EUCTR2019-001838-34-GB进行中（未招募）1 期
A Phase 2, Open Label, Multiple Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Voxelotor in Patients with Sickle Cell Disease
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 45
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
- •Participants who meet all the following criteria will be eligible for enrollment in the study:
- •1. Male or female with sickle cell disease
- •2. Documentation of SCD genotype HbSS or HbSB0
- •3. Age 18 to < 60 years, inclusive
- •4. Hemoglobin = 5.5 and = 10.5 g/dL during Screening, and considered stable and close to baseline by the investigator
- •5. For participants taking hydroxyurea (HU), the dose in mg/kg must be stable for at least 90 days prior to signing the informed consent form (ICF) and with no anticipated need for dose adjustments during the study, in the opinion of the Investigator.
- •6. Participants, who if female and of child bearing potential, agree to use highly effective methods of contraception or practicing abstinence from study start to 30 days after the last dose of study drug, and who if male, agree to use barrier methods of contraception or practice abstinence from study start to 30 days after the last dose of study drug.
- •7. Participant has provided documented informed consent
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 45
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Participants meeting any of the following exclusion criteria will not be eligible for study enrollment:
- •1. More than 10 vaso-occlusive crises (VOCs) within 12 months of screening that required a hospital, emergency room, or clinic visit
- •2. Female participant who is breast feeding or pregnant
- •3. Receiving regularly scheduled blood (red blood cell [RBC]) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or have received an RBC transfusion for any reason within 60 days of signing the ICF or at any time during the screening period
- •4. Hospitalized for sickle cell crisis or other vaso-occlusive event within 30 days prior to dosing (ie, a vaso-occlusive event cannot be within 30 days prior to dosing)
- •5. Screening laboratory test of alanine aminotransferase (ALT) > 4 × upper level of normal (ULN)
- •6. Clinically significant bacterial, fungal, parasitic, or viral infection which requires therapy, including acute bacterial infection requiring antibiotics
- •7. Known to be COVID-19 positive from within 3 weeks of screening through Day 1
- •8. Participants with known active hepatitis A, B, or C or who are known to be human immunodeficiency virus (HIV) positive
- •9. Severe renal dysfunction (estimated glomerular filtration rate [GFR] < 50 mL/min/1.73 m2 at the Screening visit; calculated by the local laboratory to assess safety) or is on chronic dialysis
- •10. 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)
- •11. History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following:
- •a. Unstable angina pectoris or myocardial infarction or elective coronary intervention
- •b. Congestive heart failure requiring hospitalization
- •c. Uncontrolled clinically significant arrhythmias
- •d. Pulmonary hypertension
- •12. Criteria related to ECG parameters:
- •a. PR interval > 220 msec in any participant
- •b. QRS interval > 120 msec or QT interval corrected using Fridericia’s formula (QTcF) > 480 msec (both genders) in participants without bundle branch block
- •c. QRS interval > 120 msec in participants with newly (within 3 months) emerged bundle branch block
- •d. A participant with stable bundle branch block with or without stable cardiac disease may be enrolled; QRS interval > 120 msec and QTcF interval > 480 msec are acceptable in these participants.
- •13. Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable)
- •14. 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
- •15. Inadequate venous access as determined by the Investigator/site staff
- •16. Medical, psychological, or behavioral conditions, which, in the opinion of the investigator, may preclude safe participation, confound study interpretation, interfere with compliance, or preclude informed consent
- •17. Received erythropoietin or other hematopoietic growth factor treatment within 28 days of signing ICF or is anticipated to require such agents during the study
- •18. Ongoing or recent (within 2 years) substance abuse
- •19. Known allergy to voxelotor
- •20. Use of herbal medications (eg, St. John’s Wort), se
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