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NCT Number: NCT07196163

An Exploratory Study of Golidocitinib in Adult Patients With ITP

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.

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Key information

Conditions

Immune Thrombocytopenia (ITP) Autoimmune Diseases Blood Coagulation Disorders Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders Immune System Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Purpura Purpura, Thrombocytopenic Purpura, Thrombocytopenic, Idiopathic Signs and Symptoms Skin Manifestations Thrombocytopenia Thrombotic Microangiopathies

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

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

Beijing, Beijing Municipality, 100010, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

golidocitinib

Drug

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

Placebo

Drug

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

Primary outcomes

  1. 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.

  2. 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

  1. Part A: 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

  2. Part B: 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.

  3. Part A and Part B: Overall response rate

    Time frame: 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

  4. Part A and Part B: Complete response rate

    Time frame: 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

  5. Part A and Part B: Early response rate

    Time frame: 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

  6. Part A and Part B: Initial response rate

    Time frame: 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

  7. Part A and Part B: Sustained response rate

    Time frame: 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

  8. Part A and Part B: Time to response

    Time frame: up to 24 weeks

    Time to first platelet counts ≥ 30 × 10⁹/L and doubling of the baseline count and without bleeding

  9. Part A and Part B: Cumulative response time

    Time frame: 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

  10. Part A and Part B: The proportion of patients requiring rescue therapy

    Time frame: up to 24 weeks

  11. Part A and Part B: The proportion of patients experencing bleeding (≥ Grade 2)

    Time frame: up to 24 weeks

  12. Part A and Part B: Changes of ITP-BAT bleeding score

    Time frame: up to 24 weeks

Sponsors and collaborators

Lead sponsor

Peking University People's Hospital

Other

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2028
Study completion
2029
First posted
Sep 29, 2025
Registry last updated
Sep 29, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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