LBCTR2021124934招募中2 期
An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 344
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized controlled trial
- 主要目的
- Treatment
- 盲法
- Blinded (masking used)
入排标准
- 年龄范围
- 18 至 65（—）
- 性别
- All
入选标准
- •Participants are eligible to be included in the study only if all the following criteria apply:
- •Informed Consent
- •1. Patient has provided documented informed consent or assent (the informed consent form [ICF] must be reviewed and signed by each patient)
- •2. Age 18 to 65 years, inclusive, at time of randomization
- •Type of Participant and Disease Characteristics
- •3. Patient has a confirmed diagnosis of sickle cell disease
- •Documentation of SCD genotype (HbSS, HbSß0-thalassemia or other sickle cell syndrome variants) based on prior history of laboratory testing; if unavailable, must be confirmed by laboratory testing during screening
- •4. Patient has had at least 2 episodes 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)
- •5. Hemoglobin = 5.5 and = 10 g/dL (= 55 and = 100 g/L) during screening
- •6. For participants taking HU, the dose of HU (mg/kg) must be stable (no more than a 20% change in dosing) for at least 90 days prior to start of study treatment with no anticipated need for dose adjustments during the study, in the opinion of the Investigator
- •Sex and Contraceptive/Barrier Requirements
- •7. Patients, who if female and of child bearing potential, are using highly effective methods of contraception and agree not to donate ova from study start to 90 days after the last dose of study drug, and who if male are willing to use barrier methods of contraception and agree not to donate sperm, from study start to 90 days after the last dose of study drug
排除标准
- •Patients are excluded from the study if they meet any of the following criteria:
- •Medical Conditions
- •1. More than 10 VOCs (as defined in Inclusion Criterion 4) within the past 12 months
- •2. Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF
- •3. Female who is breast feeding or pregnant
- •4. Hepatic dysfunction characterized by:
- •Alanine aminotransferase (ALT) > 4.0 × upper limit of normal (ULN)
- •Direct bilirubin > 3.0 × ULN
- •5. Patients with clinically significant bacterial, fungal, parasitic, or viral infection requiring systemic therapy
- •Patients with acute bacterial, fungal, parasitic, or viral infection requiring systemic therapy should delay screening/enrollment until active therapy has been completed
- •Note: Infection prophylaxis is allowed (see concomitant medication restrictions)
- •6. Known human immunodeficiency virus (HIV) positivity
- •7. Active infection with hepatitis B virus (hepatitis B surface antigen [HepBsAg] and hepatitis B core antibody [HepBcAb] positive)
- •8. Active hepatitis C infection
- •9. Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the central laboratory < 30 mL/min/1.73 m2) or on chronic dialysis
- •10. History of malignancy within the past 2 years prior to treatment Day 1 requiring systemic chemotherapy and/or radiation
- •Patients with malignancy considered surgically cured are eligible (eg, non-melanoma skin cancer, cancer of the cervix in-situ, ductal carcinoma in situ [stage 1], grade 1 endometrial cancer)
- •11. 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
- •Symptomatic pulmonary hypertension
- •12. History of overt clinical stroke within previous 2 years or any history of an intracranial hemorrhage
- •13. Any condition affecting drug absorption, such as major surgery involving the stomach or small intestine (prior cholecystectomy is acceptable)
- •14. Patients with iron deficiency (eg, serum iron less than the lower limit of normal [LLN] or ferritin < 10 µg/L) who are not taking or are unable to take iron supplements at the time of consent and during the study
- •15. Patients with folate (or folic acid or Vitamin B9) or Vitamin B12 deficiency (eg, folate or Vitamin B12 levels less than the LLN) who are not taking or are unable to take supplements before the first dose of study drug and during the study
- •16. Patients who are not taking or are unable to take antimalarial prophylaxis at the time of consent and during the study if they live in areas of endemic malaria where prophylaxis is recommended
- •Prior/Concomitant Therapy
- •17. Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion)
- •Patients who have received an RBC transfusion for any reason within 60 days of the Screening period are eligible if HbA (adult Hb) < 10% by Hb electrophoresis before start of study treatment
- •18. Receiving or use of concomitant medications that are strong inducers or moderate/strong inhibitors of CYP3A4/5 within 2 weeks of starting study treatment or anticipated need for such agents during the study
- •19. Use of voxelotor within 28 days prior to starting study treatment
研究者
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