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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
61
The SKI-O-703 capsules will contain 100 mg of drug substance.
Placebo capsules are filled with microcrystalline cellulose.
Platelet Response
Platelet count \>= 30,000/µL and doubling the baseline (average of 2 previous counts)
Time frame: Up to week 12
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Discontinuation
The number of participants with Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs leading to Discontinuation each.
Time frame: Up to week 16
Number of Participants With Vital Sign Abnormalities
Vital sign measurements considered to be clinically significant in the medical and scientific judgement of the investigator are recorded as AEs.
Time frame: Up to week 16
Number of Participants With 12-lead Electrocardiogram (ECG) Abnormalities
12-lead electrocardiogram (ECG) abnormalities that were recorded as adverse events
Time frame: Up to week 16
Number of Participants With Physical Examination Abnormalities
Physical examination abnormalities that were recorded as adverse events
Time frame: Up to week 16
Quality of Life Score
Qualtiy of Life as measured by the Short Form Questionnaire (SF-36) consists of eight health domains: physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role-emotional and mental health. Scale scores range 0-100 scores (theoritically), with higher scores indicating better health. Each health domain score contributes to the Physical Component Summary(PCS) and Mental Component Summary(MCS) scores. Both PCS and MCS are summary scores that are calculated using associated factor weights for the respective summary score applied to all eight scales. For overall ranges for PCS and MCS (no theoretical full range available), the SF-36 verion 2 utilizes norm-based scoring involving a linear T-score transformation method so that scores for each of the health domain and component summary measures have a mean of 50 and a standard deviation of 10, based on 2009 U.S. general population. Scores above and below 50 are above and velow the average.
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University of Southern California, 1441 Eastlake Ave.
Los Angeles, California, United States
Duke University Medical Center, 2301 Erwin Road
Durham, North Carolina, United States
East Carolina University, 600 Moye Boulevard
Greenville, North Carolina, United States
The Cleveland Clinic Foundation, 9500 Euclid Avenue
Cleveland, Ohio, United States
University General Hospital of Patras, Department of Internal Medicine, Hematology Division, Rio Patra
Pátrai, Achaia, Greece
Laiko General Hsoptial of Athens, 16 Sevastoupoleos Street
Athens, Attica, Greece
University Hospital of Larissa, Mezourlo
Larissa, Greece
AHEPA University General Hospital of Thessaloniki, Kyriakidi Stilponos 1
Thessaloniki, Greece
Hippokration Hospital, Konstantinoupoleos 49
Thessaloniki, Greece
Georgios Papanikolaou General Hospital of Thessaloniki, Exohi
Thessaloniki, Greece
...and 17 more locations
Time frame: Up to week 16
Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 30,000/µL)
Proportion of participants achieving two or more consecutive platelet counts of ≥ 30,000/μL separated by at least 5 days and without the use of rescue medication
Time frame: Up to week 12
Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 50,000/µL)
Proportion of participants achieving two or more consecutive platelet counts of ≥ 50,000/μL separated by at least 5 days and without the use of rescue medication
Time frame: Up to week 12