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This is a multicenter, open-label Phase1/2 study aimed at evaluating the safety, tolerability, pharmacokinetic (PK) profile, pharmacodynamics (PD), immunogenicity, and preliminary efficacy of ESG206. The study will be conducted in patients with primary immune thrombocytopenia.
Phase 1 is a dose escalation study, and Phase 2 is an extended cohort study, subjects will be treated with ESG206.
In Phase 1&2, the study includes Screening (no more than 28 days), Treatment (14 weeks), and the post-treatment follow-up (8 weeks), for those sufferring a treatment failure, the safe follow-up will be conducted 28 days after the last dose.
And the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and preliminary efficacy of ESG206 in primary ITP will be assessed in this study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ESG206 dose level 1 | Experimental | ESG206 will be administered intravenously at dose level 1. |
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| ESG206 dose level 2 | Experimental | ESG206 will be administered intravenously at dose level 2. |
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| ESG206 dose level 3 | Experimental | ESG206 will be administered intravenously at dose level 3. |
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| ESG206 dose level 4 | Experimental | ESG206 will be administered intravenously at dose level 4. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ESG206 | Drug | Administered via intravenous (IV) infusion |
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| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Experiencing Any Treatment Emergent Adverse Events and Serious Treatment Emergent Adverse Events | Treatment-emergent adverse events (TEAEs) were defined as: Any adverse event (AE) that happens after treatment initiation, or AE that was present at time of treatment initiation but worsened after treatment initiation, or AE that was present and resolved prior to treatment and reappeared after treatment initiation after the start of study drug through 28 days after the last dose of study drug or the last post-treatment follow-up. The severity was graded based on the National Cancer Institute's Common Terminology Criteria for Adverse Events. | Up to 24 weeks. |
| Confirmed response rate | Confirmed response is defined as a platelet count of equal or above 50 G/L at two (or more) consecutive assessments at least 7 days apart, in the absence of: Rescue treatment for ≥4 weeks prior to the assessment of the platelet count, and New immune thrombocytopenia (ITP) treatment before reaching a confirmed response. | Between Week 1 Day 1 and Week 25 Day 1 |
| Measure | Description | Time Frame |
|---|---|---|
| AUC0-inf | Area under the serum concentration time curve (AUC) from time 0 extrapolated to infinity | up to Week 25 |
| Cmax | Maximum observed plasma concentration |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Xiaoyan Xing, PhD | Contact | 86 21 5855 6098 | xingxiaoyan@escugen.com | |
| Ming Hou, PhD | Contact | 86 531 82169867 |
| Name | Affiliation | Role |
|---|---|---|
| Ming Hou, PhD | Qilu Hospital of Shandong University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Qilu Hospital of Shandong University | Recruiting | Jinan | Shandong | 250012 | China |
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| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
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| ESG206 | Drug | Administered via intravenous (IV) infusion |
|
| ESG206 | Drug | Administered via intravenous (IV) infusion |
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| ESG206 | Drug | Administered via intravenous (IV) infusion |
|
| up to Week 25 |
| Tmax | Time to maximum plasma concentration | up to Week 25 |
| T1/2 | Half-life | up to Week 25 |
| Response rate at each timepoint | Percentage of participants with a platelet count of at least 50 G/L in the absence of rescue treatment/new ITP treatment. | Between Week 1 Day 1 and Week 25 Day 1 |
| Complete Response rate at each timepoint | Percentage of participants with a platelet count of at least 100 G/L in the absence of rescue treatment/new ITP treatment. | Between Week 1 Day 1 and Week 25 Day 1 |
| Best Response rate at each timepoint | Percentage of participants with Response or Complete Response in the absence of rescue treatment/new ITP treatment. | Between Week 1 Day 1 and Week 25 Day 1 |
| Time to confirmed response | Time from the first administration of ESG206 to the first assessment in the first sequence of two (or more) platelet assessments meeting the criteria of a confirmed response as defined by the primary endpoint. | Between Week 1 Day 1 and Week 25 Day 1 |
| Time to complete response | Time from the first administration of ESG206 to the first assessment meeting the criteria of a complete response. | Between Week 1 Day 1 and Week 25 Day 1 |
| Time to treatment failure | Time from the first administration until platelet count below 30 G/L, need for a rescue treatment or start of a new therapy or death. | Between Week 1 Day 1 and Week 25 Day 1 |
| Duration of response | Time from achievement of response to loss of response | Between Week 1 Day 1 and Week 25 Day 1 |
| Duration of complete response | Time from achievement of complete response to loss of complete response. | Between Week 1 Day 1 and Week 25 Day 1 |
| Percentage of participants receiving rescue treatment | Assess the need of rescue treatment by percentage. | Between Week 1 Day 1 and Week 25 Day 1 |
| Change from baseline in absolute number of CD19+ B cell counts | Post baseline absolute number of CD19+ B cell counts compare with baseline | Between Week 1 Day 1 and Week 25 Day 1 |
| Change from baseline in absolute number of CD20+ B cell counts | Post baseline absolute number of CD20+ B cell counts compare with baseline | Between Week 1 Day 1 and Week 25 Day 1 |
| Incidence of anti-drug antibodies in serum (ADA assay) over time | Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants assess the immunogenicity of ESG206 | Between Week 1 Day 1 and Week 25 Day 1 |
| D006425 |
| Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |