An Exploratory Study of Golidocitinib in Adult Patients With ITP

NCT07196163 | Not Yet Recruiting Phase 1

• 1 other identifier: DZ2024J0003
• Study Type: interventional
• Target: 138
• Locations: 1 country
• Sites: 1

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.

Conditions

Immune Thrombocytopenia (ITP)

Outcome Measures

Primary Outcomes (2)

• 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.

  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

  up to 24 weeks

Secondary Outcomes (12)

• 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

Study Arms (3)

Golidocitinib Part B

EXPERIMENTAL

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

Drug: Golidocitinib

Placebo Part B

PLACEBO COMPARATOR

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

Drug: Placebo

Golidocitinib Part A

EXPERIMENTAL

Golidocitinib dose escalation, administered orally, once daily.

Drug: Golidocitinib

Interventions

Golidocitinib DRUG

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

Golidocitinib Part A; Golidocitinib Part B

Placebo DRUG

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

Placebo Part B

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• Diagnosed with secondary immune thrombocytopenia, or there is evidence that the patient has a secondary cause of immune thrombocytopenia.
• 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.

Sponsors & Collaborators

• Peking University People's Hospital: lead

Study Sites (1)

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

Beijing, Beijing Municipality, 100010, China

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Part A-Open-label, Part B-Double-blind
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Director

Model Details: Part A-Open-label Sequential, Part B-Double-blind Parallel

Study Record Dates

• First Submitted: September 19, 2025
• First Posted: September 29, 2025
• Study Start: October 30, 2025
• Primary Completion (Estimated): October 30, 2028
• Study Completion (Estimated): October 30, 2029
• Last Updated: September 29, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)