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

NCT ID: NCT04056195

Last Updated: 2024-07-10

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 61 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-10-11
• Study Completion Date: 2023-01-10

Brief 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.

Detailed 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.

Conditions

Immune Thrombocytopenia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

SKI-O-703 200 mg

2 capsules of 100 mg SKI-O-703 BID (twice a day) 12 hours apart + 2 capsules of placebo during 12 weeks

Group Type: EXPERIMENTAL

SKI-O-703

Intervention Type: DRUG

The SKI-O-703 capsules will contain 100 mg of drug substance.

Placebo oral tablet

Intervention Type: DRUG

Placebo capsules are filled with microcrystalline cellulose.

SKI-O-703 400 mg

4 capsules of 100 mg SKI-O-703 + 0 capsules of placebo during 12 weeks

Group Type: EXPERIMENTAL

SKI-O-703

Intervention Type: DRUG

The SKI-O-703 capsules will contain 100 mg of drug substance.

Placebo

4 capsules of placebo during 12 weeks

Group Type: PLACEBO_COMPARATOR

Placebo oral tablet

Intervention Type: DRUG

Placebo capsules are filled with microcrystalline cellulose.

Interventions

SKI-O-703

The SKI-O-703 capsules will contain 100 mg of drug substance.

Intervention Type: DRUG

Placebo oral tablet

Placebo capsules are filled with microcrystalline cellulose.

Intervention Type: DRUG

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 <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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Oscotec Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

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

Szpital Uniwersytecki Nr 2 im. Dr Jana Biziela w Bydgoszczy, Ujejskiego 75

Bydgoszcz, , Poland

Uniwersyteckie Centrum Kliniczne, Klinika Hematologii i Transplantologii, Smoluchowskiego 17

Gdansk, , Poland

Copernicus PL Sp. z o.o. Wojewodzkie Centrum Onkologii, Aleja Zwyciestwa 31/32

Gdansk, , Poland

EMC Instytut Medyczny S.A Przychondnia przy Łowieckiej, Łowiecka

Wroclaw, , Poland

Ajou University Hospital, 164 World Cup-ro, Yeongtong-gu

Suwon, Gyeonggido, South Korea

Asan Medical Center - PPDS, 88 Olympic-ro 43-gil, Songpa-gu

Seoul, , South Korea

Samsung Medical Center PPDS, 81 Irwon-dong Gangnam-gu

Seoul, , South Korea

Seoul National University Hospital, 101 Daehak-ro, Jongno-gu

Soeul, , South Korea

Severance Hospital Yonsei University Health System, 50-1 Yonsei-Ro, Seodaemun-Gu

Soeul, , South Korea

Hospital Universitario Quironsalud Madrid, Calle Diego De Velazquez 1

Pozuelo de Alarcón, Madrid, Spain

Hospital Universitario Ramon y Cajal, Carretera de Colmenar Viejo Km. 9100

Madrid, , Spain

Hospital Universitario 12 de Octubre, Avenida de Cordoba, s/n

Madrid, , Spain

Hospital Universitario La Paz, Paseo Castellana 261

Madrid, , Spain

Hosptial Regional Universitario de Malaga - Hospital General, Avenida Carlos Haya, s/n

Málaga, , 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

Seville, , Spain

Hospital Universitari i Politecnic La Fe de Valencia, Avda Fernando Abril Martorell no° 106

Valencia, , Spain

Countries

United States; Greece; Poland; South Korea; Spain

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

http://www.fda.gov/Safety/Recalls/

Related Info

Other Identifiers

OSCO-P2101

Identifier Type: -

Identifier Source: org_study_id