High-Dose Dexamethasone Combined With Orelabrutinib Versus High-Dose Dexamethasone Combined With Placebo in Adult Patients With Newly Diagnosed Primary Immune Thrombocytopenia

A Randomized Controlled Study of High-Dose Dexamethasone Combined With Orelabrutinib Versus High-Dose Dexamethasone Combined With Placebo in Adult Patients With Newly Diagnosed Primary Immune Thrombocytopenia

This is a randomized controlled study of high-dose Dexamethasone combined with Orelabrutinib versus high-dose Dexamethasone combined with placebo in adult patients with newly diagnosed Primary Immune Thrombocytopenia.

Study Overview

• Status: Recruiting
• Conditions: Primary Immune Thrombocytopenia (ITP)
• Intervention / Treatment: Drug: High-Dose Dexamethasone Combined with Placebo; Drug: High-Dose Dexamethasone Combined with Orelabrutinib

• Study Type: Interventional
• Enrollment (Estimated): 86
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Min Hou, Professor; Phone Number: 18560087007; Email: qlhouming@sina.com

Study Locations

• China
  • Shandong
    • Jinan, Shandong, China
      • Recruiting
      • Qilu Hospital of Shandong University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Subjects must thoroughly understand the nature, significance, potential benefits, possible inconveniences, and potential risks of the trial prior to enrollment. They must understand the study procedures and voluntarily sign the informed consent form.
2. Male or female subjects aged 18-80 years (inclusive).
3. Body weight ≥35 kg at screening.
4. Adult patients with newly diagnosed, untreated primary ITP, with platelet count (PLT) <30×10⁹/L and no active bleeding in vital organs.
5. Subjects who responded to prior treatment with oral dexamethasone 40 mg/day for 4 days, defined as meeting all three of the following criteria on any day between Day 5 and Day 7 post-treatment: ① PLT ≥30×10⁹/L; ② ≥2-fold increase from baseline; and ③ no active bleeding. Between Week 2 and Week 12, any abnormal PLT result meeting any of the following criteria must be confirmed by repeat testing at Qilu Hospital within 24-48 hours: ① PLT <30×10⁹/L; ② <2-fold increase from baseline; or ③ active bleeding.
6. Women of childbearing potential must use an effective method of contraception during the screening period, throughout the entire trial, and for 90 days following the last dose of study medication.

Exclusion Criteria:

1. Subjects with severe ITP at screening (e.g., life-threatening thrombocytopenia, major bleeding events, or requiring urgent treatment including intravenous immunoglobulin, high-dose glucocorticoids, or plasma exchange), whom the investigator anticipates will require rescue treatment within 2 weeks after enrollment.
2. Subjects with autoimmune systemic diseases other than ITP, unless the investigator determines that such conditions will not affect the evaluation of study outcomes.
3. Subjects who failed to respond to prior treatment with oral dexamethasone 40 mg/day for 4 days, defined as meeting any of the following criteria on any day between Day 5 and Day 7 post-treatment: ① PLT <30×10⁹/L; ② <2-fold increase from baseline; or ③ active bleeding.
4. History of intracranial hemorrhage within 6 months prior to screening.
5. Subjects with a history of coagulation disorders other than ITP, such as disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura.
6. Subjects with a known hypersensitivity to any component of the study drugs described in this protocol.
7. Known human immunodeficiency virus (HIV) infection, or positive serologic test results.
8. Subjects with positive tuberculosis screening test (based on interferon-gamma release assay, including T-SPOT®, etc.), or active, latent, or incompletely appropriately treated tuberculosis at screening.
9. Activated partial thromboplastin time (aPTT) ≥1.5× upper limit of normal (ULN) or international normalized ratio (INR) ≥1.5 at screening.
10. Organ dysfunction, with the following laboratory findings at screening: Absolute neutrophil count (ANC) <1.5×10⁹/L; hemoglobin <90 g/L; lymphocyte count <0.8×10⁹/L. Total bilirubin >1.2×ULN; aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >ULN. Amylase or lipase >2×ULN. Estimated glomerular filtration rate (eGFR) <40 mL/min/1.73 m² calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula. Immunoglobulin IgG <6 g/L.
11. Pregnant or lactating women.
12. Subjects unable to undergo blood collection, or with contraindications to phlebotomy.
13. Any other condition that the investigator considers unsuitable for participation in this trial.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Control Group; Dexamethasone plus Placebo Group	Drug: High-Dose Dexamethasone Combined with Placebo; Placebo 50 mg once daily (QD) (for 3 consecutive months) combined with Dexamethasone 40 mg/day for 4 days
Experimental: Experimental Group; High-Dose Dexamethasone Combined with Orelabrutinib	Drug: High-Dose Dexamethasone Combined with Orelabrutinib; Orelabrutinib 50 mg once daily (QD) (for 3 consecutive months) combined with Dexamethasone 40 mg/day for 4 days

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Overall Response Rate (ORR)	Throughout the study period, an average of 6 months

Secondary Outcome Measures

Outcome Measure	Time Frame
Overall Response Rate (ORR)	Throughout the study period, an average of 1/2/3 months

Other Outcome Measures

Outcome Measure	Time Frame
Occurrence of adverse events and serious adverse events according to CTCAE V5.0	Up to 2 years

Collaborators and Investigators

• Sponsor: Shandong University

Study record dates

Study Major Dates

• Study Start (Actual): April 8, 2026
• Primary Completion (Estimated): April 8, 2027
• Study Completion (Estimated): December 31, 2028

Study Registration Dates

• First Submitted: April 23, 2026
• First Submitted That Met QC Criteria: April 23, 2026
• First Posted (Actual): April 30, 2026

Study Record Updates

• Last Update Posted (Actual): April 30, 2026
• Last Update Submitted That Met QC Criteria: April 23, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Thrombocytopenia; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Hemic and Lymphatic Diseases; Purpura, Thrombocytopenic, Idiopathic; orelabrutinib
• Other Study ID Numbers: KYLL-2026-02-006-1

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No