An Exploratory Study of Golidocitinib in Adult Patients With ITP

Phase 2Not Yet RecruitingNCT07196163

Peking University People's Hospital138 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

138

Conditions

Immune Thrombocytopenia (ITP)

Interventions

GolidocitinibDRUG

Golidocitinib will be administered orally as capsules in a 28-day cycle.

PlaceboDRUG

Placebo will be administered orally as capsules in a 28-day cycle.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

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.

Locations (1)

Peking University Insititute of Hematology, Peking University People's Hospital

Beijing, Beijing Municipality, China

Outcomes

Primary Outcomes

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.

Time frame: 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

Time frame: up to 24 weeks

Secondary Outcomes

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

Time frame: up to 24 weeks

Part B: Incidence and severity of Adverse Events (AEs)

The severity was graded based on the National Cancer Institute's Common Terminology Criteria for Adverse Events.

Time frame: 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

Time frame: 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

Time frame: 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

Data from ClinicalTrials.gov

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