locations...
相似试验
终止
1 期
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Oral GSK4172239D Compared With Placebo in Sickle Cell Disease Participants Aged 18 to 50 YearsHematologic DiseasesAnaemia, Sickle CellNCT05660265GlaxoSmithKline11
Unknown
3 期
A Sickle CEll Disease ComplicatioN TrialSickle Cell DiseaseNCT02604368Micelle BioPharma Inc210
已完成
1 期
Phase 1/2 Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK-1001 Administered Daily in Patients With Sickle Cell DiseaseSickle Cell DiseaseNCT00842088HemaQuest Pharmaceuticals Inc.24
已完成
2 期
Acetaminophen for the Reduction of Oxidative Injury in Severe SepsisSevere SepsisNCT01739361Vanderbilt University44
已完成
2 期
A Phase 2 Clinical Study of KHK4827Moderate to Severe Plaque PsoriasisNCT01748539Kyowa Kirin Co., Ltd.140