Phase 2
Completed N=60
Study of Oral SKI-O-703, SYK Inhibitor, in Patients With Persistent and Chronic Immune Thrombocytopenia (ITP)
Source: ClinicalTrials.gov NCT04056195 ↗Enrolled (actual)
60
Serious AEs
8.3%
Results posted
Jul 2024
Primary outcomePrimary: Platelet Response — 12; 14; 4 Participants
Summary
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Platelet Response |
12; 14; 4 | — |
| SECONDARY Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Discontinuation |
15; 17; 8; 0; 2; 3 | — |
| SECONDARY Number of Participants With Vital Sign Abnormalities |
1; 2; 0 | — |
| SECONDARY Number of Participants With 12-lead Electrocardiogram (ECG) Abnormalities |
0; 0; 0 | — |
| SECONDARY Number of Participants With Physical Examination Abnormalities |
4; 4; 3 | — |
| SECONDARY Quality of Life Score |
0.772; 0.556; 1.338; 4.913; -0.930; 1.643 | — |
| SECONDARY Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 30,000/µL) |
10; 11; 1 | — |
| SECONDARY Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 50,000/µL) |
5; 9; 1 | — |
Eligibility Criteria
Inclusion Criteria
- Diagnosis of primary ITP (persistent or chronic)
- Failed to respond or relapsed after at least 1 prior therapy, with a platelet count of 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.
Data sourced from ClinicalTrials.gov (NCT04056195). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.