A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Dose Study to Evaluate the Efficacy and Safety of Oral SKI-O-703, SYK Inhibitor, in Patients With Persistent and Chronic Immune Thrombocytopenia (ITP)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Oscotec Inc.
- 入组人数
- 61
- 试验地点
- 27
- 主要终点
- Platelet Response
研究概览
简要总结
Study in patients with persistent and chronic Immune Thrombocytopenia (ITP), who have failed to respond or relapsed after prior therapy, with a platelet count <30,000/µL. Patient will be randomly assigned in 2 groups with two dose levels of SKI-O-703 200mg BID, 400 mg BID, and placebo; administered orally twice a day.
详细描述
This study will evaluate the efficacy, safety, tolerability,pharmacokinetics (PK), and pharmacodynamics (PD) of select (200 mg BID and 400 mg BID) doses of SKI-O-703 in persistent and chronic ITP patients who have failed to respond or relapsed after prior therapy, with a platelet count <30,000/µL. on 2 occasions at least 7 days apart with the confirmatory count on the first day of treatment.
subjects will participate in 3 treatment groups (24 subjects in each of the active treatment groups and 12 subjects in the placebo group). The total study duration will be 20 weeks per subject, which consists of up to 4 weeks of screening period, 12 weeks of treatment period, and 4 weeks of follow-up period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of primary ITP (persistent or chronic)
- •Failed to respond or relapsed after at least 1 prior therapy, with a platelet count of <30,000/µL on 2 occasions at least 7 days apart with the confirmatory count on the first day of treatment
- •Adequate hematologic, hepatic, and renal function
- •ECOG performance status of 0, 1, or 2
- •Male and female subjects, the subject and their partners of childbearing potential agree to use medically acceptable methods of contraception during the study and for 6 months following discontinuation of study drug (excluding women who are not of childbearing potential and men who have been sterilized. Men who have been sterilized should be confirmed to have negative sperm count on 2 consecutive occasions.)
- •Male subjects agree not to donate sperm for 90 days after the last dose of study drug
- •Female subjects have negative pregnancy tests at Screening.
排除标准
- •History of current, active malignancy requiring or likely to require chemotherapeutic or surgical treatment during the study, with the exception of non-melanoma skin cancer, carcinoma in situ of the cervix, and localized prostate cancer managed by active surveillance
- •Transfusion with blood or blood products or plasmapheresis within 2 weeks before the first administration of study drug
- •History of known inherited coagulopathy, or recent arterial or deep venous thrombosis within the preceding 6 months
- •Change in corticosteroid or immunosuppressant dose within 2 weeks prior to Day 1
- •Treatment with thrombopoietin receptor agonists within 2 weeks before Day 1
- •Treatment with rituximab or splenectomy within the 8 weeks prior to Day 1
- •Treatment with intravenous immunoglobulins (IVIGs) within 4 weeks prior to Day 1
- •Acute infection requiring oral antibiotics within 2 weeks
- •Infections requiring intravenous antibiotics or hospitalization within 3 months
- •Positive test results at Screening for human immunodeficiency virus, hepatitis B surface antigen, or hepatitis C virus antibody or positive result for hepatitis B core antibody with a negative result for hepatitis B surface antigen
- •Received live vaccine within 28 days prior to Day 1 or plan to receive one during the study
- •History or presence of any gastrointestinal, hepatic, or renal disease or any other condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs
- •Uncontrolled hypertension
- •Subject had 12-lead electrocardiogram (ECG) findings of corrected QT interval by Fridericia formula (QTcF) > 450 msec (males) or > 470 msec (females), cardiac arrhythmias, or clinically significant cardiac or ECG abnormalities
- •Subject received any investigational medication within 30 days or 5 half-lives - Concomitant use of any anticoagulants and platelet aggregation inhibiting drugs including aspirin (within 14 days of planned dosing through end of follow-up)
- •Female subject who is currently pregnant or breastfeeding
- •Prior treatment with a SYK inhibitor
- •Planned surgery in the time frame of the dosing period.
研究组 & 干预措施
SKI-O-703 200 mg
2 capsules of 100 mg SKI-O-703 BID (twice a day) 12 hours apart + 2 capsules of placebo during 12 weeks
干预措施: SKI-O-703 (Drug)
SKI-O-703 200 mg
2 capsules of 100 mg SKI-O-703 BID (twice a day) 12 hours apart + 2 capsules of placebo during 12 weeks
干预措施: Placebo oral tablet (Drug)
SKI-O-703 400 mg
4 capsules of 100 mg SKI-O-703 + 0 capsules of placebo during 12 weeks
干预措施: SKI-O-703 (Drug)
Placebo
4 capsules of placebo during 12 weeks
干预措施: Placebo oral tablet (Drug)
结局指标
主要结局
Platelet Response
时间窗: Up to week 12
Platelet count \>= 30,000/µL and doubling the baseline (average of 2 previous counts)
次要结局
- Number of Participants With Vital Sign Abnormalities(Up to week 16)
- Number of Participants With 12-lead Electrocardiogram (ECG) Abnormalities(Up to week 16)
- Number of Participants With Physical Examination Abnormalities(Up to week 16)
- Quality of Life Score(Up to week 16)
- Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 30,000/µL)(Up to week 12)
- Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 50,000/µL)(Up to week 12)
- Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Discontinuation(Up to week 16)