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

NCT04056195 | Completed Phase 2 Results FDA Drug

• 1 other identifier: OSCO-P2101
• Study Type: interventional
• Target: 61
• Locations: 5 countries
• Sites: 27

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.

Conditions

Immune Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• Platelet Response

  Platelet count \>= 30,000/µL and doubling the baseline (average of 2 previous counts)

  Up to week 12

Secondary Outcomes (7)

• Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Discontinuation

  Up to week 16

• Number of Participants With Vital Sign Abnormalities

  Up to week 16

• Number of Participants With 12-lead Electrocardiogram (ECG) Abnormalities

  Up to week 16

• Number of Participants With Physical Examination Abnormalities

  Up to week 16

• Quality of Life Score

  Up to week 16

Study Arms (3)

SKI-O-703 200 mg

EXPERIMENTAL

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

Drug: SKI-O-703; Drug: Placebo oral tablet

SKI-O-703 400 mg

EXPERIMENTAL

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

Drug: SKI-O-703

Placebo

PLACEBO COMPARATOR

4 capsules of placebo during 12 weeks

Drug: Placebo oral tablet

Interventions

SKI-O-703 DRUG

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

SKI-O-703 200 mg; SKI-O-703 400 mg

Placebo oral tablet DRUG

Placebo capsules are filled with microcrystalline cellulose.

Placebo; SKI-O-703 200 mg

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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)

Sponsors & Collaborators

• Oscotec Inc.: lead

Study Sites (27)

University of Southern California, 1441 Eastlake Ave.

Los Angeles, California, 90033, United States

Location

Duke University Medical Center, 2301 Erwin Road

Durham, North Carolina, 22705, United States

Location

East Carolina University, 600 Moye Boulevard

Greenville, North Carolina, 27834, United States

Location

The Cleveland Clinic Foundation, 9500 Euclid Avenue

Cleveland, Ohio, 44195, United States

Location

University General Hospital of Patras, Department of Internal Medicine, Hematology Division, Rio Patra

Pátrai, Achaia, 26500, Greece

Location

Laiko General Hsoptial of Athens, 16 Sevastoupoleos Street

Athens, Attica, 11526, Greece

Location

University Hospital of Larissa, Mezourlo

Larissa, 41110, Greece

Location

AHEPA University General Hospital of Thessaloniki, Kyriakidi Stilponos 1

Thessaloniki, 54636, Greece

Location

Hippokration Hospital, Konstantinoupoleos 49

Thessaloniki, 54642, Greece

Location

Georgios Papanikolaou General Hospital of Thessaloniki, Exohi

Thessaloniki, 57010, Greece

Location

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

Bydgoszcz, 85-168, Poland

Location

Uniwersyteckie Centrum Kliniczne, Klinika Hematologii i Transplantologii, Smoluchowskiego 17

Gdansk, 80-214, Poland

Location

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

Gdansk, 80-219, Poland

Location

EMC Instytut Medyczny S.A Przychondnia przy Łowieckiej, Łowiecka

Wroclaw, Poland

Location

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

Suwon, Gyeonggido, 16499, South Korea

Location

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

Seoul, 5505, South Korea

Location

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

Seoul, 6351, South Korea

Location

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

Soeul, 3080, South Korea

Location

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

Soeul, 3722, South Korea

Location

Hospital Universitario Quironsalud Madrid, Calle Diego De Velazquez 1

Pozuelo de Alarcón, Madrid, 28223, Spain

Location

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

Madrid, 28034, Spain

Location

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

Madrid, 28041, Spain

Location

Hospital Universitario La Paz, Paseo Castellana 261

Madrid, 28046, Spain

Location

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

Málaga, 29010, Spain

Location

Complejo Asistencial Universitario de Salamanca - H. Clinico, Paseo de San Vincent, 58

Salamanca, 37007, Spain

Location

Hospital Universitario Virgen del Rocio, Avenida Manuel Siurot, Centro de Diagnostico y Tratamiento

Seville, 41013, Spain

Location

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

Valencia, 46026, Spain

Location

Related Links

• Related Info

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

cevidoplenib

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Results Point of Contact

• Title: Sungsil Lee/Team Leader of Clinical Development
• Organization: Oscotec Inc.

sslee@oscotec.com

+82316287624

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 12, 2019
• First Posted: August 14, 2019
• Study Start: October 11, 2019
• Primary Completion: January 10, 2023
• Study Completion: January 10, 2023
• Last Updated: July 10, 2024
• Results First Posted: July 10, 2024

Record last verified: 2023-07

Data Sharing

• IPD Sharing: Will not share

Locations

PL (4); US (4); KR (5); GR (6); ES (8)