Efficacy and safety of oral SKI-O-703 in persistent and chronic immune thrombocytopenia
- Conditions
- Persistent and Chronic Immune Thrombocytopenia (ITP)MedDRA version: 22.1Level: LLTClassification code 10066667Term: Chronic thrombocytopeniaSystem Organ Class: 100000004851Therapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- EUCTR2018-003329-26-PL
- Lead Sponsor
- Oscotec Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 60
1. Able to provide written informed consent and agreeable to the schedule of assessments
2. Male or female subjects, aged 18 years or older
3. Diagnosis of primary ITP (persistent or chronic) made according to the American Society of Hematology 2011 evidence-based guideline
4. 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. Response to such prior therapy is defined as platelet count of =30,000/µL and >2 times increase from baseline in the absence of bleeding. Failure to respond and relapse to such prior therapy are defined as failure to achieve and/or failure to maintain response on a tolerable regiment of prior therapy.
5. Adequate hematologic, hepatic, and renal function (absolute neutrophil count = 1.5 X 109/L, hemoglobin [Hgb] = 10.0 g/dL, aspartate aminotransferase [AST] and alanine aminotransferase [ALT] = 2.0 x ULN, bilirubin <1.5 x ULN, albumin = 3 g/dL, estimated glomerular filtration rate [eGFR] =40 mL/min/1.73m2, and creatinine = 1.5 x ULN)
6. ECOG performance status of 0, 1, or 2
7. Both male and female subjects must agree to take the following steps to reduce the potential for transmission of genetic material containing the investigational product
a. Both male and female subjects, the subject and their partners of
childbearing potential must agree to use 2 of the following medically acceptable methods of contraception from the time of randomization, during the study, and for 6 months following discontinuation of study drug, of which:
o One must be a highly reliable method of contraception, such as:
? An intrauterine device or intrauterine system implanted for at least 30 days prior to Day 1
? Surgical sterilization of one of the partners for at least 6 months prior to the date of informed consent (assuming this will be the only partner for the whole duration of the clinical trial)
? Consistent and correct use of hormonal contraceptives (hormonal implants, injectables, contraception pills, transdermal patches, or contraceptive rings) for at least 30 days prior to Day 1
o One supplementary barrier method, such as:
? Male or female condom always with spermicide (a spermicidal
foam/gel/film/cream)
? Diaphragm or cervical/vault caps always with spermicide (a
spermicidal foam/gel/film/cream)
? Double-barrier methods (which means a barrier method used by both partners at the same time), even when used with spermicide, are not considered to be highly reliable contraception methods, and as such, may not be the only forms of contraception used
One of the other listed highly reliable methods must be used in
conjunction with a barrier method.
b. Female subjects must agree not to breastfeed starting from the time of Screening, throughout the study, and until after 6 months following the last dose of study drug.
c. Male subjects must agree not to donate sperm starting from the time of randomization, throughout the study, and until after 6 months following the last dose of study drug
d. For subjects and partners considered not of childbearing potential, the following conditions apply:
a. Menopausal females must have experienced their last period more than 12 months prior to the date of informed consent to be classified as not of childbearing potential.
b. Male and female subjects are in a situation of abstinence from
heterosexual intercourse from Screening until after 6 months following the las
1. History of any clinically significant disease or disorder that in the opinion of the investigator or the sponsor may put the subject at risk due to study participation, impact the subject’s ability to participate in the study, or influence the study results
2. 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
3. Transfusion with blood or blood products or plasmapheresis within 2 weeks before the first administration of study drug (Day 1)
4. PT INR > 1.5
5. History of known inherited coagulopathy (including prothrombotic conditions such as Factor V Leiden, APC resistance, AT-III deficiency, and lupus anticoagulant), or recent arterial or deep venous thrombosis within the preceding 6 months
6. Change in corticosteroid or immunosuppressant (azathioprine, mycophenolate mofetil, cyclosporine) dose within 2 weeks prior to Day 1 (more than 10% variation from Day 1 daily doses)
7. Treatment with thrombopoietin (TPO) receptor agonists within 2 weeks before Day 1
8. Treatment with rituximab or splenectomy within the 8 weeks prior to Day 1
9. Treatment with IVIGs within 4 weeks prior to Day 1
10. Infections requiring intravenous antibiotics or hospitalization within 3 months prior to Day 1
11. Subject had positive test results at Screening for human
immunodeficiency virus, hepatitis B surface antigen (HBsAg), or
hepatitis C virus antibody Subjects with past or resolved hepatitis B
infection (defined as having a negative HBsAg test and a positive IgG
antibody to hepatitis B core antigen [anti-HBc]) are eligible but hepatitis B virus (HBV) DNA must be obtained in these patients prior to Cycle 1, Day 1, and must demonstrate no active infection.
Subjects positive for hepatitis C virus (HCV) antibody are eligible only if polymerase chain reaction (PCR) is negative for HCV RNA
12. Live vaccine within 28 days prior to Day 1 or plan to receive one during the study
13. 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
14. Subject has not recovered from a recent medical/surgical procedure or trauma by Day 1 as determined by the investigator
15. Uncontrolled hypertension (as defined by systolic blood pressure =160 mmHg or diastolic blood pressure =100 mmHg)
16. Subject had ECG findings of corrected QT interval by Fridericia formula (QTcF) > 480 msec, cardiac arrhythmias, or clinically significant cardiac or ECG abnormalities
17. Subject have received any investigational medication within 30 days or 5 half lives prior to Day 1, whichever was longer
18. Concomitant use of any anticoagulants and platelet aggregation inhibiting drugs including aspirin (within 14 days of planned dosing through end of follow-up)
19. Female subject who is currently pregnant or breastfeeding
20. Prior treatment with a SYK inhibitor
21. Planned surgery in the time frame of the dosing period.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method