An Exploratory Study of Golidocitinib in Adult Patients With ITP
- Conditions
- Immune Thrombocytopenia (ITP)
- Interventions
- Drug: Placebo
- Registration Number
- NCT07196163
- Lead Sponsor
- Peking University People's Hospital
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 138
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Golidocitinib Part B Golidocitinib Golidocitinib at recommended phase 2 doses, administered orally, once daily. Placebo Part B Placebo Placebo at recommended phase 2 doses, administered orally, once daily. Golidocitinib Part A Golidocitinib Golidocitinib dose escalation, administered orally, once daily.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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: 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: 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: 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: Time to response up to 24 weeks Time to first platelet counts ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding
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 and Part B: Changes of ITP-BAT bleeding score up to 24 weeks
Trial Locations
- Locations (1)
Peking University Insititute of Hematology, Peking University People's Hospital
🇨🇳Beijing, Beijing Municipality, China
Peking University Insititute of Hematology, Peking University People's Hospital🇨🇳Beijing, Beijing Municipality, China