Low-dose Baricitinib Plus High-dose Dexamethasone for Patients With Newly Diagnosed Immune Thrombocytopenia

NCT05932524 | Unknown Phase 2 DMC

• 1 other identifier: PKU-BAITP-03
• Study Type: interventional
• Target: 132
• Locations: 1 country
• Sites: 10

Brief Summary

A randomized, open-label, multicenter, phase 2 trial to compare the efficacy and safety of baricitinib plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).

Conditions

Immune Thrombocytopenia

Keywords

Baricitinib; High-dose dexamethasone

Outcome Measures

Primary Outcomes (1)

• Durable response

  The maintenance of a platelet count ≥30,000/μL, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up.

  6 months

Secondary Outcomes (9)

• Complete response (CR)

  1 month

• Response (R)

  1 month

• Time to response

  6 months

• Duration of response

  6 months

• Early response

  7 days

Study Arms (2)

Low-dose baricitinib plus high-dose dexamethasone

EXPERIMENTAL

Oral baricitinib is given at a dose of 2 mg daily for 6 consecutive months. Dexamethasone is administrated at 40 mg per day for 4 consecutive days (the 4-day course of dexamethasone will be repeated in the case of lack of response by day 10). Treatment will be discontinued if very severe or life-threatening adverse events developed or at the patients' request.

Drug: Baricitinib 2 MG; Drug: Dexamethasone

High-dose dexamethasone

ACTIVE COMPARATOR

Dexamethasone is administrated at 40 mg per day for 4 consecutive days (the 4-day course of dexamethasone will be repeated in the case of lack of response by day 10). Treatment will be discontinued if very severe or life-threatening adverse events developed or at the patients' request.

Drug: Dexamethasone

Interventions

Baricitinib 2 MG DRUG

Baricitinib 2 mg q.d., p.o., for 6 consecutive months.

Also known as: Olumiant

Low-dose baricitinib plus high-dose dexamethasone

Dexamethasone DRUG

Dexamethasone 40 mg q.d. for 4 consecutive days (the 4-day course of dexamethasone will be repeated in the case of lack of response by day 10)

Also known as: HD-DXM

High-dose dexamethasone; Low-dose baricitinib plus high-dose dexamethasone

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Confirmed newly-diagnosed, treatment-naive ITP;
• A platelet count \<30,000/μL, or a platelet count \<50,000/μL with clinically significant bleeding symptoms (WHO bleeding scale 2 or above) at the enrollment;
• Willing and able to sign written informed consent.

You may not qualify if:

• Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
• Have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct Coombs test;
• Active or a history of malignancy;
• Pregnancy or lactation;
• Received first-line and second-line ITP-modifying therapy;
• Previously received corticosteroids or immunosuppressive agents for non-ITP diseases within 6 months before enrollment;
• A history of clinically significant adverse reactions to previous corticosteroid therapy;
• Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia);
• Current or recent (\<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection;
• A history of symptomatic herpes zoster infection within 12 weeks prior to screening;
• Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV);
• Have evidence of active tuberculosis (TB), or have previously had evidence of active TB and did not receive appropriate and documented treatment, or have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB;
• Have experienced a clinically significant thrombotic event within 24 weeks of screening or are on anticoagulants and in the opinion of the investigator are not well controlled;
• Myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage IV heart failure;
• A history or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, neurological, or neuropsychiatric disorders or any other serious and/or unstable illness that, in the opinion of the investigator, could constitute an unacceptable risk when taking investigational product or interfere with the interpretation of data;

Sponsors & Collaborators

• Peking University People's Hospital: lead
• Beijing Luhe Hospital: collaborator
• Chinese PLA General Hospital: collaborator
• Navy General Hospital, Beijing: collaborator
• Beijing Hospital: collaborator
• Beijing Friendship Hospital: collaborator
• Peking University First Hospital: collaborator
• Peking University Third Hospital: collaborator
• China-Japan Friendship Hospital: collaborator
• Beijing Tsinghua Changgeng Hospital: collaborator

Study Sites (10)

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

Beijing, 100044, China

RECRUITING

Beijing Friendship Hospital

Beijing, China

RECRUITING

Beijing Hospital

Beijing, China

RECRUITING

Beijing Luhe Hospital

Beijing, China

RECRUITING

Beijing Tsinghua Changgeng Hospital

Beijing, China

RECRUITING

China-Japan Friendship Hospital

Beijing, China

RECRUITING

Chinese PLA General Hospital

Beijing, China

RECRUITING

Peking University First Hospital

Beijing, China

RECRUITING

Peking University Third Hospital

Beijing, China

RECRUITING

The Sixth Medical Center of PLA General Hospital

Beijing, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

baricitinib; Dexamethasone

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

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated

Study Officials

• Xiaohui Zhang

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

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiaohui Zhang

CONTACT

+8613522338836; zhangxh@bjmu.edu.cn

Peng Zhao

CONTACT

+8618810323668; zpeng702@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Vice president of Peking Univeristy Institute of Hematology

Model Details: Patients are randomly assigned at a 1:1 ratio to receive baricitinib plus high-dose dexamethasone or high-dose dexamethasone alone. Each group requires 66 patients (considering 10% drop-off).

Study Record Dates

• First Submitted: June 28, 2023
• First Posted: July 6, 2023
• Study Start: July 7, 2023
• Primary Completion: June 1, 2024
• Study Completion: June 1, 2025
• Last Updated: July 12, 2023

Record last verified: 2023-07

Locations

CN (10)