An Exploratory Clinical Study of Golidocitinib in Adult Patients With Primary Immune Thrombocytopenia (ITP)
Trial Snapshot
- Phase
- Phase 1
- Status
- Not yet recruiting
- Enrollment
- 138
- Locations
- 1
- Primary Endpoint
- Part A: Incidence and severity of Adverse Events (AEs)
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description
Part A-Open-label, Part B-Double-blind
Eligibility Criteria
- Ages
- 18 Years to 80 Years (Adult, Older Adult)
- Sex
- All
- Accepts 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 exclusion criteria.
- •Patients with major caridiovascular disease, active infetion, maligancy or uncontrolled systemic disease.
- •Women who are breast feeding.
- •History of hypersensitivity to sudy drug with a similar chemical structure or class.
- •Previously received JAK inhibitors.
Arms & Interventions
Golidocitinib Part B
Golidocitinib at recommended phase 2 doses, administered orally, once daily.
Intervention: Golidocitinib (Drug)
Placebo Part B
Placebo at recommended phase 2 doses, administered orally, once daily.
Intervention: Placebo (Drug)
Golidocitinib Part A
Golidocitinib dose escalation, administered orally, once daily.
Intervention: Golidocitinib (Drug)
Outcomes
Primary Outcomes
Part A: Incidence and severity of Adverse Events (AEs)
Time Frame: 1 year
The severity was graded based on the National Cancer Institute's Common Terminology Criteria for Adverse Events.
Part B: Durable response rate
Time Frame: 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 Outcomes
- Part A: Durable response rate(up to 24 weeks)
- Part B: Incidence and severity of Adverse Events (AEs)(1 year)
- Part A and Part B: Overall response rate(up to 24 weeks)
- Part A and Part B: Complete response rate(up to 24 weeks)
- Part A and Part B: Early response rate(up to 1 week)
- Part A and Part B: Initial response rate(up to 4 weeks)
- Part A and Part B: Sustained response rate(up to 24 weeks)
- Part A and Part B: Time to response(up to 24 weeks)
- Part A and Part B: Cumulative response time(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)
Investigators
Xiao Hui Zhang
Associate Director
Peking University People's Hospital