EUCTR2016-003370-40-FR进行中（未招募）1 期
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of GBT440 Administered Orally to Patients With Sickle Cell Disease - HOPE
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 350
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
- •All participants must meet the following inclusion criteria:
- •1. Male or female study participants with Sickle Cell Disease:
- •Documentation of SCD genotype (HbSS, HbSC, HbSß thalassemia or other sickle cell syndrome variants) may be based on history of laboratory testing or must be confirmed by
- •laboratory testing during screening
- •2. Participants have had at least 1 episode of VOC in the past 12 months. For study eligibility, VOC is defined as a previously documented episode of ACS or acute painful crisis (for which there was no explanation other than VOC) which required prescription or healthcare professional-instructed use of analgesics for moderate to severe pain (documentation must exist in the patient medical record prior to Screening)
- •3. Age 12 to 65 years
- •4. Hemoglobin (Hb) =6.0 and =10.5 g/dL during screening
- •5. Absolute reticulocyte count and % reticulocyte count must be >1.5 × ULN during Screening
- •6. For participants taking hydroxyurea (HU), the dose of HU (mg/kg) must be stable for at least 3 months prior to signing the ICF and with no anticipated need for dose adjustments during the study, in the opinion of the Investigator
- •7. Participants must demonstrate 75% compliance with ePRO measure completion to be enrolled (participants will be given an ePRO device for at least 28 days during Screening; participants who are 60 to 74% compliant can re-screen once with Investigator approval; re-screening is not allowed for participants who are <60% compliant)
- •8. Participants, who if female and of child bearing potential, are using highly effective methods of contraception from study start to 3 months after the last dose of study drug, and who if male are willing to use barrier methods of contraception, from study start to 3 months after the last dose of study drug
- •9. Participant has provided documented informed consent or assent (the informed consent form [ICF] must be reviewed and signed by each participant; in the case of pediatric participants, both the consent of the participant’s legal representative or legal guardian, and the participant’s assent must be obtained)
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 50
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 350
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range 0
排除标准
- •Any participant who meets one or more of the following criteria will be excluded from participation:
- •1. More than 10 VOCs within the past 12 months that required a hospital, emergency room or clinic visit
- •2. Female who is breast feeding or pregnant
- •3. Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or have received a RBC transfusion for any reason within 28 days of signing the ICF
- •4. Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF
- •(i.e., a vaso-occlusive event cannot be within 14 days prior to ICF)
- •5. Hepatic dysfunction characterized by alanine aminotransferase (ALT) >4 × ULN
- •6. Participants with clinically significant bacterial, fungal, parasitic or viral infection which require therapy:
- •Participants with acute bacterial infection requiring antibiotic use should delay screening/enrollment until the course of antibiotic therapy has been completed.
- •Participants with known active hepatitis A, B, or C or who are known to be human immunodeficiency virus (HIV) positive
- •7. Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the central laboratory) <30mL/min/1.73² or on chronic dialysis
- •8. History of malignancy within the past 2 years prior to treatment Day 1 requiring chemotherapy and/or radiation (with the exception of local therapy for non-melanoma skin malignancy)
- •9. History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following:
- •Unstable angina pectoris or myocardial infarction or elective coronary intervention
- •Congestive heart failure requiring hospitalization
- •Uncontrolled clinically significant arrhythmias
- •10. Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable)
- •11. Participated in another clinical trial of an investigational agent (or medical device) within 30 days or 5 half-lives of date of informed consent, whichever is longer, or is currently participating in another trial of an investigational agent (or medical device)
- •12. Inadequate venous access as determined by the Investigator/site staff
- •13. Medical, psychological, or behavioral conditions, which, in the opinion of the Investigator, may confound study interpretation, interfere with compliance, or preclude informed consent
研究者
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