None listed
Conditions
Interventions
Drug : 1. SKI-O-703 treatment group
- Drug: SKI-O-703 (SKI-O-703: 100 mg)
- Frequency: 200 mg (twice daily after meals) or, if dose-escalated, 400 mg (twice daily after meals)
- Duration: 24 weeks
- R
Sponsors
The Catholic University of Korea, Seoul St. Mary's Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Male or female adults aged 19 years or older 2) Diagnosed with primary ITP within 3 months prior to the screening visit - Initiated first-line standard therapya with a platelet count of less than 30,000/µL at maximum and showed a response during the screening period (platelet count = 50,000/µL in at least 2 tests conducted at 1-week [±3 days] intervals during the screening period) a. First-line standard therapy: For up to 8 weeks, patients are typically treated as follows, either alone or in combination at the discretion of the investigator, and the dose may be adjusted. - Dexamethasone: 20 or 40 mg/day for 4 consecutive days; re-treatment is possible after 3 to 4 weeks if the response is insufficient (total of 2 times) - Prednisone: 0.5-2.0 mg/kg/day for 2 weeks; if a response is confirmed, the dose will be reduced by 50% at 2-week intervals to complete tapering within 8 weeks - IVIG: 1 g/kg/day for 1-2 days; re-treatment is possible after 3 to 4 weeks if the response is insufficient (total of 2 times) - Anti-D: 125-300 IU/kg/day for 1-2 days; re-treatment is possible after 3 to 4 weeks if the response is insufficient (total of 2 times) 3) Have been fully informed about this study, have voluntarily agreed to participate, and have signed a written informed consent form
Exclusion criteria
Exclusion criteria: 1) Hypersensitivity to the active ingredient or any excipients of the investigational product (IP), or contraindications to administration of the IP 2) Diagnosed with secondary ITP 3) Severe ITPb b. Severe bleeding symptoms or requiring platelet enhancing agents or additional therapeutic interventions other than first-line standard therapy and the IP 4) Diagnosed with arterial thrombosis or deep vein thrombosis within 24 weeks prior to study participation 5) PT INR > 1.5 6) Severe renal or hepatic impairment 7) Positive test results at screening visit for hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (HCV Ab), or human immunodeficiency virus (HIV) 8) Uncontrolled hypertension (as defined by systolic blood pressure (SBP] = 160 mmHg or diastolic blood pressure (DBP) = 100 mmHg) 9) Any of the following cardiac function abnormalities detected on at least 2 consecutive occasions prior to study participation ? QT interval cannot be measured on electrocardiogram (ECG) ? QTcF > 450 msec 10) History of clinically proven myocardial infarction or other clinically significant heart disease 11) Have had a malignancy within 5 years prior to screening [However, those who have achieved a complete response (CR) after treatment with no recurrence for at least 2 years prior to screening, or in the case of basal cell carcinoma, squamous cell carcinoma of the skin, thyroid cancer, or carcinoma in-situ of other sites, those with a medical history of malignancy within 5 years who have been successfully treated and have had no recurrence for at least 3 years are eligible according to the investigator’s medical judgement] 12) Received a blood transfusion within 2 weeks prior to the screening visit (However, they may be enrolled at the discretion of the investigator). 13) Have taken anticoagulants, including aspirin, within 2 weeks prior to the baseline visit (Visit 2), or require continuous use of anticoagulants during the study period, or are at high risk of bleeding 14) Evidence of active infection 15) Received a vaccine within 28 days prior to the screening visit or are scheduled to receive a vaccine during the study period 16) Received treatment other than first-line standard therapya within 3 months prior to the screening visit [e.g., thrombopoietin receptor agonists (TPO-RA), splenectomy, SYK inhibitors, etc.] a. First-line standard therapy: See inclusion criteria #2 17) Currently pregnant or breastfeeding 18) Women of childbearing potential or men who do not agree to maintain abstinence or to use adequate contraceptionc for the duration of this study and for 30 days after the last dose of IP c. ? Hormonal contraceptives (subdermal implants, injectables, oral contraceptives, etc.), ? implantation of an intrauterine device or intrauterine system, ? double barrier methods (condoms and contraceptive diaphragm, vaginal sponge, or cervical cap) in men or women of childbearing potential, ? sterilization (vasectomy, bilateral tubal ligation, etc.) 19) History of severe neurologic disease, psychiatric disease, drug abuse, or alcoholism that would affect participation in this study. 20) Received another investigational product or used an investigational device within 30 days prior to the screening visit (however, if 5 half-lives of the previously administered/applied medical product exceed 30 days, that period will apply) 21) Ineligible for participation in this study according to the investigator’s judgement
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Relapse-free rate within 48 weeks of IP administration | — |
Secondary
| Measure | Time frame |
|---|---|
| Relapse-free rate within 24 weeks of IP administration;elapse-free duration after IP administration;Rate of CR after IP administration ;Duration of CR after IP administration;Stable response rate up to 24 weeks after IP administration;Stable response rate from 24 to 48 weeks after IP administration;Rate of rescue therapy use after IP administration | — |
Countries
Korea, Republic of
Contacts
Public ContactYoungbin Kim
The Catholic University of Korea, Seoul St. Mary's Hospital
Outcome results
None listed