Immune ThrombocytopeniaPhase 2NCT04056195
Study of Oral SKI-O-703, SYK Inhibitor, in Patients With Persistent and Chronic Immune Thrombocytopenia (ITP)
A Phase 2 study testing SKI-O-703 in people with Immune Thrombocytopenia, run by Oscotec Inc. at 27 sites in 5 countries.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- SKI-O-703
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 61 participants
- Where
- 27 sitesin 5 countries
- Study dates
- 11 Oct 2019to 10 Jan 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 18Show fewer
- 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.
Trial locations and contacts
27 sites in 5 countries.
27 sites in 5 countries
Spain8 sites
Hospital Universitario Ramon y Cajal, Carretera de Colmenar Viejo Km. 9100
Madrid, 28034
Hospital Universitario 12 de Octubre, Avenida de Cordoba, s/n
Madrid, 28041
Hospital Universitario La Paz, Paseo Castellana 261
Madrid, 28046
Hosptial Regional Universitario de Malaga - Hospital General, Avenida Carlos Haya, s/n
Málaga, 29010
Hospital Universitario Quironsalud Madrid, Calle Diego De Velazquez 1
Pozuelo de Alarcón, Madrid, 28223
Complejo Asistencial Universitario de Salamanca - H. Clinico, Paseo de San Vincent, 58
Salamanca, 37007
Hospital Universitario Virgen del Rocio, Avenida Manuel Siurot, Centro de Diagnostico y Tratamiento
Seville, 41013
Hospital Universitari i Politecnic La Fe de Valencia, Avda Fernando Abril Martorell no° 106
Valencia, 46026
Greece6 sites
Laiko General Hsoptial of Athens, 16 Sevastoupoleos Street
Athens, Attica, 11526
University Hospital of Larissa, Mezourlo
Larissa, 41110
University General Hospital of Patras, Department of Internal Medicine, Hematology Division, Rio Patra
Pátrai, Achaia, 26500
AHEPA University General Hospital of Thessaloniki, Kyriakidi Stilponos 1
Thessaloniki, 54636
Hippokration Hospital, Konstantinoupoleos 49
Thessaloniki, 54642
Georgios Papanikolaou General Hospital of Thessaloniki, Exohi
Thessaloniki, 57010
South Korea5 sites
Asan Medical Center - PPDS, 88 Olympic-ro 43-gil, Songpa-gu
Seoul, 5505
Samsung Medical Center PPDS, 81 Irwon-dong Gangnam-gu
Seoul, 6351
Seoul National University Hospital, 101 Daehak-ro, Jongno-gu
Soeul, 3080
Severance Hospital Yonsei University Health System, 50-1 Yonsei-Ro, Seodaemun-Gu
Soeul, 3722
Ajou University Hospital, 164 World Cup-ro, Yeongtong-gu
Suwon, Gyeonggido, 16499
United States4 sites
The Cleveland Clinic Foundation, 9500 Euclid Avenue
Cleveland, Ohio, 44195
Duke University Medical Center, 2301 Erwin Road
Durham, North Carolina, 22705
East Carolina University, 600 Moye Boulevard
Greenville, North Carolina, 27834
University of Southern California, 1441 Eastlake Ave.
Los Angeles, California, 90033
Poland4 sites
Szpital Uniwersytecki Nr 2 im. Dr Jana Biziela w Bydgoszczy, Ujejskiego 75
Bydgoszcz, 85-168
Uniwersyteckie Centrum Kliniczne, Klinika Hematologii i Transplantologii, Smoluchowskiego 17
Gdansk, 80-214
Copernicus PL Sp. z o.o. Wojewodzkie Centrum Onkologii, Aleja Zwyciestwa 31/32
Gdansk, 80-219
EMC Instytut Medyczny S.A Przychondnia przy Łowieckiej, Łowiecka
Wroclaw
About this study
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)
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.
Read the 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
- Being tested
- SKI-O-703, Placebo oral tablet
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SKI-O-703Drug
The SKI-O-703 capsules will contain 100 mg of drug substance.
Placebo oral tabletDrug
Placebo capsules are filled with microcrystalline cellulose.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: Drug: SKI-O-703 |
| Placebo | Placebo comparator | 4 capsules of placebo during 12 weeks Receives: Drug: Placebo oral tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Platelet Response
Platelet count >= 30,000/µL and doubling the baseline (average of 2 previous counts)
Measured: Up to week 12
Secondary outcomes (7)
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.
Measured: 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.
Measured: Up to week 16
Number of Participants With 12-lead Electrocardiogram (ECG) Abnormalities
12-lead electrocardiogram (ECG) abnormalities that were recorded as adverse events
Measured: Up to week 16
Number of Participants With Physical Examination Abnormalities
Physical examination abnormalities that were recorded as adverse events
Measured: 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.
Measured: 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
Measured: 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
Measured: Up to week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Aug 2019
- Study start11 Oct 2019
- Primary completion10 Jan 2023
- Study completion10 Jan 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 61 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oscotec Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04056195
- Sponsor's protocol ID
- OSCO-P2101
- Status last verified
- Jul 2023
- First submitted
- 12 Aug 2019
- Last updated
- 10 Jul 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04056195
- Acronym / sponsor ID
- OSCO-P2101
- Conditions
- Immune Thrombocytopenia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 61 participants (actual)
- Lead sponsor
- Oscotec Inc.Industry (pharma, biotech, devices)
- Study start
- 11 Oct 2019 (actual)
- Primary completion
- 10 Jan 2023 (actual)
- Study completion
- 10 Jan 2023 (actual)
- First posted
- 14 Aug 2019 (actual)
- Results
- Posted 10 Jul 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| SKI-O-703 200 mg | Experimental | Drug: SKI-O-703, Drug: Placebo oral tablet2 capsules of 100 mg SKI-O-703 BID (twice a day) 12 hours apart + 2 capsules of placebo during 12 weeks |
| SKI-O-703 400 mg | Experimental | Drug: SKI-O-7034 capsules of 100 mg SKI-O-703 + 0 capsules of placebo during 12 weeks |
| Placebo | Placebo comparator | Drug: Placebo oral tablet4 capsules of placebo during 12 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.)
Key exclusion criteria (18 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SKI-O-703The SKI-O-703 capsules will contain 100 mg of drug substance. | Drug | SKI-O-703 200 mg, SKI-O-703 400 mg |
| Placebo oral tabletPlacebo capsules are filled with microcrystalline cellulose. | Drug | Placebo, SKI-O-703 200 mg |
Primary outcome
Platelet Response
Time frame: Up to week 12
Platelet count >= 30,000/µL and doubling the baseline (average of 2 previous counts)
7 secondary outcomes
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Discontinuation
Time frame: Up to week 16
Number of Participants With Vital Sign Abnormalities
Time frame: Up to week 16
Number of Participants With 12-lead Electrocardiogram (ECG) Abnormalities
Time frame: Up to week 16
Number of Participants With Physical Examination Abnormalities
Time frame: Up to week 16
Quality of Life Score
Time frame: Up to week 16
Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 30,000/µL)
Time frame: Up to week 12
Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 50,000/µL)
Time frame: Up to week 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
27 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SKI-O-703 in people with Immune Thrombocytopenia, run by Oscotec Inc. at 27 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in Spain, Greece, South Korea, United States, Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Jul 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Platelet Response (time frame: Up to week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jul 2024.