Study of Oral SKI-O-703, SYK Inhibitor, in Patients With Persistent and Chronic Immune Thrombocytopenia (ITP)

Immune Thrombocytopenia Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04056195
• Other study ID #: OSCO-P2101
• Status: Completed
• Phase: Phase 2
• Start date: October 11, 2019
• Est. completion date: January 10, 2023

Study information

• Verified date: July 2023
• Source: Oscotec Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 61
• Est. completion date: January 10, 2023
• Est. primary completion date: January 10, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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 <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.

Exclusion Criteria:

• 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.

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• SKI-O-703
The SKI-O-703 capsules will contain 100 mg of drug substance.
• Placebo oral tablet
Placebo capsules are filled with microcrystalline cellulose.

Locations

Country	Name	City	State
Greece	Laiko General Hsoptial of Athens, 16 Sevastoupoleos Street	Athens	Attiki
Greece	University Hospital of Larissa, Mezourlo	Larissa
Greece	University General Hospital of Patras, Department of Internal Medicine, Hematology Division, Rio Patra	Patras	Achaia
Greece	AHEPA University General Hospital of Thessaloniki, Kyriakidi Stilponos 1	Thessaloníki
Greece	Georgios Papanikolaou General Hospital of Thessaloniki, Exohi	Thessaloníki
Greece	Hippokration Hospital, Konstantinoupoleos 49	Thessaloníki
Korea, Republic of	Asan Medical Center - PPDS, 88 Olympic-ro 43-gil, Songpa-gu	Seoul
Korea, Republic of	Samsung Medical Center PPDS, 81 Irwon-dong Gangnam-gu	Seoul
Korea, Republic of	Seoul National University Hospital, 101 Daehak-ro, Jongno-gu	Soeul
Korea, Republic of	Severance Hospital Yonsei University Health System, 50-1 Yonsei-Ro, Seodaemun-Gu	Soeul
Korea, Republic of	Ajou University Hospital, 164 World Cup-ro, Yeongtong-gu	Suwon-si	Gyeonggido
Poland	Szpital Uniwersytecki Nr 2 im. Dr Jana Biziela w Bydgoszczy, Ujejskiego 75	Bydgoszcz
Poland	Copernicus PL Sp. z o.o. Wojewodzkie Centrum Onkologii, Aleja Zwyciestwa 31/32	Gdansk
Poland	Uniwersyteckie Centrum Kliniczne, Klinika Hematologii i Transplantologii, Smoluchowskiego 17	Gdansk
Poland	EMC Instytut Medyczny S.A Przychondnia przy Lowieckiej, Lowiecka	Wroclaw
Spain	Hospital Universitario 12 de Octubre, Avenida de Cordoba, s/n	Madrid
Spain	Hospital Universitario La Paz, Paseo Castellana 261	Madrid
Spain	Hospital Universitario Ramon y Cajal, Carretera de Colmenar Viejo Km. 9100	Madrid
Spain	Hosptial Regional Universitario de Malaga - Hospital General, Avenida Carlos Haya, s/n	Málaga
Spain	Hospital Universitario Quironsalud Madrid, Calle Diego De Velazquez 1	Pozuelo De Alarcón	Madrid
Spain	Complejo Asistencial Universitario de Salamanca - H. Clinico, Paseo de San Vincent, 58	Salamanca
Spain	Hospital Universitario Virgen del Rocio, Avenida Manuel Siurot, Centro de Diagnostico y Tratamiento	Sevilla
Spain	Hospital Universitari i Politecnic La Fe de Valencia, Avda Fernando Abril Martorell no° 106	Valencia
United States	The Cleveland Clinic Foundation, 9500 Euclid Avenue	Cleveland	Ohio
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	University of Southern California, 1441 Eastlake Ave.	Los Angeles	California

Sponsors (1)

Lead Sponsor	Collaborator
Oscotec Inc.

Countries where clinical trial is conducted

United States,  Greece,  Korea, Republic of,  Poland,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Platelet Response	Platelet count >= 30,000/µL and doubling the baseline (average of 2 previous counts)	Up to week 12
Secondary	Number of participants with Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs leading to Discontinuation		Up to week 16
Secondary	Number of participants with vital sign abnormalities		Up to week 16
Secondary	Number of participants with 12-lead electrocardiogram (ECG) abnormalities		Up to week 16
Secondary	Number of participants with physical examination abnormalities		Up to week 16
Secondary	Number of participants with clinical laboratory abnormalities		Up to week 16
Secondary	Bleeding score	Measured by Immune thrombocytopenic purpura (ITP) bleeding score (Br. J. Haematol 2007;138(2): 245-8)	Up to week 16
Secondary	Quality of Life score	Measured by the SF-36 score	Up to week 16

External Links

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