Immune Thrombocytopenia (ITP)
Conditions
Brief summary
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.
Interventions
DRUGGolidocitinib
Golidocitinib will be administered orally as capsules in a 28-day cycle.
DRUGPlacebo
Placebo will be administered orally as capsules in a 28-day cycle.
Sponsors
Peking University People's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
Part A-Open-label, Part B-Double-blind
Intervention model description
Part A-Open-label Sequential, Part B-Double-blind Parallel
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Male and female participants between 18 and 80 years old. * Primary ITP for \>3 months. * An average of two platelet counts (⩾ 7 days apart) of \< 30 × 10⁹/L. * Have relapsed or treatment-resistant to previous ITP therapies, including corticosteroids and at least one other ITP therapy. * Have history of response to previous treatments. * Adequate hematologic, hepatic, and renal fuction. * Participants willing to comply with contraceptive restrictions.
Exclusion criteria
* Diagnosed with secondary immune thrombocytopenia, or there is evidence that the patient has a secondary cause of immune thrombocytopenia. * Previous or current therapy and comedications meet
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Part A: Incidence and severity of Adverse Events (AEs) | 1 year | The severity was graded based on the National Cancer Institute's Common Terminology Criteria for Adverse Events. |
| Part B: Durable response rate | up to 24 weeks | The proportion of patients with a platelet count ≥ 50 × 10⁹/L on at least four of six scheduled visits between weeks 14 and 24 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Part A and Part B: Cumulative response time | up to 24 weeks | Number of cumulative days with platelet counts ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding by Week 24 |
| 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: Durable response rate | up to 24 weeks | The proportion of patients with a platelet count ≥ 50 × 10⁹/L on at least four of six scheduled visits between weeks 14 and 24 |
| Part B: Incidence and severity of Adverse Events (AEs) | 1 year | The severity was graded based on the National Cancer Institute's Common Terminology Criteria for Adverse Events. |
| Part A and Part B: Sustained response rate | up to 24 weeks | 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 |
| Part A and Part B: Complete response rate | up to 24 weeks | The proportion of patients with a platelet count ≥ 100 × 10⁹/L on 2 consecutive visits at least 7 days apart and without bleeding |
| Part A and Part B: Early response rate | up to 1 week | The proportion of patients with a platelet count ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding within 1 week |
| Part A and Part B: Initial response rate | up to 4 weeks | The proportion of patients with a platelet count ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding within 4 weeks |
| Part A and Part B: Changes of ITP-BAT bleeding score | up to 24 weeks | — |
| Part A and Part B: Overall response rate | up to 24 weeks | The proportion of patients with a platelet count ≥ 100 × 10⁹/L on 2 consecutive visits at least 7 days apart and without bleeding |
| Part A and Part B: Time to response | up to 24 weeks | Time to first platelet counts ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding |
Countries
China
Outcome results
None listed