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Clinical Trials/NCT07196163
NCT07196163Not yet recruitingPhase 1

An Exploratory Clinical Study of Golidocitinib in Adult Patients With Primary Immune Thrombocytopenia (ITP)

Peking University People's Hospital1 site in 1 country138 target enrollmentStarted: October 30, 2025Last updated:
Interventions

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

Experimental

Golidocitinib at recommended phase 2 doses, administered orally, once daily.

Intervention: Golidocitinib (Drug)

Placebo Part B

Placebo Comparator

Placebo at recommended phase 2 doses, administered orally, once daily.

Intervention: Placebo (Drug)

Golidocitinib Part A

Experimental

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

Sponsor Class
Other
Responsible Party
Principal Investigator
Principal Investigator

Xiao Hui Zhang

Associate Director

Peking University People's Hospital

Study Sites (1)

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