LBCTR2021064783尚未招募2 期
A Phase 2a, double-blind, randomised, placebo-controlled, ascending dose and maintenance dose, efficacy, and safety study of multiple doses of VIT-2763 in subjects with sickle cell disease
适应症
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 25
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 50（—）
- 性别
- All
入选标准
- •Inclusion Criteria:
- •1. Subject has provided the appropriate written informed consent before any study-specific procedures are performed including screening procedures.
- •2. Ability to understand the requirements of the study and abide by the study restrictions, and agreement to return for the required assessments.
- •3. Male or female subjects with confirmed diagnosis of SCD, including HbS/S or HbS/ßT0 genotype.
- •4. Subjects who had at least 1 and no more than 6 VOC episodes reported within 12 months prior to screening. Note: A VOC episode is defined as a
- •documented episode of acute chest syndrome or acute painful crisis for the main indication of SCD, which led to health-professional instructed prescription or use of analgesics for moderate to severe pain.
- •5. 18 to 50 years of age inclusive at the time of screening.
- •6. Body weight =50 kg and =100 kg at screening and baseline.
- •7. Absolute reticulocyte count and percentage reticulocyte count >1.5 × upper limit of normal (ULN) during screening.
- •8. Subjects on concomitant hydroxyurea must be on a stable dose (mg/kg) for =3 months prior to screening Visit V1. There should be no planned dose adjustments during the course of the study in the opinion of the Investigator.
- •9. Female subjects of childbearing potential, must have negative pregnancy tests at screening and before randomisation, must have stopped breastfeeding as of first dose, and must either commit to true abstinence from heterosexual contact (which must be reviewed on a monthly basis and source documented) or must be willing to use adequate contraceptive precautions, i.e., highly effective method of birth control. Abstinence should only be used as a contraceptive method if it is in line with the subjects’ usual and preferred lifestyle, and periodic abstinence (calendar, symptothermal, postovulation methods) is not an acceptable method of contraception. Female subjects must agree to use
- •adequate contraception during the study and for 1 month after the last dose of investigational medicinal product (IMP) or according to local requirements, whichever is longer. Effective contraception (highly effective method of birth control, i.e., with a failure rate of <1% per year, when used consistently and correctly) such as implants, injectables, combined oral contraceptives (see below), intrauterine devices, sexual abstinence, or vasectomised partner must be used. Non-childbearing potential includes being surgically sterilised at least 6 months prior to the study.
- •Note: For female subjects participating in this study, continuous use of hormonal contraception alone is not sufficient, because potential interactions
- •via cytochrome P450 (CYP) enzymes may alter the efficacy of hormonal contraception. The continuous use of hormonal contraception by a female
- •subject should be combined with the use of a condom by the male partner; the condom should then be used together with a spermicide or adequate andapproved alternatives.
- •10. Male subjects must practice true abstinence (abstinence should only be used as a contraceptive method if it is in line with the subjects’ usual and preferred lifestyle, and it is continuous throughout the study) or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, and for at least 1 month–sufficiently exceeding 5 times the mean t1/2 of VIT-2763 based on multiple dose human PK data following IMP discontinuation, even if he has und
排除标准
- •Exclusion Criteria:
- •1. Subjects with confirmed diagnosis of HbS/ßT+ genotype or HbSC disease.
- •2. Hb level <6.0 g/dl or >10.4 g/dl at screening Visit V1 (based on local laboratory value).
- •Note: The Hb value at screening Visit V1 will be used for eligibility determination. However, the baseline Hb value determined at Visit V2 (Day 1
- •pre-dose) also needs to be within the above specified range.
- •3. Having received RBC transfusion therapy within 4 weeks prior to screening, or ongoing or planned RBC transfusion therapy during the course of the study (including chronic, prophylactic, or preventive transfusion to treat SCD).
- •4. Ferritin level <30 µg/l or calculated transferrin saturation (TSAT) level <25% or total iron-binding capacity (TIBC) level <250 µg/dl at screening.
- •5. Subjects being hospitalised for SCD-related events (including pain crisis and VOC) within 30 days before the screening visit. Note: SCD must have been the main cause for the hospitalisation to fulfil this criterion.
- •6. Chronic liver disease or history of liver cirrhosis, and/or alanine aminotransferase or aspartate aminotransferase, above 3-fold the ULN range at
- •7. Estimated glomerular filtration rate (eGFR) <45 ml/min/1.73 m2, and/or significant urinary albumin/creatinine ratio >30 mg/g (>3.39 mg/mmol) at
- •screening or on chronic dialysis. Note: eGFR should be estimated according to Chronic Kidney Disease Epidemiology Collaboration formula (CKD-EPI).
- •8. Newly diagnosed folate deficiency anaemia (i.e., folic acid <2 ng/ml), which is considered clinically relevant by the Investigator at screening. Subjects with known folate deficiency anaemia who are on =12 weeks stable replacement therapy at screening are eligible. Note: A subject fulfilling this criterion will be excluded but can be re-screened at a later time point.
- •9. Subjects with history of partial or total splenectomy within 6 months prior tothe screening visit.
- •10. Any history or clinically important finding of cardiac or pulmonary disorders, including (but not limited to) clinically relevant or uncontrolled cardiac
- •arrhythmia, cardiomyopathy, coronary disease (unstable angina pectoris or myocardial infarction or elective coronary intervention), valve disorder, or
- •heart failure according to New York Heart Association classification 3-4.
- •11. Known pulmonary hypertension, defined as a tricuspid regurgitant velocity (TRV) =2.5 m/s, NT-proBNP =160 pg/ml or confirmed by right heart
- •catheterisation.
- •12. Any clinically relevant abnormal 12-lead electrocardiogram (ECG) finding during screening or prior to randomisation (as deemed by the Investigator)including (but not limited to) any of the following:
- •PR interval >0.21 seconds
- •Evidence or history of second- or third-degree atrioventricular block
- •QRS interval >0.12 seconds
- •13. Family history of long-QT syndrome or sudden death without a preceding diagnosis of a condition that could be causative of sudden death (such as known coronary artery disease, congestive heart failure, or terminal cancer), or subjects with QT interval corrected (QTcF) >450 msec.
- •14. Clinically significant bacterial, fungal, parasitic, or viral infection which requires therapy. Note: A subject meeting this criterion should delay screening and/or enrolment for a minimum of 2 weeks, or if excluded can be re-screened at maximum 2 times at a later time point.
- •Known history, and/or positive result on screening for hepatitis B surface antigen, hepatitis B virus, hepati
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