NCT00842088已完成1 期
A Randomized, Blinded, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics 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
- 1 期
- 状态
- 已完成
- 入组人数
- 24
- 试验地点
- 9
- 主要终点
- Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs, and (4) physical exam.
研究概览
简要总结
The purpose of this study is to assess the safety and tolerability of HQK-1001 administered for a total of 12 weeks (with one dosing break) 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
- Triple (Participant, Care Provider, Investigator)
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
- •Diagnosis of SCD or sickle beta thalassemia (excluding Hemoglobin C)
- •Between 12 and 60 years of age, inclusive
- •At least one episode of a SCD-related crisis or complication (e.g., vaso-occlusive crisis, acute chest syndrome, priapism) per year for an average of 3 years or one episode of acute chest syndrome over the prior 5 years
- •Screening (untransfused) HbF level >/= 2% as analyzed by a central laboratory
- •If receiving hydroxyurea therapy, must be receiving a stable dose for at least 6 months
- •Able and willing to give informed consent
- •If female, must have a negative serum pregnancy test within 7 days of dosing
- •If female, must not be of childbearing potential defined as post-menopausal by at least 2 years or surgically sterile, or must agree to use a medically accepted form of contraception throughout the study
- •If the sexual partner of a male subject is a WCBP, she must agree to use a medically accepted form of birth control for themselves or their partner throughout the study
- •In the view of the Investigator, able to comply with necessary study procedures
排除标准
- •Red blood cell (RBC) transfusion within 3 months prior to beginning study medication
- •Participation in a regular blood transfusion program
- •More than 4 hospitalizations for acute sickle cell-related events in the previous 12 months
- •An acute vaso-occlusive event within 3 weeks prior to receiving first dose of study medication
- •Pulmonary hypertension requiring oxygen
- •QTc > 450 msec on screening
- •Alanine transaminase (ALT) > 3X upper limit of normal (ULN)
- •Creatinine phosphokinase (CPK) > 20% above the ULN
- •Serum creatinine >1.2 mg/dL
- •An acute illness (e.g., febrile, gastrointestinal [GI], respiratory) within 72 hours prior to receiving first dose of study medication
- •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 administration of study medication
- •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
Active
Experimental
干预措施: HQK-1001 (Drug)
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
结局指标
主要结局
Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs, and (4) physical exam.
时间窗: 126 days
次要结局
- Pharmacokinetics assessed by plasma drug concentration levels.(Days 0, 6, 69 and 97 post first dose)
- Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin.(Every 2 weeks through Day 126 post first dose)
研究者
研究点 (9)
Loading locations...
相似试验
已完成
1 期
Phase 1/2 Study of HQK-1001 in Patients With Beta ThalassemiaBeta ThalassemiaNCT00790127HemaQuest Pharmaceuticals Inc.21
已完成
2 期
A Study of HQK-1001 in Patients With Sickle Cell DiseaseSickle Cell DisordersHemoglobin S DiseaseSickling Disorder Due to Hemoglobin SSickle Cell DiseaseSickle Cell AnemiaNCT01322269HemaQuest Pharmaceuticals Inc.52
已完成
1 期
Phase 1 Study With OAB Assessing the Safety and Activity of hMaxi-K Gene TransferOveractive BladderNCT01870037Urovant Sciences GmbH13
已完成
1 期
Dose Escalation Study of KB001 in Cystic Fibrosis Patients Infected With Pseudomonas AeruginosaCystic FibrosisNCT00638365Humanigen, Inc.27
已完成
1 期
A Single Dose Study of KHK6640 in Japanese Patients With Alzheimer's Disease.Alzheimer's DiseaseNCT02377713Kyowa Kirin Co., Ltd.20