EUCTR2021-005267-48-IT招募中1 期
A Phase 2 Open-Label Study to Evaluate Safety and Clinical Activity of FT-4202 in Patients with Thalassemia or Sickle Cell Disease - 4202-HEM-201
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 60
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •All Cohorts:
- •1. Patient has provided documented informed consent or assent (the informed consent form [ICF] must be reviewed and signed by each patient; in the case of adolescent patients, both the consent of the patient’s legal representative or legal guardian, and the patient’s assent must be obtained)
- •2. Age 12 to 65 years, inclusive, at time of first dose.
- •3. Patients, who if female and of childbearing 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.
- •Cohort A (SCD Transfusion Cohort):
- •4. Male or female study patient with a confirmed diagnosis of sickle cell disease
- •Documentation of SCD genotype (HbSS, HbSß0-thalassemia or other sickle cell syndrome variants) may be based on history of laboratory testing or must be confirmed by laboratory testing during Screening.
- •5. Chronically RBC transfused for primary stroke prevention or due to previous stroke. Chronic RBC transfusion is defined as: >= 6 RBC units in the previous 24 weeks before the first dose of study treatment and no transfusion-free period for > 35 days during that period.
- •6. Receiving chronic RBC transfusion by straight transfusions.
- •7. At least 24 months of chronic monthly RBC transfusions for primary stroke prevention or treatment of primary stroke (initial completed overt clinical stroke with document infarction on brain computed tomography [CT] or magnetic resonance imaging [MRI]).
- •8. On iron chelation therapy for > 3 months prior to enrollment.
- •9. Documented adequate monthly transfusions with average HbS <= 45% (the upper limit of the established academic community standard) for the previous 12 weeks of RBC transfusions before the first dose of study treatment.
- •Cohort B (Thalassemia Transfusion Cohort):
- •10. Male or female study patients with documented diagnosis of ß-thalassemia, Hemoglobin E/ ß-thalassemia or Hemoglobin H (a-thalassemia).
- •11. Chronically transfused, defined as: >= 6 RBC units in the previous 24 weeks before the first dose of study treatment and no transfusion-free period for > 35 days during that period.
- •12. On iron chelation therapy for > 3 months prior to enrollment.
- •Cohort C (Thalassemia Non-transfused Cohort):
- •13. Male or female study patients with documented diagnosis of ß-thalassemia, Hemoglobin E/ ß-thalassemia or Hemoglobin H (a-thalassemia).
- •14. Hemoglobin <= 10 g/dL.
- •Are the trial subjects under 18? yes
- •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 44
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •1.Female breast feeding or pregnant
- •2.Hepatic dysfunction characterized by
- •ALT >4.0 × ULN
- •Direct bilirubin >3.0 × ULN
- •History of cirrhosis
- •3.Clinic significant and active bacterial, fungal, parasitic, or viral infect
- •Patients with acute bacterial,fungal,parasitic,or viral infect. requiring systemic therapy should delay Screening/enrollment until active therapy has been completed
- •Patients with acute viral infections without available therapies should delay Screening/enrollment until the acute infect. has resolved
- •4.Known HIV positivity
- •5.Active infection with hepatitisB virus
- •6.Active hepatitisC infect
- •7.Severe renal dysfunct(estimated glomerular filtration rate at Screening visit; calculated by local lab <30 mL/min/1.73 m2) or on chronic dialysis
- •8.History of malignancy within past 2years prior to treatment D1 requiring systemic chemotherapy and/or radiation
- •Patients with malignancy considered surgically cured are eligible
- •9.History of unstable or deteriorating cardiac or pulmonary disease within 6months prior to consent including but not limited to:
- •Unstable angina pectoris or myocardial infarction or elective coronary intervention
- •Heart disease, heart failure as classified by the NYHA classification 3 or higher,or significant arrhythmia requiring treatment
- •Pulmonary fibrosis or pulmonary hypertension are clinically significant ie>=Grade3 NCI Common Terminology Criteria for Adverse Events(CTCAE)version 4.0(or higher)
- •10.Condition affecting drug absorption,such as major surgery involving stomach or small intestine(prior cholecystectomy acceptable)
- •11.Chronic systemic glucocorticoids <=3months(90D) prior to first dose of study treatment(physiologic replacement therapy for adrenal insufficiency is allowed).Single day glucocorticoid treatment(eg, for prevention or treatment of transfusion reactions allowed)
- •12.Receiving or use of concomitant medications are strong inducers or moderate/strong inhibitors of cytochrome P450(CYP)3A4/5 within 2 weeks of starting study treatment or anticipated need for such agents during study
- •13.Use of erythropoietin or other hematopoietic growth factor treatment within 30D of starting study treatment or anticipated need for such agents during study
- •14.Receipt of prior cellular-based therapy(eg, hematopoietic cell transplant, gene modification therapy)
- •15.Initiation of new chelation therapy within 3months before first dose of study treatment
- •16.Participated in another clinical trial within 30D or 5 half-lives of date of ICF, whichever is longer,or is currently participating in another trial
- •17.Inadequate venous access as determined by Investig/site staff
- •18.Medical,psychological,or behavioral conditions,which may preclude safe participation,confound study interpretation,interfere with compliance,or preclude ICF
- •19.History of severe brain vasculopathy by MRA showing moya-moya disease
- •20.Undergone an exchange RBC transfusion(manual or erythrocytopheresis)within previous 3months
- •21.New auto- or alloantibody affecting the ability to phenotypically match donor RBCs at the start of study treatment(D1)
- •22.Current use of other therapeutic agents for SCD(eg, HU, voxelotor, crizanlizumab)within 30D of starting study treatment and until end of study treatment period
- •23.Hemoglobin S/ß-thalassemia
- •24.New auto- or alloantibody affecting the ability to phenotypically match donor RBCs at start of study treatment(D1)
- •25.Current use of HU within 30D of starting study treatm and until end of study treatm
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