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This is a multicenter clinical study to evaluate the safety and efficacy of golidocitinib in patients with primary immune thrombocytopenia (ITP). The study consists of two parts: Part A dose escalation and Part B dose expansion. Part A is designed to obtain the safety profile of golidocitinib in patients with ITP and the recommended dose for the randomized cohort in Part B. Part B is a randomized, double-blind, placebo-controlled study, and the primary objective of this part is to evaluate the preliminary efficacy of golidocitinib in patients with ITP.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Golidocitinib Part B | Experimental | Golidocitinib at recommended phase 2 doses, administered orally, once daily. |
|
| Placebo Part B | Placebo Comparator | Placebo at recommended phase 2 doses, administered orally, once daily. |
|
| Golidocitinib Part A | Experimental | Golidocitinib dose escalation, administered orally, once daily. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Golidocitinib | Drug | Golidocitinib will be administered orally as capsules in a 28-day cycle. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Incidence and severity of Adverse Events (AEs) | The severity was graded based on the National Cancer Institute's Common Terminology Criteria for Adverse Events. | 1 year |
| Part B: Durable response rate | The proportion of patients with a platelet count ≥ 50 × 10⁹/L on at least four of six scheduled visits between weeks 14 and 24 | up to 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Durable response rate | The proportion of patients with a platelet count ≥ 50 × 10⁹/L on at least four of six scheduled visits between weeks 14 and 24 | up to 24 weeks |
| Part B: Incidence and severity of Adverse Events (AEs) |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking University Insititute of Hematology, Peking University People's Hospital | Beijing | Beijing Municipality | 100010 | 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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Part A-Open-label Sequential, Part B-Double-blind Parallel
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Part A-Open-label, Part B-Double-blind
| Placebo | Drug | Placebo will be administered orally as capsules in a 28-day cycle. |
|
The severity was graded based on the National Cancer Institute's Common Terminology Criteria for Adverse Events.
| 1 year |
| Part A and Part B: Overall response rate | The proportion of patients with a platelet count ≥ 100 × 10⁹/L on 2 consecutive visits at least 7 days apart and without bleeding | up to 24 weeks |
| Part A and Part B: Complete response rate | The proportion of patients with a platelet count ≥ 100 × 10⁹/L on 2 consecutive visits at least 7 days apart and without bleeding | up to 24 weeks |
| Part A and Part B: Early response rate | The proportion of patients with a platelet count ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding within 1 week | up to 1 week |
| Part A and Part B: Initial response rate | The proportion of patients with a platelet count ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding within 4 weeks | up to 4 weeks |
| Part A and Part B: Sustained response rate | The proportion of patients with a platelet count ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding between weeks 20 and 24 | up to 24 weeks |
| Part A and Part B: Time to response | Time to first platelet counts ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding | up to 24 weeks |
| Part A and Part B: Cumulative response time | Number of cumulative days with platelet counts ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding by Week 24 | up to 24 weeks |
| Part A and Part B: The proportion of patients requiring rescue therapy | up to 24 weeks |
| Part A and Part B: The proportion of patients experencing bleeding (≥ Grade 2) | up to 24 weeks |
| Part A and Part B: Changes of ITP-BAT bleeding score | up to 24 weeks |
| 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 |